We are conducting a study on depression and suicide risk, and participants can earn up to $300 for taking part. Suicide rates in the U.S. are rising, and new treatments are urgently needed. This study focuses on the brain chemical glutamate, which may play an important role in mood and mental health. We will compare glutamate levels at rest and during…
The study uses brain imaging and cortisol to understand risk and protective factors for mental health among adolescents on social media. We are currently recruiting adolescents ages 14-18 who struggle with mental health. To participate in our study, we would ask your child to: (a) complete a mental health interview (over Zoom), (b) complete a Magnetic…
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…
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…
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…
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.
Anorexia Nervosa is a serious illness and relapse rates are high. We have shown that behavioral routines get stuck/entrenched. Our newest research, Relapse Prevention and Changing Habits (REACH+) for Anorexia Nervosa, aims to help patients minimize unhelpful routines and develop more healthful recovery-oriented behavior. Participants will complete initial…
Our lab is conducting brain imaging research studies investigating people's behavior. We are looking for people between the ages of 18 and 65 to be study participants. As a schizophrenia study participant, you will be asked to complete a set of self-report questionnaires probing personality traits. You will also complete a number of computer-based…
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.
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.
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…
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…
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…
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…
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.
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…
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…
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…
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…
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…
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.
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…
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…
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…
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…
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-…
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,…
In this study, we are trying to learn more about the symptoms of psychosis, specifically to learn how alterations in the brain may produce certain experiences (including voice hearing or auditory hallucinations) that are common in people with psychosis or schizophrenia. The study involves clinical interviews, computer tests, and magnetic resonance imaging (…
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…
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…
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…
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…
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.
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…
Participate in learning more about Impulsivity and Compulsivity in Spinocerebellar ataxia (SCA). By investigating individuals with SCA, we will gain valuable insights into the specific mechanisms through which the cerebellum participates in cognitive processes. Eligible participants: Adults over the age of 18, diagnosed with Spinocerebellar ataxia (SCA) and…
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…
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).
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…
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…
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.
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?"
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…
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…
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)…
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…
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…
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…
Depression and some antidepressants may be associated with an increased risk of breaking a bone. It is not clear why this could be the case. It is possible that depression and/or certain medications used for the treatment of depression cause bone loss, increase the risk of falls, are associated with lower physical activity, or effects on muscle function.…
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…
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…
The Center for OCD Research at Columbia University Irving Medical Center is seeking individuals with obsessive-compulsive disorder (OCD) to participate in a research study, at no cost to you. This study is investigating the medicinal effects of nabilone for OCD symptoms. Nabilone is a synthetic form of tetrahydrocannabinol (THC), which is one of the active…
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…
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.…
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…
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…
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…
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…
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…
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…
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…
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.
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…
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…
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…
Our lab is conducting research studies investigating people's behavior. We are looking for people between the ages of 18 and 65 who have been diagnosed with Schizophrenia/Depression to be study participants. As a study participant, you will be asked to complete a set of self-report questionnaires probing personality traits and computer-based tasks.…
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-…
This research study seeks to better understand the biological and behavioral aspects of specific phobia and evaluate how the "Self-Distancing" technique might help youth confront their fears. The study will be conducted at Columbia University in Manhattan. Participation involves completing questionnaires and attending a single visit for a…
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…
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…
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…
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…
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.
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…
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…
Are you suffering from chronic depression? We are seeking participants for a depression treatment research study at Columbia University using Transcranial Magnetic Stimulation (TMS). This study develops a more accurate TMS to treat chronic depression. Participation includes clinical assessments and magnetic resonance imaging (MRI). You will receive 27…
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.
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 (…
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…
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…
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…
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.
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.
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…
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.
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…
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…
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…
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.
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.
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…
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…
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…
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…
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…
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…
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…
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.
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…
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…
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)…
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.
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.
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.
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…
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…
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…
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…
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-…
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…
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.
