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Displaying 1 - 200 of 252

Genome Research in African American Scleroderma Patients (GRASP Study)

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Currently Recruiting
This research is being done to determine how variations in genes may explain the different risk to develop scleroderma (or CREST) seen in African American patients compared to other populations. People who are African American with scleroderma (or CREST) may join. This research involves a blood draw and physical exams.
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A Clinical Research Study of Nucresiran for Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN)

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
TRITON-PN is a global Phase 3 randomized, open-label study designed to evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN). The study will also evaluate any changes in quality of life experienced by study participants. Nucresiran is a TTR silencer that aims to prevent…
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A Research Study to Learn Whether DNTH103 Is Safe and Effective for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
This is a global, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of DNTH103 in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The study is comprised of a Part A and a Part B and an Optional open label extension. Treatment duration will be 65 weeks with an Optional open…
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Cognitive Training

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Currently Recruiting
People with mild cognitive impairment (MCI) are at increased risk of dementia, including Alzheimer's disease. Cognitive training may be a useful treatment strategy in MCI. The COGIT-2 study, which is funded by the National Institutes of Health, will compare the effects on cognition and function and brain MRI changes of high dose crosswords (4 puzzles…
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Pathway to Prevention of Type 1 Diabetes (T1D)- Pediatrics

Condition: Pediatrics
Investigator: Robin Goland, MD
Status: Currently Recruiting
A future without Type 1 Diabetes (T1D) starts with you. Get screened! Thanks in large part to T1D family members, we understand T1D as a disease that progresses in three distinct stages. This new definition in conjunction with TrialNet's Pathway to Prevention screening, allows for earlier detection and intervention.
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Phase 3 Study Evaluating Suzetrigine for Pain Caused by Diabetic Peripheral Neuropathy

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
The purpose of this study is to evaluate the safety, tolerability and effectiveness of VX-548 in participants with pain due to Diabetic Peripheral Neuropathy (DPN). To be in this study you must have DPN and be 18 through 80 years of age, inclusive, with presence of bilateral pain in lower extremities due to DPN for at least 1 year.
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Phase 3 Study Comparing Riliprubart and Intravenous Immunoglobulin (IVIg) in People with CIDP

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
This is a Phase 3, randomized, double blind, study evaluating efficacy and safety of riliprubart versus intravenous immunoglobulin (IVIg) in participants with chronic inflammatory demyelinating polyneuropathy. Overall the study will last about 109 weeks (2 years). Participants must be 18 years old at the time of signing the informed consent form.
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A Natural History Study of HNRNP-Related Disorders

Condition: Neurological Disorders
Investigator: Jennifer Bain, MD
Status: Currently Recruiting
A natural history study of hnRNP and other genetic neurodevelopmental disorders.
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BEELINE: a Phase 1b/3 Study in GRIN-related Neurodevelopmental Disorder

Condition: Neurological Disorders
Investigator: Jennifer Bain, MD
Status: Currently Recruiting
The purpose of this study is to investigate the long-term safety, effectiveness, and tolerability of Radiprodil, which is an investigational drug for the treatment of GRIN related Neurodevelopmental Disorder (GRIN-NDD). The study is broken up into 2 parts: Part A, which includes Screening, Medication Titration, and Maintenance periods and lasts 20-35 weeks…
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A Study to Test the Effects and Safety of Riliprubart in People with Chronic CIDP for Which the Usual Treatment Does Not Work

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
This is a global, multicenter, placebo-controlled, phase 3 study evaluating the efficacy and safety of riliprubart in adult participants with CIDP who are refractory or had an insufficient response to standard of care (SOC) therapies, defined as Ig administered IV (IVIg) or SC (SCIg), or corticosteroids. Treatment duration will be 48 weeks. Participant must…
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ALS Families Project

Condition: Neurological Disorders / Neuromuscular
Investigator: Matthew Harms, MD
Status: Currently Recruiting
This program provides family members of individuals with familial ALS the opportunity to contribute to research focused on learning more about why motor neuron degeneration begins and how or why it progresses. This study provides genetic counseling and testing to help participants understand and manage their risk and determine if they want to learn their…
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AutoImmune Hemolytic Anemia (AIHA) Registry

Condition: Autoimmune Disorders
Investigator: Eldad Hod, MD
Status: Currently Recruiting
We are studying the natural course of autoimmune hemolytic anemia (AIHA). If you are an adult between 18 and 120 years old, receive care at Columbia University Irving Medical Center-New York Presbyterian Hospital, are willing to provide a blood sample every 6 months for research, and have one of the following diagnoses: AIHA, mixed AIHA, Evans Syndrome, or…
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CAUSE: Childhood Asthma in Urban Settings

Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Currently Recruiting
We are interested in learning more about these diseases and how to treat them. Our research program is conducting several studies including: a study to learn if treatment with a drug named dupilumab can help reduce asthma attacks in children, a study to observe a specific type of irritation in the lungs and how it relates to asthma symptoms, and more! If…
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IntraVenous iron in Kids with Iron Deficiency and Scoliosis (IV KIDS) Study

Condition: Pediatrics
Investigator: Lisa Eisler, MD
Status: Currently Recruiting
We are studying the effects of iron supplementation in patients undergoing surgery for scoliosis. If you are scheduled or are planning to schedule a scoliosis surgery, please contact us to determine eligibility.
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Participate in Learning More About the Physiology of Essential Tremor

Condition: Neurological Disorders
Investigator: Sheng-Han Kuo, MD
Status: Currently Recruiting
Participate in learning more about the physiology of Ataxia or Essential Tremor to evaluate how brain networks are affected when frequency-specific, non-invasive, brain stimulation is applied to people with ataxia or essential tremor as compared to healthy control subjects. This study is for adults over the age of 18, diagnosed with Ataxia or Essential…
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DHREAMS: A genetic study of congenital diaphragmatic hernia (CDH)

Condition: Pediatrics
Investigator: Yufeng Shen, PhD
Status: Currently Recruiting
Each year more than 1,000 babies in the United States are born with a Congenital Diaphragmatic Hernia (CDH). For the majority of these babies, the cause of the CDH is unknown. The DHREAMS (Diaphragmatic Hernia Research & Exploration; Advancing Molecular Science) study was created to improve the understanding of the molecular genetic basis of CDH. This…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Neurological Disorders / Dementia
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Neurological Disorders
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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Kids with Iron Deficiency and Scoliosis (KIDS) Study

Condition: Pediatrics / Child and Adolescent Health
Investigator: Lisa Eisler, MD
Status: Currently Recruiting
We are studying the effects of iron supplementation in patients undergoing surgery for scoliosis. If you are scheduled or are planning to schedule a scoliosis surgery in greater than 3 months, please contact us to determine eligibility.
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Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias (Children and Young Adults)

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
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Genetics of Early Childhood Obesity (GECO) Study at Columbia

Condition: Pediatrics
Investigator: Vidhu Thaker, MD
Status: Currently Recruiting
We will enroll children with severe early onset obesity (BMI greater than 99th percentile) diagnosed prior to 6 years of age or other endocrine disorders. We will ask questions about their health, eating behavior and perform a brief physical examination. We will collect saliva or blood to perform genetic testing. We will also invite family members to…
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CONQUER Registry to Track Disease Progression in Systemic Sclerosis (Scleroderma)

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Currently Recruiting
The purpose of this study is to develop a cohort of patients with early scleroderma and to collect data on clinical outcomes, radiological tests, laboratory tests, and to obtain biological specimens for testing.
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Non-Invasive ultrasound treatment for low back and leg pain

Condition: Neurological Disorders / Neuromuscular
Investigator: Clark Smith, MD
Status: Currently Recruiting
The purpose of this research is to study whether Discogen's non-invasive, low-intensity pulsed ultrasound treatment can help reduce pain for people with leg pain and low back pain. Eligible participants will be randomized into either a Discogen treatment group or a sham control group. All participants will be asked to come to the Columbia University…
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Low Field MRI Study

Condition: Neurological Disorders
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
We're testing a new type of MRI scanner that's smaller and portable - like a mobile MRI unit. This makes it easier to provide brain scans to people who have difficulty traveling to hospitals or live far from medical centers. We'll be studying people with multiple sclerosis (MS) and similar brain conditions. Our goal is to learn how well this…
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TReatment for ImmUne Mediated PathopHysiology (TRIUMPH)

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Currently Recruiting
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
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Study of People with First-Degree Relatives (Parents, Siblings, Children) with Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
Genes and Environment in MS (GEMS) is a longitudinal cohort study investigating how genetics and environment can lead to the onset of Multiple Sclerosis. We are recruiting first-degree relatives (siblings, parents, and children) of people who have been diagnosed with MS. This study involves the collection of magnetic resonance imaging (MRI) of the brain,…
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Immune Responses in Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Julian Agin-Liebes, MD
Status: Currently Recruiting
We are looking for participants for a study on the role of immune response in Parkinsons disease (PD) and Alzheimer's disease (AD). We are inviting people WITH PD, WITH AD, WITH amnestic Mild Cognitive Impairment or WITHOUT PD or AD to come and participate. We will be doing several questionnaires, a motor exam, and a short cognitive assessment, in…
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Immune Responses in Parkinson's Disease