The goal of the project is to identify differences in learning in obsessive-compulsive disorder (OCD) by measuring the activity of the brain while participants complete computer tasks. Eligible participants will be asked questions about their medical and psychiatric history and perform tasks on a computer while an electroencephalogram (EEG) is recorded.…
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…
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…
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.
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.
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.
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…
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…
This study uses smartphone technology to better understand changes in adolescent moods over time. As a participant in our study, we would: (a) interview your child about current and past thoughts, feelings, and behaviors and (b) collect data from your child's smartphone for 6 months through an app called the Effortless Assessment of Risk States (EARS;…
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).
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…
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…
We are conducting research to explore self-stigma around depression, racial/ethnic and cultural experiences, and barriers to care (e.g., embarrassment, limited access, low expectations/motivation). Self-stigma refers to negative beliefs a person has about themself due to their mental illness. We are looking for Latinx or Black young adults ages 18-25 who…
We are conducting research to explore the level of self-stigma around depression, racial/ethnic and cultural experiences, and barriers to care (e.g., embarrassment, limited access, low expectations/motivation). Self-stigma refers to negative beliefs a person has about themself due to their mental illness. We are looking for Latinx or Black young adults ages…
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…
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…
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…
People with bulimia nervosa engage in risky behaviors more often than people without this disorder. We are researching the neurobiological underpinnings of risky decision-making in bulimia nervosa. As a participant in this study, you would: (1) complete a series of questionnaires and interviews, (2) complete an MRI scan (no radiation), and (3) complete…
People with bulimia nervosa engage in risky behaviors more often than people without this disorder. We are researching the neurobiological underpinnings of risky decision-making in bulimia nervosa. As a participant in this study, your teen would: (1) complete a series of questionnaires and interviews, (2) complete an MRI scan (no radiation), and (3)…
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…
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…
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…
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…
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…
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…
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…
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…
This research study uses non-invasive brain imaging called functional near-infrared spectroscopy (fNIRS). For fNIRS, a small infrared bulb shines light through the skull, and a special light detector is able to measure oxygen use in the brain. Our goal is to understand oxygen use by brain mitochondria cells in major depression and how this is affected by…
This is a research study to understand what causes bipolar disorder and how medications treat bipolar depression. Particularly, we are looking at the importance of inflammation in the process. If you participate, you will receive an experimental medication for your depression named ethyl eicosapentaenoic acid (ethyl EPA), an omega 3 fatty acid. You will…
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…
The goal of this study is to examine how effective and well-tolerated a possible new treatment for individuals who injure themselves frequently will be. All research procedures can be done from home no in-person visits are required. We are studying a minimal-risk form of electrical brain stimulation called transcranial direct current stimulation, or tDCS.…
The goal of this study is to examine how effective and well-tolerated a possible new treatment for individuals who injure themselves frequently will be. All research procedures can be done from home no in-person visits are required. We are studying a minimal-risk form of electrical brain stimulation called transcranial direct current stimulation, or tDCS.…
The goal of this study is to examine how effective and well-tolerated a possible new treatment for individuals who injure themselves frequently will be. All research procedures can be done from home no in-person visits are required. We are studying a minimal-risk form of electrical brain stimulation called transcranial direct current stimulation, or tDCS.…
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…
Sertraline (also known as Zoloft) is an FDA approved medication that is used to treat anxiety in children and adolescents. We aim to better understand how children and adolescents with anxiety respond to sertraline and whether we can predict medication response. If your child is eligible, he or she can participate in a research study and will receive both a…
We are currently enrolling individuals with Major Depressive Disorder (MDD) to participate in this research study. This is a one-year open-label study to evaluate the long-term safety and tolerability of an investigational drug called REL-1017 (esmethadone) when taken alone (monotherapy) or when taken with other antidepressants (adjunctive therapy). Study…
Are you between the ages of 18-35? Do you have schizophrenia or schizoaffective disorder? Researchers at the New York State Psychiatric Institute are currently seeking participants who are interested in a brief trial of anti-psychotic medication. This study will include: - magnetic resonance imaging (MRI) - a physical exam - clinical and neurocognitive…
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…
We are currently enrolling individuals with post-traumatic stress disorder (PTSD) to participate in this research study. The purpose of this research study is to evaluate the effectiveness of an investigational medication, brexpiprazole, for PTSD. Eligible participants will be randomly assigned to receive either brexpiprazole, sertraline (also known as…
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…
Do you suffer from depression? Have standard treatments not been helpful? Researchers at Columbia University Irving Medical Center are conducting a study of an experimental antidepressant called ketamine, which can provide rapid relief of depressive symptoms in some patients. The study involves an inpatient stay, during which participants undergo brain…
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…
Our lab is conducting a series of research studies that investigate how specific brain regions are related to functioning in work, school, and in other social situations. As a study participant, you will be asked to complete clinical and neuropsychological assessments, an eye-tracking task, and a non-invasive MRI scan. Study participants earn between $15-50…
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.