Condition: Neurological Disorders / Parkinson's Disease
Investigator: Julian Agin-Liebes, MD
Status: Currently Recruiting
We are looking for participants for a study on the role of immune response in Parkinsons disease (PD) and Alzheimer's disease (AD). We are inviting people WITH PD, WITH AD, WITH amnestic Mild Cognitive Impairment or WITHOUT PD or AD to come and participate. We will be doing several questionnaires, a motor exam, and a short cognitive assessment, in…
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Multiple Sclerosis (MS) Genetics

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
MS Genetics aims to better understand the genetic component of demyelinating diseases by doing genetic testing on blood samples of participants. This study involves a one-time blood draw and completion of questionnaires spread out over a couple of years in hopes to better understand the genes that makes one predisposed to Multiple Sclerosis and various…
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Alzheimer's Variant Imaging Study (ADVISe)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Patrick Lao, PhD
Status: Currently Recruiting
The purpose of this study is to determine how inflammation is related to changes in the brain that occur during the progression of Alzheimer's disease. If you are eligible for this study, we will ask you to complete 3-4 study visits over the course of about 5 months. Participation includes memory testing, a blood draw, MRI, PET scans, and a lumbar…
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A Study to Identify Genes Underlying Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Richard Mayeux, MD, MSc
Status: Currently Recruiting
The goal of this study is to focus on the genetic influences on Alzheimer's disease (AD) risk. We are looking for families and/or individuals (affected or unaffected) of any ethnic background with a family history of Alzheimer's disease and willing to participate.
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Safety Planning Study for Elementary School-Aged Children (Ages 6-12)

Condition: Pediatrics
Investigator: Liat Itzhaky, PhD
Status: Currently Recruiting
Your child is invited to participate in a research study. Participation in the study includes one meeting at Washington Heights. Results from this study will assist in modifying an existing behavioral intervention to fit children of this age. For more details, please contact Dr. Liat Itzhaky at 646-774-7634 or e-mail: li2185@cumc.columbia.edu. Participants…
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Impulsivity and Compulsivity in Spinocerebellar ataxia (SCA) - Healthy Controls

Condition: Neurological Disorders
Investigator: Sheng-Han Kuo, MD
Status: Currently Recruiting
Participate in learning more about Impulsivity and Compulsivity in Spinocerebellar ataxia (SCA). Why? By investigating individuals with SCA, we will gain valuable insights into the specific mechanisms through which the cerebellum participates in cognitive processes. Who? Healthy adults over the age of 18, who would like to participate as controls. What?…
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The Child OCD Study

Condition: Pediatrics
Investigator: Rachel Marsh, PhD
Status: Currently Recruiting
This research study will examine how an iPad-based cognitive training game combined with cognitive behavioral therapy (CBT) helps children with excessive worries and related repetitive behaviors (obsessive compulsive disorder). After completing the training, all children will be offered CBT, delivered by experts at no cost. This study will take place at…
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Assessment of Pruritis (Itch) in Children with Primary Sclerosing Cholangitis

Condition: Pediatrics
Investigator: Mercedes Martinez, MD
Status: Currently Recruiting
The purpose of this study is to better understand how common and how severe pruritis (itching) is in children and adults under 21 years of age with Primary Sclerosing Cholangitis (PSC). The study involves the completion of two short online surveys. If you have any questions regarding this study, please contact the Transplant Clinical Research Center ( TCRC).
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The Mindful Brain Project in Teens

Condition: Pediatrics
Investigator: Randy Auerbach, PhD
Status: Currently Recruiting
We are recruiting depressed adolescents ages 13-18 years. As a participant in our study, we will: (1) interview your child about current and past thoughts, feelings, and behaviors, (2) teach your child mindfulness strategies, (3) collect brain activity data with MRI (non-invasive) while your child practices mindfulness, and (4) send short surveys to your…
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Investigating Brain Changes in People with Cerebral Palsy

Condition: Neurological Disorders / Cerebral Palsy
Investigator: Jason Carmel, MD, PhD
Status: Currently Recruiting
A key part of the CP definition is that it is a non-progressive brain injury. However, as people with CP age, their function often deteriorates. This study seeks to determine whether changes in the brain structure may result in function changes, as they do in other types of brain injuries.
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Walking and Memory Study

Condition: Neurological Disorders
Investigator: Shayan Shirazian, MD
Status: Currently Recruiting
Columbia University, School of Nursing is conducting a walking research study for persons aged between 55 years or older. You may be eligible if you answer YES to the following question: "Do you feel like your memory or thinking skills have gotten worse over the past few years?"
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Resource for Early Onset Alzheimer's Disease Research (READR)

Condition: Neurological Disorders / Dementia
Investigator: Christiane Reitz, MD, PhD
Status: Currently Recruiting
We are recruiting individuals and/or families with members under the age of 65 who have dementia or are experiencing memory problems. Our initial screening can be completed in person or over the phone. The evaluation will take about 1 to 1.5 hours per person and includes a: 1. Blood sample (5 tablespoons) or saliva (1 teaspoon) collection: These blood…
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Alzheimer's Disease Research Center (ADRC)

Condition: Neurological Disorders / Dementia
Investigator: Scott Small, MD
Status: Currently Recruiting
For over 30 years, The Columbia University Alzheimer's Disease Research Center (ADRC) has worked with thousands of participants who have helped provide important insights into how the brain ages and why Alzheimer's disease may develop. We are actively recruiting adults 65 years of age and older who share our commitment to be part of this…
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Study of Memory Loss in Patients with Alzheimers Disease or Mild Cognitive Impairment (MCI)

Condition: Neurological Disorders
Investigator: Catherine Clelland, PhD
Status: Currently Recruiting
This research study aims to obtain a greater understanding of the causes of cognitive decline in Alzheimer's disease. We are currently enrolling male and female participants (ages 55 years and older) with Alzheimer's disease, mild cognitive impairment, or healthy controls. Enrolled participants with undergo a small blood draw (about 8 teaspoons)…
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CHORD Clinical Trial - Gene Therapy DB-OTO for Children with Hearing Loss from Otoferlin (OTOF) Gene Mutation

Condition: Pediatrics
Investigator: Lawrence Lustig, MD
Status: Currently Recruiting
The CHORD clinical trial, sponsored by Decibel Therapeutics, is looking at the use of the gene therapy DB-OTO for children born with hearing loss related to changes in the OTOF gene. The goal of this trial is to assess different doses of DB-OTO to find out if it is safe, well-tolerated, and its effect on improving hearing. DB-OTO is a gene therapy that has…
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Congenital Anomalies Research Exploration - CARE study

Condition: Pediatrics
Investigator: Yufeng Shen, PhD
Status: Currently Recruiting
Study examining the genetic causes of birth defects
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Cerebral Palsy Patient Registry

Condition: Neurological Disorders / Cerebral Palsy
Investigator: Jason Carmel, MD, PhD
Status: Currently Recruiting
The Weinberg Family Cerebral Palsy Center's (WFCPC) research team maintains a cerebral palsy (CP) patient registry of more than 1,000 enrolled patients. This registry contains information collected from children and adults with CP. The goal of the registry is to help us learn more about CP and neuromuscular diseases, to understand the quality of life…
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The Dawn Study - Genetics of Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Giuseppe Tosto, MD PhD
Status: Currently Recruiting
This study aims to find genes that cause Alzheimer's Disease in African Americans and Hispanics/Latinos. Participants are eligible if African American or Hispanic/Latino and 60 years of age or older. Study participation would entail completing a memory assessment, having their blood drawn, and a brief physical exam with a questionnaire. Compensation of…
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COVID-19 Vaccine Biomarker Study in Multiple Sclerosis and Other Neuroimmunological Diseases

Condition: Neurological Disorders
Investigator: Rebecca Straus Farber, MD
Status: Currently Recruiting
We need your help today to gather information about the experience of patients with autoimmune diseases of the central nervous system (multiple sclerosis (MS), neuromyelitis optica (NMO), and anti-MOG associated disease (MOGAD)) in regards to COVID19 vaccines. Because individuals with MS, NMO, and MOGAD were not included in the COVID19 vaccine trials, we…
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MS Snapshot: A Study to Better Understand Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Philip De Jager, PhD
Status: Currently Recruiting
The Snapshot study aims to understand brain and spinal cord aging in healthy individuals and individuals with multiple sclerosis (MS). We aim to identify new tools that will help us to achieve successful brain aging in the general population and in people with an underlying autoimmune disease. A critical aspect of this study is that we are looking for…
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Family Nurture Intervention in the NICU

Condition: Pediatrics
Investigator: Martha Welch, MD
Status: Closed
The purpose of this study is to compare neurodevelopment and activity in infants born very preterm (26 to 33 6/7 weeks gestational age (GA)) receiving Standard Care (SC) or Family Nurture Intervention (FNI) in the neonatal intensive care unit (NICU). This study investigator hypothesizes that FNI will improve: i) neonatal electroencephalographic activity ii…
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A study for patients with temporal epilepsy using laser ablation

Condition: Neurological Disorders
Investigator: Guy McKhann, MD
Status: Closed
This research study plans to learn more about a device called the Visualase Thermal Therapy System (Visualase system). This device uses a thin laser probe that is inserted into the brain. The laser is turned on, heats up and destroys surrounding soft tissue. Before the surgeon turns on the laser, you will be in a magnetic resonance imaging (MRI) scanner.…
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PTC124-GD-041-DMD (ALSO KNOWN AS STUDY 041) CLINICAL TRIAL OF ATALUREN IN NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY (NMDMD)

Condition: Neurological Disorders / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Columbia University is currently enrolling in an Ataluren Study from PTC Therapeutics, called PTC-041. We are looking for 5 year old boys, ambulating, confirmed nonsense mutation DMD and on steroids for at least 12 months. If you would like more information about this please reach out to Ameneh Onativia, Columbia Sr. Clinical Research Coordinator II at 212…
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Study in Parkinson Disease of Exercise (SPARX3)

Condition: Neurological Disorders / Parkinson's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Closed
Most neurologists and other clinicians treating people with Parkinson's disease (PD) are still unclear about how best to prescribe exercise despite the fact that it is now axiomatic that exercise is therapeutically beneficial for PD. In contrast, drugs for PD are prescribed with care and specificity due to large scientific investigations showing…
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Cognition and Psychosocial Functioning During Puberty Blocking

Condition: Pediatrics / Child and Adolescent Health
Investigator: Ilene Fennoy, MD
Status: Closed
We know that puberty is a major time of change! Adolescents develop new decision-making skills and independence. It is possible that some of these changes are due to the hormonal changes during puberty. One of the ways we treat transgender and gender non-conforming teens is to suppress or "block" puberty. We know that this is a safe thing to do…
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A Study for Patients with Advanced Melanoma, Solid Tumors or Lymphoma Using Study Drug Pembrolizumab

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to determine if a study drug called pembrolizumab is safe and effective in the treatment of children and adolescents who have advanced melanoma, or advanced, relapsed, or refractory PD-L1 positive malignant solid tumor or other lymphoma, relapsed or refractory classical Hodgkin lymphoma (rrcHL), advanced, relapsed or refractory…
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DfHD - Dance for Huntington's Disease

Condition: Neurological Disorders / Huntington's Disease
Investigator: Ashwini Rao, EdD, OTR
Status: Closed
Dance is a complex activity involving the integration of rhythm, spatial patterns, synchronization to external stimuli and whole-body coordination. While regular exercise is often recommended for people with Huntington's Disease (HD), there are no studies examining the use of dance to improve balance and mobility and positively affect the quality of…
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ChANGE HD

Condition: Pediatrics
Investigator: Ashwini Rao, EdD, OTR
Status: Closed
Child to Adult Neurodevelopment in Gene Expanded Huntingtons Disease (ChANGE HD) HD has long been considered an adult-onset disease. However, research has shown that brain changes are evident before motor symptoms begin. In fact, the gene responsible for HD plays a role in brain development and is present throughout the lifespan. With gene knockdown therapy…
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BenfoTeam Trial: A new approach in early Alzheimer's disease research, including Mild Cognitive Impairment and early dementia

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Jose Luchsinger, MD
Status: Closed
The BenfoTeam clinical research trial is based on evidence from decades of research on the role of thiamine in Alzheimer's disease (AD). BenfoTeam scientists have found that thiamine (vitamin B1) does not enter the brains of people with early Alzheimer's disease despite having a sufficient supply of thiamine in their blood. Fortunately, there is…
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Learn more about Lock & Protect: Reducing Adolescent Access to Lethal Means for Suicide

Condition: Pediatrics
Investigator: Ashley Blanchard, MD
Status: Closed
The purpose of the study is to study how to make the home safer for youth at risk of suicide. Our goal is to learn how to better help families whose children are seen in the emergency room for self-injury or suicidal thoughts or actions.
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HDWI High Grade Glioma Brain MRI

Condition: Neurological Disorders / Brain Tumors
Investigator: Tony J. Wang, MD
Status: Closed
The purpose of this study is to find out if performing Magnetic Resonance Image (MRI) scans of your brain with adaptive planning during your radiation treatment of your high-grade glioma will help determine how sensitive the images collected of the brain are at detecting the response of the tumor to the cancer treatment. In this study, four or seven MRI…
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Live-Viral Vaccination after Pediatric Liver Transplantation

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
In this study, we will collect information from the medical record of pediatric liver transplant recipients who received live vaccines as part of their routine post-transplant care. This data will be combined with data from other centers across the United States and Canada to help give us a better understanding of the safety and efficacy of live vaccines in…
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Understanding the Causes of Apathy in Alzheimer's Disease (Male subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Catherine Clelland, PhD
Status: Closed
Apathy is a very common symptom experienced by people with Alzheimer's disease, and includes a lack of motivation and loss of interest in many activities. Patients and their caregivers often find apathy to be particularly distressing. This study aims to obtain a greater understanding of the causes of apathy in Alzheimer's disease. We are currently…
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STARZL Network for Excellence in Pediatric Transplantation (SNEPT)

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
In this research, we want to learn more about how to improve care for children who are listed for or who have received a liver transplant. The goal of this research is to look at how different hospitals treat these children and to look at their outcomes to help improve care, decrease complications, shorten length of hospital stay, reduce hospital re-…
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Study of Lutathera in Patients with Glioblastoma (GB)

Condition: Neurological Disorders / Brain Tumors
Investigator: Mikhail Doubrovin, MD
Status: Closed
The purpose of the study is to evaluate the safety of Lutathera alone in patients with recurrent glioblastoma and the combination of Lutathera with radiotherapy with temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB) and to establish the appropriate dose of this combination. Lutathera is a radioligand therapy, which is a targeted…
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Study to treat postural orthostatic tachycardia syndrome (POTS)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this research is to see if an experimental drug called REGN7544 is effective to treat individuals with postural orthostatic tachycardia syndrome (POTS). REGN7544 is an antibody. An antibody is a kind of protein that your immune (defense) system normally makes to fight bacteria and viruses in your body. Scientists can now make antibodies in…
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Study of OCR/RTX vs. Diroximel for Patients with Relapsing-Remitting Multiple Sclerosis (RRMS) or Clinically Isolated Syndrome (CIS)

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
The purpose of this study is to evaluate the differences between immune profiles of individuals remaining on Ocrelizumab/Rituximab versus those transitioning to diroximel fumarate at two years. Baseline visits for subjects transitioning to diroximel fumarate (DRF) will take place 6 months after last Ocrelizumab (OCR) or Rituximab (RTX) dose, prior to…
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Learn More About PRoMPT BOLUS!

Condition: Pediatrics
Investigator: Maria Kwok, MD, MPH
Status: Closed
What's fast-moving, all-encompassing, and plays no favorites? Sepsis, an indiscriminate systemic condition that escalates so rapidly, national guidelines recommend treatment begin within 20 minutes of arrival to the emergency department. Crystalloid resuscitation with normal saline or balanced fluids (including lactated Ringers and Plasma-Lyte) is the…
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Study of Alogabat in Children and Adolescents with Angelman Syndrome (AS)

Condition: Pediatrics
Investigator: Jennifer Bain, MD
Status: Closed
This is a Phase IIA Multicenter, open-label, 12-week study to investigate the pharmacokinetics and safety and to provide proof of mechanism of the drug, Alogabat (RO7017773) in children and adolescents aged 5-17 years old with Angelman Syndrome with the deletion genotype.
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Safety and Efficacy of NMD670 in Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
The main purpose of this study is to learn how the study drug (NMD670) works and how safe the study drug is compared with placebo. A placebo is an inactive material that looks like the study drug but does not have any active study drug. Researchers use a placebo to see if the study drug works better or is safer than taking nothing. You will be in this study…
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Synaptic Therapy Alzheimers Research Trial (START)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
We are seeking men and women, ages 50-85 years, who have mild cognitive impairment due to Alzheimer's disease or mild Alzheimer's disease dementia for an 18-month treatment, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to determine whether the CT1812 study drug beneficially affects cognition and function in…
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Pediatric Type 1 Diabetes and Bone Health Study

Condition: Pediatrics
Investigator: Mishaela Rubin, MD
Status: Closed
We are looking for children between the ages of 8-14 with Type 1 diabetes and without Type 1 diabetes who are interested to participate on a research study to find out how bones are affected in children and adolescents with Type 1 diabetes as compared to people without Type 1 diabetes.
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Assess Treatments for Painful Diabetic Peripheral Neuropathy (PDPN)