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…
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-…
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…
The purpose of this pilot research study is to test whether certain components of the marijuana plant, known as "cannabinoids", may help to reduce symptoms in patients with OCD. Specifically, patients enrolled in the study will smoke marijuana containing different concentrations of 2 different cannabinoids, THC and CBD. Both of these agents act on…
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
Children with Attention-Deficit/Hyperactivity Disorder (ADHD) are typically treated with two types of medications: stimulants and non-stimulants. These medications work in different ways to treat ADHD symptoms, but we do not fully understand the brain mechanisms underlying the medications' effects. This study will use MRI scanning to look at changes in…
This study is investigating how the brains of people with OCD may change with treatment. The study uses Magnetic Resonance Imaging (MRI) to scan the brain, and the treatment used is a form of Cognitive-Behavioral therapy consisting of Exposure and Ritual Prevention (EX/RP). Participants will undergo a scan, which lasts for approximately 2 hours and takes…
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…
This is a phase I, multicenter, dose escalation study of MK-1775 in combination with radiation. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG). MK-1775 is an oral…
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…
Eligible participants will be asked questions about their symptoms, complete questionnaires, and receive sessions of Cognitive-Behavioral therapy consisting of Exposure and Ritual Prevention (EX/RP) accompanied by the use of a mobile app. The purpose of this study is to assess whether the mobile app may be effective in improving symptoms of OCD.
The goals of this study are to find the highest safe dose of ramucirumab that can be given without causing severe side effects, to learn what kind of side effects ramucirumab can cause, to learn more about the effects of ramucirumab on cells and proteins in the blood and to determine whether ramucirumab is a beneficial treatment for patients with solid…
Columbia University Medical Center is presently recruiting individuals ages 18-60, who haveexperienced a traumatic event, to participate in a research treatment study on PTSD. For yourparticipation, you'll receive brief treatment at no cost and $100 in compensation.
The purpose of this study is to find out more about the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558) when given alone or when combined with ipilimumab in children/ adolescents/ young adults with high grade primary central nervous system (CNS) malignancies (certain…
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…
We are trying to understand what causes bipolar disorder and how medications treat bipolar depression. If you participate, you will have two different brain scans (MRI and PET scan). You will also have antidepressant treatment for free. We will then be able to see whether information on the brain scans connects with how people do on the medications. The…
This study is a double-blind and placebo-controlled study to assess its efficacy of the new drug Lumateperone for patients with Major Depressive Episodes associated with Bipolar I and Bipolar II disorder. Lumateperone is thought to exert its antidepressant effect through interaction with sereotogenic, dopaminergic, and glutamergic neurotransmitter targets.…
The Breathe Easy Early ORBEX Study is a three -year study to identify if a daily capsule of Broncho-Vaxom may help prevent or delay young children from developing wheezing or asthma- like symptoms. Other studies have found that Broncho-Vaxom can help prevent upper respiratory infections and stimulate immune defenses, and has been safely and effectively used…
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…
The purpose of this study is to assess the safety and effectiveness of a study drug called axitinib on treating tumors that have returned (recurrent) or have not responded to standard therapy (refractory). Children will take axitinib twice a day for 28 days (1 cycle). Children may take study drug for up to 24 cycles (2 years).