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to Assess Treatments for Painful Diabetic Peripheral Neuropathy (PDPN). To be in this study you must have type II Diabetes Mellitus, PDPN, must be 18 years of age or older, and be able to stop taking some of your current pain medications including pills, creams, patches, marijuana, cannabidiol (CBD) oil, etc. Acetaminophen (…
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Study of Focused Ultrasound (FUS) with DEFINITY Microbubbles in Children and Young Adults with Progressive Diffuse Midline Glioma (DMG)

Condition: Pediatrics / Cancer
Investigator: Stergios Zacharoulis, MD
Status: Closed
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
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Study of Focused Ultrasound (FUS) with DEFINITY Microbubbles in Children and Young Adults with Progressive Diffuse Midline Glioma (DMG)

Condition: Neurological Disorders / Brain Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
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Studies of Pediatric Liver Transplantation (SPLIT)

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
The purpose of the study is to collect scientific data on pediatric liver transplantation to learn more about how children do after liver transplant. Collecting these data is expected to help researchers learn more about liver transplant in children. This information may improve medical care of your child and other children who need and who have received…
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Efficacy and Safety of Nipocalimab in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
This is a multicenter, Phase 2/3 study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Adults 18 yrs old or greater at the time of signing the informed consent.
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REST: Sleep Study for High School Students

Condition: Pediatrics
Investigator: Brooke Aggarwal, EdD, MS, FAHA
Status: Closed
High school students ages 14-18 and their parent(s) may be eligible to participate in a 12-week (consisting of a 6-week pre-pilot and 6-week pilot) sleep health education program.
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Genetic Variants of Microglial Activation in AD

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Philip De Jager, PhD
Status: Closed
The purpose of this study is to determine whether different genes are related to the amount of immune cell activity that occurs in the brain in patients with Alzheimer's disease. Participation in this study involves memory testing, a blood draw, MRI, and three PET scans. The second PET scan will involve putting a catheter in your wrist to draw blood…
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Study in Patients With Diabetic Peripheral Neuropathic Pain (DPNP) (PROGRESS)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
To be in this study you must have Diabetic Peripheral Neuropathic (DPN) and be 18 years of age, inclusive, with the presence of bilateral pain in lower extremities due to DPN for at least 6 months prior to Screening and be in a stable regimen for the treatment of T1DM or T2DM for 3 months prior to Screening.
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PEPN2113: Study of Uproleselan in Children with AML, MDS, or MPAL

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
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SAGE HD-201

Condition: Neurological Disorders / Huntington's Disease
Investigator: Karen Marder, MD, MPH
Status: Closed
The DIMENSION Study is evaluating the safety and effect on cognitive symptoms of an investigational oral drug in adults with early Huntington's disease. Cognitive symptoms may include difficulty paying attention or trouble thinking through steps of an activity or complex problems. Symptoms may also include difficulty planning, remembering, and staying…
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Understanding the Causes of Apathy in Alzheimer's Disease (Female subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Catherine Clelland, PhD
Status: Closed
Apathy is a very common symptom experienced by people with Alzheimer's disease, and includes a lack of motivation and loss of interest in many activities. Patients and their caregivers often find apathy to be particularly distressing. This study aims to obtain a greater understanding of the causes of apathy in Alzheimer's disease. We are currently…
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Natural History Study of Children with SCID (Severe Combined Immunodeficiency)

Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
This study is being done to identify the best outcomes for hematopoietic cell transplantation (HCT) or other treatment (enzyme replacement or gene therapy), which is life-saving therapy for children with SCID (severe combined immunodeficiency), leaky SCID, Omenn syndrome and reticular dysgenesis.
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Learn More About How We Decide Which Children With Headaches Need to Have a Picture of Their Brain Taken While They Are in the ED

Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to create a decision-making tool that will help doctors decide which children with headaches require a CT ("CAT scan," "computed tomography") or MRI scan of their head as part of their emergency department care.
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Trial Studying Metformin vs. Placebo in People with Mild Cognitive Impairment (55-90 Years of Age without Diabetes)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Jose Luchsinger, MD
Status: Closed
MAP is a research study looking at whether metformin can help prevent memory decline for people with mild memory concerns. You may be able to join if: you are 55-90 years old, you or your loved ones have noticed some changes in your memory, and you are not already being treated for diabetes or dementia. Potential benefits: You will receive some of your…
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Impact on Learning and Development in Down Syndrome(CHILD-DS)

Condition: Pediatrics / Down Syndrome
Investigator: Michael DiLorenzo, MD
Status: Closed
Down syndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Down syndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known to be associated with developmental problems, few studies have explored how CHD can affect the development and behavior of…
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Understanding the Life Course of Primary Immune Regulatory Disorders (PIRD) Patients and Their Family Members

Condition: Autoimmune Disorders
Investigator: Olatundun Williams, MD
Status: Closed
The purpose of this study is to understand the life course of PIRD patients and their family members that have the same gene change but do not have the same symptoms. PIRD is mostly due to inherited changes in the genes that normally have immune cells fight infections and regulate the immune system. There are many types of problems that patients with PIRD…
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Understanding the Life Course of Primary Immune Regulatory Disorders (PIRD) Patients and Their Family Members

Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
The purpose of this study is to understand the life course of PIRD patients and their family members that have the same gene change but do not have the same symptoms. PIRD is mostly due to inherited changes in the genes that normally have immune cells fight infections and regulate the immune system. There are many types of problems that patients with PIRD…
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Study of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

Condition: Pediatrics
Investigator: Dana Goldner, MD
Status: Closed
This study evaluates whether odevixibat is safe and effective compared to placebo in children with biliary atresia who have recently undergone a Kasai procedure. This is a double-blind study, so the study doctors and the child/parent will not know whether the child is receiving odevixibat or placebo. Participation will involve several research visits over…
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Study of Batoclimab in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
This study is a Phase 2b, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study of Batoclimab Treatment in Adult Participants with Active Chronic Inflammatory Demyelinating Polyneuropathy CIDP): patients who are being treated with batoclimab defined as intravenous immunoglobulin IVIg, subcutaneous immunoglobulin SCIg) or plasma exchange (PLEX…
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Rapid Dementia screening using Magnetic Resonance Imaging

Condition: Neurological Disorders / Dementia
Investigator: Sairam Geethanath, PhD
Status: Closed
A significant focus of healthcare research is aimed at biomarker discoveries in brain and heart diseases in particular, and their impact on the human body in general. MRI is a key component in these healthcare services. It provides a non-invasive and non-ionizing approach to diagnose pathologies along with significant internal and external contrast…
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Study of Quizartinib in Combination with Chemotherapy for Children and Young Adults with Recurrent /Refractory Acute Myeloid Leukemia (AML)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
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ProChocolate Study - Patients with Multiple Sclerosis (MS) and Family Members of Patients with MS

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
We are currently recruiting individuals with Multiple Sclerosis (MS), first-degree family members of persons with MS, and healthy individuals to take part in our research on the effects of probiotics. Our goal is to better understand how the health of the gut microbiome influences the immune system.
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Study of BAN2401 in Subjects with Preclinical Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
We are seeking men & women, ages 55 to 80, who have preclinical Alzheimer's disease for a 216-week treatment, multi-center, double-blind, placebo-controlled, parallel-treatment arm study to determine whether the BAN2401 study drug will reduce the risk of developing Alzheimer's disease. The study participant will receive MRI, and PET scans and…
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Study of CT1812 in Patients with Dementia with Lewy Bodies (DLB)

Condition: Neurological Disorders / Dementia
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This study is of a drug called CT1812, that is being evaluated as a possible treatment for Dementia with Lewy Bodies. In this study, which is carried out at multiple sites in the country, participants receive either CT1812 at one of two doses or placebo. The duration of the study is up to about 35 weeks. Participants receive blood tests, cerebrospinal fluid…
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Longitudinal Early-Onset Alzheimers Disease Study (LEADS) (Alzheimer's Subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The Longitudinal Early-onset Alzheimer's Disease Study (LEADS) is a natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment . Clinical/cognitive, imaging, biomarker, and genetic characteristics will be assessed across two cohorts: early onset Alzheimer's Disease (EOAD)…
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New IDEAS: Brain Imaging Study for Memory Loss

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a research study for persons with memory problems that examines the utility of performing positron emission tomography (PET) scans for the brain amyloid protein found in Alzheimer's disease.
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New IDEAS: Brain Imaging Study for Memory Loss

Condition: Neurological Disorders / Dementia
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a research study for persons with memory problems that examines the utility of performing positron emission tomography (PET) scans for the brain amyloid protein found in Alzheimer's disease.
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Learn About Nasal Spray Medication That Helps Children Stay Calm and Hold Still When They Have a Cut in Their Skin That Needs to be Fixed

Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to find out what is the best dose of intranasal midazolam for children to help them stay calm and hold still while they are having a cut in their skin fixed.
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Dominantly Inherited Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: James Noble, MD, MS
Status: Closed
Funded by a multi-year research grant from the National Institute on Aging, the DIAN Observational Study enrolls family members who have parents with a mutated gene known to cause dominantly inherited Alzheimer's (DIAD). Through the study, researchers follow individuals and family members to see if insights gained could lead to better understanding of…
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FAST TRIAL REGISTRY - Study for Female, Pregnant Patients Affected by Atrial Flutter (AF) or Supraventricular Tachycardia (SVT)

Condition: Pediatrics
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
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Study of Nivolumab in Combination with 5-Azacytidine in Acute Myeloid Leukemia (AML) - Children and Adults Age 1 to 30

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
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Study for older adults with dementia with lewy bodies (DLB) to develop early detection methods

Condition: Neurological Disorders
Investigator: Karen Marder, MD, MPH
Status: Closed
The aim of this study is to develop better biomarkers for the diagnosis and treatment of Lewy Body disorders. In particular, there is a need to understand how the contribution of coexisting Alzheimer's disease may affect the diagnosis and clinical course of Lewy Body disorders. This study will recruit a group of individuals with Lewy Body disorders…
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Adipose Inflammation in Rheumatoid Arthritis - AIR ART (RA Treatment Arm)

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Jon Giles, MD
Status: Closed
We are inviting patients with active Rheumatoid Arthritis (RA) to help us better understand how treatment options affect inflammation in soft tissues. With a combination of blood work, a full-body FDG PET-CT scan, questionnaires, and a soft tissue biopsy (from an area around the belly button), we hope to see how RA interacts with these multiple areas of…
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A Study for Children with Acute Lymphoblastic Leukemia Using Study Drug Carfilzomib

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and teenagers whose leukemia has come back (has relapsed) or has not gone away after other treatments (is refractory). Participants will receive the study drug, called carfilzomib in combination with standard chemotherapy, that are dexamethasone, daunorubicin, vincristine and PEG-…
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Parkinson's Disease Genetic Study: FoxBioNet ECV-004

Condition: Neurological Disorders / Parkinson's Disease
Investigator: Julian Agin-Liebes, MD
Status: Closed
FoxBioNet ECV-004 is an observational study sponsored by the Michael J. Fox Foundation for Parkinson's Research. The purpose of this study is to investigate a gene called LRRK2. Mutations in the LRRK2 gene are associated with Parkinson's disease (PD), and mutation carriers have greater LRRK2 protein activity. The goal of this study is to identify…
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ICON3: A Phase 3 Study of Eltrombopag vs. Standard Therapy for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Condition: Pediatrics
Investigator: Cindy Neunert, MD
Status: Closed
The purpose of this study is to investigate the safety and effectiveness of eltrombopag (investigational drug) in treating children and adolescents with newly-diagnosed immune thrombocytopenia (ITP), which is a rare blood disorder.
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PsA-FAT

Condition: Autoimmune Disorders
Investigator: Jon Giles, MD
Status: Closed
We are looking for patients who have been diagnosed with Psoriatic Arthritis (PsA) to help us better understand the involvement of inflammation on the body. With a combination of bloodwork, a bone density scan, questionnaires, and a one-time soft tissue biopsy (from an area around the belly button), we hope to see how PsA interacts with these multiple areas…
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Study of AL002 for Patients with Early Alzheimers Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a phase 2 study evaluating a new drug, AL002, in persons with early Alzheimer's Disease. Participants will be randomized to receive either AL002 or placebo, intravenously every four weeks, for a total period of about two years. The purpose is to evaluate the efficacy and safety of this drug, and this study involves pen and paper testing, and…
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Fetal Atrial Flutter & Supraventricular Tachycardia (FAST) Therapy Trial (Fetus diagnosed with a fast heart rate)

Condition: Pediatrics / Cardiology
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
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Study of New Drug (VX-548) in Patients with Painful Diabetic Peripheral Neuropathy

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the safety, tolerability, and effectiveness of VX-548 in participants with pain due to Diabetic Peripheral Neuropathy (DPN). To be in this study you must have DPN and be 18 through 80 years of age, inclusive, with presence of bilateral pain in lower extremities due to DPN for at least 1 year.
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Learn More About Positive Pressure Ventilation for Asthma

Condition: Pediatrics / Child and Adolescent Health
Investigator: Maria Kwok, MD, MPH
Status: Closed
The purpose of this study is to evaluate the effectiveness and adverse reactions of the various types of noninvasive positive pressure supportive use in the management of children with moderate to severe acute asthma exacerbation.
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Solutions for Celiac Study

Condition: Autoimmune Disorders
Investigator: Peter Green, MD
Status: Closed
This research study is sponsored by ImmunogenX and The National Institutes of Health (NIH, Bethesda, MD). The objective of this study is to determine if the investigational product (latiglutenase) will help decrease symptoms in subjects with celiac disease while maintaining a gluten-free diet while undergoing periodic gluten exposure. In addition, this…
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Study of Olanzapine in Children Receiving Chemotherapy for Hematopoietic Stem Cell Transplant (HSCT)

Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
This research study is to find out whether adding olanzapine to standard antiemetics will be helpful in controlling chemotherapy-induced vomiting (CINV) in children receiving high-dose cyclophosphamide for Blood/Bone Marrow Transplant (BMT) conditioning.
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Study of MTX110 in Combination with Gadolinium in Children with Newly Diagnosed Brain Cancer (Diffuse Midline Gliomas)

Condition: Pediatrics / Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
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Study of MTX110 in Combination with Gadolinium in Children with Newly Diagnosed Brain Cancer (Diffuse Midline Gliomas)

Condition: Neurological Disorders / Brain Tumors
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
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Bone Structure in Men with Celiac Disease

Condition: Autoimmune Disorders
Investigator: Marcella Walker, MD
Status: Closed
Celiac disease is associated with an increased risk of osteoporosis and fractures. Men with celiac disease were found to be at higher risk for metabolic bone disease compared to women with celiac disease. The mechanisms underlying metabolic bone disease in celiac disease remain incompletely understood. The goal of this study is to assess skeletal…
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Prebiotic vs. Probiotic Treatment in Multiple Sclerosis (MS) or Clinically Isolated Syndrome (CIS)

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Rebecca Straus Farber, MD
Status: Closed
This study will be used to evaluate the immunologic effects of Prebiotics (soluble fiber required by beneficial bacteria), in comparison to Probiotics (beneficial bacteria), in individuals with Multiple Sclerosis (MS) and Clinically Isolated Syndrome (CIS).
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Study of Cabergoline in Adolescents and Adult Women with Endometriosis

Condition: Pediatrics
Investigator: Jin Hee Kim, MD
Status: Closed
In this research study we want to learn more about a potential new therapy for pain associated with endometriosis. We want to determine if a non-hormonal medication, cabergoline, will help patients with endometriosis. Cabergoline is a medication that is approved by the Food and Drug Administration (FDA) for treatment of other medical conditions. In this…
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Teens' Thoughts About Depression and Mental Health Related Discrimination

Condition: Pediatrics
Investigator: Doron Amsalem, MD
Status: Closed
We are conducting a research study to explore discrimination around depression and barriers to care in youth ages 14-18. We will use what we learn to create videos to reduce such discrimination. Parental permission is required for this study. Adolescents (ages 14-18) will participate in an individual interview that will be approximately 45 minutes long. We…
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Non-Invasive Blood-Brain Barrier Opening in Alzheimer's Disease Patients Using a Focused Ultrasound Device

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Elisa Konofagou, PhD
Status: Closed
A clinical trial that uses focused ultrasound to non-invasively open the blood-brain barrier in Alzheimer's disease patients, in order to help get medication into their brain. In this study, we will focus on the safety and efficacy of this technique, without the use of an anti-Alzheimer's drug. We will also test whether treatment with focused…
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Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients (6 months-17 years)

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find out the effects and safety of an investigational (experimental) new drug called copanlisib in pediatric patients from 6 months to 21 years of age. This study consists in two-phase, Phase I part will determine which is the right dose of copanlisib to be used in children/adolescents/adults with relapsed (has returned) or…
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Study of Intranasal Oxytocin for Frontotemporal Dementia (FOXY)

Condition: Neurological Disorders / Dementia
Investigator: Edward Huey, MD
Status: Closed
Columbia Medical Center is recruiting for a study to test whether the drug oxytocin can improve symptoms of emotion and behavior (e.g. apathy) in people with frontotemporal dementia. The study will last for up to 24 weeks (6 months). This is a crossover study, which means that each participant will receive both oxytocin and placebo (medically inactive…
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Dementia and Psychiatric Symptoms (Ages 40-90) (Alzheimer's Disease)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Edward Huey, MD
Status: Closed
The purpose of this study is to carefully examine how dementia can affect mood and behavior. In this study, we will use Magnetic Resonance Imaging (MRI) to better understand the relationship between thinking, mood, behavior and the brain. We are currently seeking participants who have a diagnosis of dementia, such as Alzheimer's Disease, who are…
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Study of Drug Injection with Chemotherapy in Children, Adolescents, and Young Adults with Relapse of Actue Lymphoblastic Leukemia