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…
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…
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)…
Patients who are either interested in discontinuing their current antidepressant or have discontinued an antidepressant in the past 6 weeks will be provided with an FDA approved medicine, Vortioxetine (brand name Trintellix). Treatment with Vortioxetine will start after the past antidepressant is discontinued. Vortioxetine dosage is adjustable throughout…
We are trying to understand what causes bipolar disorder and how medications treat bipolar depression. Particularly, we are looking at the importance of dopamine signaling in the process. If you participate, you will have two different brain scans (MRI and PET scan). You will also have treatment for your depression with an FDA approved medication,…
The Center for OCD and Related Disorders is currently conducting a study in treating Obsessive-compulsive Disorder (OCD), which is a severe illness characterized by distressing intrusive thoughts, images, or impulses about a variety of themes (e.g., fears of contamination or harm) and repetitive behaviors such as excessive washing or checking. The study is…
This is a Phase 1 multicenter study of a drug called ABI-009 (nab-Rapamycin) in combination withtemozolomide and irinotecan in children and young adults with solid tumors (including CNS tumors)which have come back or have not responded to standard therapy. The study is considered experimental because ABI-009 (nab-Rapamycin) is not approved by the United…
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…
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.
Your child has been asked to participate in this study because he/she may have or has growth hormone deficiency (i.e. not enough growth hormone in his/her body) and your doctor has already determined that your child may be suitable to be screened for this study. OPKO Biologics Ltd., the Sponsor of this clinical study, has developed an…
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
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)…
This is a pre-marketing (Phase 2A) multi-site study of an investigational drug, ganaxolone, as a potential treatment for non-psychotic post-partum depression. Eligible subjects who agree by signing informed consent will spend four days on the 5-South Research Unit (5-S) of the New York State Psychiatric Institute. For 6 hours they will have an tube in a…
Babies born prematurely frequently have difficulty breathing because of immature lung development. A substance called surfactant is usually present in mature lungs to help keep the lungs inflated normally. The treatment for breathing problems in premature infants can include giving surfactant as a medicine. When surfactant is given as a medicine a…
In this study, we are trying to learn more about how stimulant medication produces an improvement in emotional lability (frequent mood changes and excessive emotional reactions) in patients with Attention Deficit Hyperactivity Disorder (ADHD.) Although ADHD is primarily associated with inattention, hyperactivity, and impulsivity, emotional ability plays a…
The purpose of this study is to find out more about how the study drug called entrectinib will be used in children, adolescents and young adults with treatment-resistant solid tumor in brain or body. This is a phase 1/1b study, divided into a dose escalation portion (different dose will be tested) and a dose expansion portion (one dose will be tested) in…
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…
Columbia University Medical Center is presently recruiting individuals who have experienced trauma to participate in an fMRI research study. For your participation, you will receive up to $270 and an opportunity to advance research in the area of trauma and PTSD. This study involves two visits.
This is a Phase 1 multicenter study of temsirolimus in combination with etoposide and cyclophosphamide. The study treatment is considered experimental because it is not approved by the Unites States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) and non-Hodgkins lymphoma (NHL) who have relapsed. The…
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…
The purpose of this study is to investigate how mothers with babies with congenital heart disease feel about breastfeeding. We will ask you to fill out a survey while you are in the hospital at around the time of delivery and another survey approximately three months later to see how you feel about breastfeeding. In addition to the survey data, the study…
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…
We are currently enrolling individuals with social anxiety disorder to participate in this study. The purpose of this study is to assess whether a brief computer-based research treatment helps improve social anxiety symptoms. For eligible participants, this study will involve a brief, novel research treatment for social anxiety, delivered in 8 or 12…