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Vincristine Sulfate Liposome Injection (Marqibo) in combination with UK ALL R3 induction chemotherapy. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) who have relapsed…
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Study of Flotetuzumab for Children, Adolescents, and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the highest dose of Flotetuzumab that can be given safely to patients who have a certain type of leukemia called Acute Myeloid Leukemia (AML) that has relapsed (has come back after treatment) or is refractory (never responded to treatment). Flotetuzumab is an experimental treatment that works by targeting certain…
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Effects of Abatacept on Heart Inflammation in Rheumatoid Arthritis (AMiRA)

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Laura Geraldino-Pardilla, MD, MPH
Status: Closed
This study aims to understand how two commonly prescribed drugs impact myocardial inflammation in rheumatoid arthritis.
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Study of Posiphen in Subjects with Early Alzheimer's Disease (AD) DISCOVER

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This study is an early phase study of a medication for Alzheimer's Disease, with examination of cerebrospinal fluid.
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Study of Selinexor in Pediatric Solid Tumors, including CNS Tumors

Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
We are testing new experimental drugs such as selinexor in the hopes of finding a treatment that may be effective against tumors that have come back or that have not responded to standard therapy. The goals of this study are: To find the highest safe dose of selinexor that can be given without causing severe side effects; To learn what kind of side effects…
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Study of Maralixibat in Infants with Biliary Atresia Who Have Had Kasai Surgery

Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The purpose of this study is to see how effective and safe the drug maralixibat is when compared to placebo in infants with biliary atresia who have had Kasai surgery within 3 weeks before participation. This is a 26-week, multicenter, double-blind, placebo-controlled, randomized parallel-group study, followed by an open-label extension (OLE). Approximately…
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ASPIRE: Aspirin for Exercise in Multiple Sclerosis

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Victoria Leavitt, PhD
Status: Closed
ASPIRE is a clinical trial to investigate aspirin as a treatment to improve exercise performance in people with multiple sclerosis (MS) who experience overheating during exercise. Persons with multiple sclerosis benefit from exercise, but many avoid it because of exhaustion and overheating. This randomized controlled trial (RCT) tests aspirin as a method to…
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Anti-Viral therapy in Alzheimer's disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
In the U.S., 82% of older adults test positive for antibodies to the herpes viruses. Basic and clinical evidence point to the herpes viruses as a possible cause or contributor to Alzheimer's disease. The long-standing theory that viruses may cause Alzheimer's disease has never been tested in a clinical trial. Anti-viral therapy in Alzheimer's…
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Social and Contextual Impact on Children Undergoing Liver Transplant

Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The social determinants of health have a large impact on health. We have previously demonstrated that an index of neighborhood socioeconomic deprivation was associated with an increased risk of medication non-adherence in children after liver transplant and an increased risk of graft failure and death in children after transplant. The present proposal seeks…
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A Study to Test a New Drug (PXT3003-06) in Patients with Charcot-Marie-Tooth Type 1A (CMT1A)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
The main purpose of this study is to investigate how effective PXT3003 is when compared to a placebo, on the disease progression and symptoms of CMT1A. The study also aims to assess the safety and tolerability (whether side effects of a medication can be handled by patients) of PXT3003. We are enrolling male and non-pregnant female subjects, aged 16 to 65…
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Study of Metronomic and Targeted Anti-Angiogenesis Therapy For Children with Medullobastoma (Brain Tumor)

Condition: Pediatrics / Cancer
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and young adults patient with relapsed (has returned) and progressive medulloblastoma (brain tumor). This study is being done to find out if bevacizumab can be safely given in combination with five study drugs given by mouth, thalidomide, fenofibrate, celecoxib and alternating every…
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RESTORE: A Study of Patients with symptomatic neurogenic orthostatic hypotension (NOH)

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
You are being asked to participate in this study because you have a condition known as symptomatic neurogenic orthostatic hypotension (NOH) associated with Primary Autonomic Failure Parkinsons Disease (PD), Multiple System Atrophy (MSA) or Pure Autonomic Failure (PAF), or Non-Diabetic Autonomic Neuropathy (NDAN) or Dopamine Beta Hydroxylase (DBH) deficiency…
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Improvement of exercise capacity in patients with Fontan physiology

Condition: Pediatrics / Cardiology
Investigator: Marc Richmond, MD
Status: Closed
Over time, children and young adults who have had a Fontan operation may find they are more tired, have difficulty breathing and cannot exercise as well as before. Currently, there are no approved medicines to prevent this decline. The purpose of this research study is to learn whether children who have had the Fontan operation can improve their ability to…
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A study for patients with lupus using the study drug XmAb5871.

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this clinical trial is to learn about the ability of an experimental treatment XmAb5871 to maintain lupus disease activity improvement achieved by a brief course of disease-suppressing intramuscular (IM) steroid therapy in lupus patients.
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Early Developmental Changes, Risk for Diabetes, and the Microbiome in Children

Condition: Pediatrics
Investigator: Sharon Oberfield, MD
Status: Closed
We are recruiting boys between 3-9 years old and girls between 3-8 years old to participate in a research study. Children who would like to participate must be healthy and not showing signs of puberty. The goal of the study is to understand the relationship between early body development, metabolism (sugars and fats processing), and the saliva microbiome (…
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A study for children with an Inammatory Bowel Disease (IBD) such as Crohn's disease or ulcerative colitis

Condition: Pediatrics
Investigator: Joel Lavine, MD
Status: Closed
Researchers at Columbia University are conducting a clinical research study of children with inammatory bowel disease (IBD). This study will examine the eectiveness and safety of an investigational medication inchildren with IBD.
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Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment with Subcutaneous Immunoglobulin

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to see how effective two doses of an investigational new drug called IgPro20 is when given subcutaneously (a shot given into the fat layer between the skin and the muscle) to treat CIDP in patients that still require IVIG and also to look at how safe the IgPro20 is for patients.
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Study of Drug in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors

Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This is a Phase 1 multicenter study of LY2606368, a CHK1/2 inhibitor, in pediatric patients with refractory solid tumors, including CNS tumors. The study treatment is considered experimental because LY2606368 is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric patients with refractory solid tumors, including…
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Study of Study Drug (ACE-083) in Patients with Carcot-Marie-Tooth Disease Types 1 and X

Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Closed
Patients with Charcot-Marie-Tooth (CMT) disease have a degenerative nerve disease that can cause muscle weakness and decreased muscle size in different muscles in their body. This study drug is designed to evaluate ACE-083 for its ability to increase the size of your muscles, which could improve your muscle strength. The purpose of this research study is…
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A study for male adolecents with muscular dystrophy using study drug Eteplirsen

Condition: Pediatrics / Neuromuscular
Investigator: Jacinda Sampson, MD
Status: Closed
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
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A Study for Patients With Generalized Myasthenia Gravis Using Intravenous Human Immunoglobulin Concentrate, IGIV-C.

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves your muscle weakness associated with MG symptoms. A placebo is a medication that looks like the study medication but has no active ingredient. Immunoglobulin is an antibody produced by white blood cells that is used by the immune…
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Effects of varying bubble nasal continuous airway pressure in very low birth weight infants

Condition: Pediatrics
Investigator: Rakesh Sahni, MD
Status: Closed
The purpose of this study is to see if the use of bubble nasal continuous positive airway pressure (NCPAP) at a pressure of 6-7 centimeters of water (cm H2O) in very low birth weight (VLBW) infants is associated with improved gas exchange without any related changes in vital signs.
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A study of safety and efficacy of IV BMS-986168 in patients with Progressive Supranuclear Palsy.

Condition: Neurological Disorders
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The Primary objective of the study is to evaluate the efficacy of the study drug, compared to placebo, as measured by a change from baseline in the PSP Rating Scale (PSPRS) after one year, and to assess the safety and tolerability of the study drug, relative to placebo.
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A study for patients with Idiopathic Parkinson's Disease using study drug CVT-301

Condition: Neurological Disorders
Investigator: Cheryl Waters, MD
Status: Closed
The purpose of this research is to find out whether an investigational drug called CVT-301, is helpful to people with Parkinson's Disease (PD). The treatment involves a new method for taking levodopa (LD) through an inhaler (which is like an asthma inhaler) to provide relief of motor fluctuations (OFF episodes), which are experienced by many Parkinson…
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A Study Evaluating Abatacept (Orencia) for Treatment of Systemic Sclerosis

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Closed
Systemic sclerosis (SSc) is a systemic connective tissue disease characterized by fibrosis and atrophy, namely of the skin, internal organs, joints, and skeletal muscles. This is a double-blind, placebo-controlled trial to test the efficacy of abatacept (Orencia) on the treatment of diffuse cutaneous systemic sclerosis in patients with a disease duration of…
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A study for children and adolescents with relapsed or refractory acute myeloid leukemia using study drug Lenalidomide

Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This study is for children with relapsed or refractory acute myeloid leukemia (rrAML). Acute myeloid leukemia is a fast growing cancer of the blood and bone marrow. This study examines the effects of study drug Lenalidomide on children with rrAML. Lenalidomide is an anti-cancer drug that belongs to a group of drugs known as immunomodulating drugs (IMiDs), a…
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A Study to Assess the Treatment-Related Fluctuations in CIDP Patients Using Daily Grip Strength Measurements

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Subjects with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) will be enrolled in the study and Intravenous Immunoglobulin (IVIg) will be given to eligible subjects on the study. CIDP is a chronic inflammatory condition that causes the immune system to attack the nerves. IVIg is one common treatment for CIDP. IVIG is a blood product that is…
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Small Fiber Neuropathy in Juvenile Fibromyalgia

Condition: Pediatrics
Investigator: Alexis Boneparth, MD
Status: Closed
Juvenile fibromyalgia (JFM) is a cause of chronic, whole-body pain. The cause of JFM is unknown. One of the possible causes of JFM is a problem with nerves called "small fiber neuropathy." This is a problem where small nerve fibers in the skin are not working properly and patients can have pain as a result. Recent studies have found evidence of…
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A study for patients with Amyotrophic Lateral Sclerosis (ALS) using study drug tirasemtiv

Condition: Neurological Disorders
Investigator: Hiroshi Mitsumoto, MD
Status: Closed
The purpose of this study is to investigate the safety and effectiveness of tirasemtiv as a potential new therapy for the improvement of breathing, muscle weakness and muscle fatigue in patients with Amyotrophic Lateral Sclerosis (ALS). The effects of tirasemtiv on the performance of your elbow, wrist, knee, ankle, and hand muscles and how long you can…
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NSAIDs in Ankylosing Spondylitis

Condition: Autoimmune Disorders
Investigator: Runsheng Wang, MD
Status: Closed
This is a 6-week randomized, double-blind trial of 4 different non-steroidal anti-inflammatory drugs in patients with axial spondyloarthritis to compare the change of pain score from baseline at 4 weeks to the change of pain score from baseline at 6 weeks. Patients with ankylosing spondylitis or axial spondyloarthritis who fulfills the inclusion and…
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A study for patients with Lupus using study drug ustekinumab

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
If you have active Systemic Lupus Erythematosus, also known as SLE or Lupus, you may be interested to learn about a clinical research study that is being conducted to study the safety and effectiveness of an investigational medication called ustekinumab for active Lupus. Eligible study participants will be provided with study medication and study-related…
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Brave Trial, A Clinical Research Study for Duchenne Muscular Dystrophy (DMD)

Condition: Pediatrics / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
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Study for patients with Amyloidsis (TTR) using an investigational drug, ALN-TTR02 for treatment

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to collect information about how patients with FAP respond to the study drug ALN-TTR02 over 18 months. This includes studying any potential improvements in FAP and any side effects that participants may have from the study drug. In addition, the study will examine how the body handles (distributes, break down, eliminates) the…
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Bowel Prep Study

Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This research study is for children age 12 to 16. In this research study, we will evaluate the safety, effectiveness and tolerability of two different doses of a bowel cleansing medication referred to as BLI-800. One group of study participants will receive the larger dose (6 oz.), which dose has been approved by the FDA for use by adults. A second group of…
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Body Composition Changes after Bariatric Surgery in Adolescents

Condition: Pediatrics
Investigator: Wei Shen, MD
Status: Closed
This is a study of body changes that take place in teenagers who undergo bariatric surgery as a treatment for obesity and obesity-related conditions. Changes will be measured by serial MRI andmetabolomic profiling.
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A study for children with pediatric acute Lymphoblastic Leukemia using study drug Moxetumomab Pasudotox

Condition: Pediatrics / Cancer
Investigator: Maria Sulis, MD
Status: Closed
The purpose of this study is to determine the effectiveness and safety of the study drug moxetumomab pasudotox in treating children with relapsed or refractory pALL or Lymphoblastic Lymphoma of B-cell origin. Moxetumomab pasudotox is made up of two parts: 1) a modified mouse antibody that attaches to a protein from the immune system, called CD22, which can…
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Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves the ability to taper participant's dose of corticosteroids without causing significant increase in your muscle weakness and worsening MG symptoms. A placebo is a medication that looks like the study medication but has no…
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Study of Patients Receiving an Allogeneic Stem Cell Transplantation for Non-Malignant Disease

Condition: Pediatrics
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
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Cognitive Training

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
The purpose of this study is to evaluate if systematic cognitive training can improve cognitive performance in participants (ages 55-95 years) with memory loss. This study will evaluate the effects of Computerized Cognitive Training (CCT) for improvement in everyday cognitive and function status, in addition to long-term changes in brain networks over an 18…
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Study of E2027 in Subjects with Lewy Body Dementia (LBD) (DELPHIA)

Condition: Neurological Disorders / Dementia
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a 22-week treatment, multicenter, double-blind, placebo-controlled, parallel group, Phase 2 study to evaluate the efficacy, safety, and tolerability of the study drug E2027, in participants with Dementia with Lewy Bodies (DLB) Lewy Body Dementia - LBD. The main purpose of this research study is to find if a new drug called E2027 works in people who…
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A study for patients with multiple system atrophy (MSA) using study drug AZD3241 for treatment

Condition: Neurological Disorders
Investigator: William Kreisl, MD
Status: Closed
This study is for patients who have been diagnosed with Multiple System Atrophy (MSA). The main goals of this study are to determine how safe and well tolerated AZD3241 is in patients who have MSA, and whether AZD3241 has certain effects in the brain of patients with MSA. These effects in the brain are measured with positron emission tomography (PET)…
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A study for patients with lupus nephritis, using an investigational treatment design with rituximab, cyclophosphamide, and solumedrol

Condition: Autoimmune Disorders
Investigator: Anca Askanase, MD, MPH
Status: Closed
In this experimental study, researchers will try to find out if treatment of lupus nephritis with a combination of rituximab and cyclophosphamide (CTX), or a combination of rituximab and CTX followed by treatment with belimumab is safe and if this drug combination can block the immune system attacks.
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Double-blind trial to Test Efficacy of Rituxan (Rituximab) on Systemic Sclerosis-Associated Pulmonary Arterial Hypertension

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Closed
Systemic sclerosis (SSc) is a systemic connective tissue disease characterized by fibrosis and atrophy, namely of the skin, internal organs, joints, and skeletal muscles. There are numerous manifestations of SSc that affect multiple organ systems. The major pulmonary manifestations of SSc, interstitial lung disease and systemic sclerosis-associated…
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Study of Posaconazole (POS) in Immunocompromised Pediatric Subjects with Neutropenia (Low Neutrophils Type of White Blood Cell Count)

Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to: 1) Test the safety and tolerability of the research study drug, MK-5592 posaconazole (POS), intravenously (IV) and in powder for oral suspension (PFS), 2) Evaluate the pharmacokinetics (how the study drug is absorbed and broken down in the body), of POS IV solution and POS PFS, and 3) Evaluate the taste and reaction to the…
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A study for pediatric patients with mild to moderate Ulcerative Colitis using study drug MMX Mesalamine/Mesalazine

Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This study is evaluating an investigational medication to see if it is safe and if it works well in children andadolescents who have mild to moderate ulcerative colitis (UC) or are in remission. Ulcerative colitis (UC) is a chronic disease of the last section of the intestine, which is also known as the colon. If you have UC, the lining of your colon…
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A Study to Assess the Clinical and Biological Predictors of Disease Course in Patients with Guillain-Barre Syndrome

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
International GBS Outcome Study (IGOS) is a study of the Inflammatory Neuropathy Consortium (INC) and Peripheral Nerve Society (PNS) on disease course and outcome in Guillain-Barr syndrome (GBS). Guillian Barre syndrome is an autoimmune disorder affecting the peripheral nervous system, leading to paralysis and potential fatality if it progresses to involve…
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Testing combination drugs in children with solid tumors that have come back or are resistant to current anti-cancer therapy

Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of pevonedistat in combination with Temozolomide and Irinotecan in pediatric patients with refractory solid tumors, including CNS tumors and lymphoma. The study treatment is considered experimental because pevonedistat is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric…
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Top-CSPN Study

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
This study is looking at using topiramate as a potential therapy for patients with cryptogenic sensory peripheral neuropathy. Patients who are randomized into the study will return to the clinic for follow up every 16 weeks. The total length of the study is 96 weeks.
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A study for patients with early Alzheimer's Disease using study drug AZD3293

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to see if the study drug AZD3293 is effective and safe in treating early Alzheimer's disease compared with a placebo. A placebo is a tablet containing no drug. This study will also measure the levels of AZD39293 and its metabolite in the blood and evaluate the relationship between drug levels, efficacy (how well it works)…
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Treatments Against RA and Effect on FDG PET-CT: The TARGET Trial

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Joan Bathon, MD
Status: Closed
The study aims to compare the effect two different RA treatments on cardiovascular health in patients who are considering a treatment change. The study is recruiting patients who are only taking methotrexate and are switching treatment due to a lack of improvement. The trial will randomize participants to take a TNF inhibitor (Enbrel or Humira) in addition…
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A study for patients with systemic sclerosis using study drug abituzumab

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Closed
The purpose of this study is to determine the effectiveness and safety of a study drug called abituzumab in subjects with systemic sclerosis-associated interstitial lung disease (SSc-ILD). The study will also look at the following: How the study drug affects other symptoms of SSc-ILD -How safe the study drug is (i.e. if there are any side effects) -How the…
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A clinical trial for children with 15Q duplication syndrome or CDKL5 deficiency disorder to help control seizures.

Condition: Pediatrics
Investigator: Wendy Chung, MD, PhD
Status: Closed
The purpose of this research is to determine whether a new investigational drug called TAK-935 is safe and whether it may help treat epilepsy in children with 15q duplication syndrome or CDKL5 deficiency disorder.
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A study for children and adolescents with advanced non central nervous system (CNS) tumors using study drug Talimogene laherparepvec

Condition: Cancer / Pediatric Solid Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to find out more about how the study drug called talimogene laherparepvec will be used in children/adolescents/young adults with advanced solid tumors not involving the central nervous system that are available for direct injection. This is a phase 1 study, multicenter, open-label study, that means all subject enrolled in the…
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Study for treating patients with neuropathy using study drug fingolimod

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of study drug, fingolimod taken daily compared with a placebo (does not contain active medication) on delaying disability progression in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study will consist of 3 periods: a Screening Period (lasting for up to 45…
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Study of Glecaprevir/Pibrentasvir in Pediatric Subjects with Chronic Hepatitis C Virus (HCV) Infection

Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
Open-labeled hepatitis C pediatric treatment trial with a two drug direct acting viral oral combination (already approved in adults) across all genotypes for naive or treatment experienced subjects.
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A study of children with relapsed or refractory solid tumors and large cell lymphoma using study drug, PF-02341066 for treatment

Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
The purpose of this study is to examine the effectiveness and tolerability of a study drug called PF-02341066. Additional goals of this study are to learn how the body handles the study drug as well as determine whether or not PF-02341066 is a beneficial treatment for your tumor This study is for children with a recurrent or progressive tumor or lymphoma…
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A study for patients with Parkinson's Disease using study drug Levodopa (APL-130277)

Condition: Neurological Disorders
Investigator: Cheryl Waters, MD
Status: Closed
The purpose of this study is to test the effectiveness of investigational drug APL-130277 for patients with Parkinson's disease. APL-130277 is an investigational drug, which means that the study drug is currently being tested. It is not approved by the U.S. Food and Drug Administration (FDA). The active ingredient in APL-130277 is apomorphine, a…
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CD34 Stem Cell Selection for Malignant Condition

Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a malignant condition.
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A study assessing the safety and effectiveness of study drug Crenezumab in patients with mild to moderate Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to better understand why certain patients are more likely to respond totreatment than others, to facilitate the development of personalized medicinesto get the rightmedicine to the right patient. To achieve this goal, samples of blood will be collected from patients with Alzheimer's for future research. The sample will be…
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A study for patients with Lupus Nephritis using ACTHAR GEL

Condition: Autoimmune Disorders / Lupus
Investigator: Wooin Ahn, MD
Status: Closed
The purpose of this study is to determine if Acthar gel is a safe and effective therapy for patients with membranous (Class V) lupus nephritis. Class V lupus nephritis is associated with the development of chronic kidney disease and end-stage renal (kidney) disease, and there is no clear therapy for treatment. Currently used therapies arent completely…
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Genomic Translation for ALS Care

Condition: Neurological Disorders / Neuromuscular
Investigator: Matthew Harms, MD
Status: Closed
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern. For up to three years, subjects will be followed every 3 months at the Columbia ALS…
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A study in children, adolescents, and young adults recurrent or refractory solid tumors using study drug nivolumab

Condition: Cancer / Pediatric Solid Tumors
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to learn what kind of side effects nivolumab as a single agent and nivolumab in combination with ipilimumab can cause and if these treatments are beneficial for solid tumors. Nivolumab (Dose Level 1) will be given as an intravenous infusion every 14 days of a 28 day cycle. If Dose Level 1 is not tolerable, then it will be…
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Treatment of Agitation in Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
This NIH funded research study involves a 12-week double blind treatment trial in which Alzheimer's disease (ages 55-95) who have agitation/aggression with or without psychosis (hallucinations or delusions) will receive either low-dose lithium or placebo. Patients are allowed to receive other specific psychiatric medications if needed during the study…
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Study of New Drug in Patients with Cognitive Impairment or Mild Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
You have been selected as a possible participant in this study because you have memory or thinking problems (cognitive impairment) or mild Alzheimer's disease. Alzheimer's disease is a slow progressive disease of the brain that affects memory and brain function. Currently available medications for Alzheimer's disease provide limited…
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A study for patients with Parkinson's Disease with recurring OFF episodes using a Levodopa inhalation powder

Condition: Neurological Disorders
Investigator: Cheryl Waters, MD
Status: Closed
This study is for patients with Parkinson's disease. For most patients with Parkinsons disease, doctors use levodopa as a treatment to manage their symptoms. Levodopa pills have been used for many years to treat the symptoms of Parkinsons disease patients. However, with long-term treatment, the effectiveness of levodopa pills often lessens and can lead…
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Rheumatoid Arthritis Study - RHYTHM Renewal

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Joan Bathon, MD
Status: Closed
We are doing a research study to learn more about why people with rheumatoid arthritis (RA) are prone to heart disease. We are recruiting patients diagnosed with Rheumatoid Arthritis without a personal history of heart disease. This study involves one initial full visit as described below, and a brief follow up visit that will take place approximately 2 or…
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A study for patients with lupus nephritis using the study drug Obinutuzumab.

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this study is to test the safety and effectiveness of the study drug Obinutuzumab and to find out what effects, good or bad, it may have as an additional therapy in treating your lupus nephritis symptoms.
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Study of BHV-4157 in Alzheimer's Disease (T2 Protect AD)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
T2 Protect AD is a clinical trial testing the investigational drug troriluzole in people with mild to moderate Alzheimer's disease (AD). The study is designed to determine whether this new drug can protect against, slow down, or potentially improve memory and thinking problems that increase as Alzheimer's disease progresses.
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Study of patients with neuropathy using study drug, ISIS TTR Rx

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of ISIS TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy (FAP). The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate FAP. To be in this study you must have FAP and be able to walk without help or walk with using only one cane.
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A study for patients with dementia using study drug RVT-101

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to investigate how well a drug called RVT-101 works to improve cognitiveand overall function as well as to investigate the safety of RVT-101. RVT-101 is an investigational drug.Investigational means that the drug tested in this study has not been approved by the U.S. Food and Drug Administration (FDA) or any other Health…
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Study of Entinostat in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors and Lymphoma

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This study is being carried out by the Childrens Oncology Group (COG) Phase 1 Consortium. COG is an international research group that consists of more than 200 hospitals that treat children with cancer in the United States, Canada, Australia, and Switzerland. The Phase 1 Consortium is the group within COG that consists of 21 hospitals, and participation in…
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Efficacy and Safety Study of Drug (MTAU9937A) in Patients with Alzheimer's Disease Preclinical-to-Mild or Moderate Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a Phase II, randomized, double-blind, placebo-controlled, parallel-group study that will evaluate the efficacy, safety and tolerability, pharmacokinetics, and pharmacodynamics of the study drug (an antibody to the tau protein called MTAU9937A) in patients with prodromal or mild Alzheimer's disease. The study consists of a screening period, a…
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A study for patients with spinal muscular atrophy using study drug, ISIS 396443 to test safety and effectiveness

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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A study for patients with Alzheimer's Disease using study drug azeliragon (TTP488)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This study is for patients with Alzheimer's Disease. Alzheimers disease is a disorder characterized by progressive loss of memory, thinking, and behavior. An earlier study in patients having mild to moderate Alzheimers disease has demonstrated more pronounced benefit of treatment with azeliragon among participants with mild disease than among…
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AURORA Study: Iloprost for Subjects with Systemic Sclerosis (SSc-Raynaud's Phenomenon)

Condition: Autoimmune Disorders
Investigator: Elana Bernstein, MD
Status: Closed
The purpose of this study is to find out whether a drug called iloprost is safe to use in people with systemic sclerosis (SSc) (Raynaud's phenomenon), and whether it may help decrease symptomatic digital ischemic episodes (episodes in the fingers).
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Study of Hematopoietic Stem Cell Transplantation (HSCT) in non-cancer disease

Condition: Pediatrics
Investigator: Monica Bhatia, MD
Status: Closed
The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation with mild and safer chemotherapy as pretransplant conditioning. This treatment is chosen because it is milder and is likely to have fewer long and short term side effects unlike older chemotherapy and radiation treatments. Yet, it…
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Tacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score (TEAMMATE) (Pediatric)

Condition: Pediatrics / Cardiology
Investigator: Irene Lytrivi, MD
Status: Closed
This is a Phase 3 multi-center open label randomized clinical trial. The purpose of this study is to learnmore about which anti-rejection (or immunosuppressive) medications best protect infants, children,adolescents, and young adults after they have had a heart transplant. Two different drug regimens will bewill be compared. All of these drugs are FDA-…
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