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Displaying 201 - 281 of 281

A study for adult patients with recurrent glioblastoma multiforme using study drug SL-701

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to find out if SL-701 is safe and tolerable for patients with recurrent glioblastoma multiforme (GBM). The study will also look at what effect SL-701 has on patient's brain tumor. SL-701 is a type of anti-cancer vaccine made up of parts 3 different short proteins called "peptides" which are frequently found on…
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Study of DSP-7888 in Combination with Bevacizumab in Patients with Recurrent or Progressive Glioblastoma

Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
The purpose of this study is to see if the newly discovered vaccine drug, DSP-7888, will help your immune system to fight against cancer cells that have a protein called WT1. The study will also compare the effectiveness of treatment with DSP-7888 in combination with Bevacizumab versus Bevacizumab alone in patients with glioblastoma (GBM). Bevacizumab has…
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A study for patients with Multiple Sclerosis (MS) using study drug ocrelizumab for disease-modifying treatment

Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Claire Riley, MD
Status: Closed
The purpose of this study is to see if ocrelizumab (study drug) will stabilize, or potentiallyimprove the signs and symptoms of your MS. Ocrelizumab is a type of drug called amonoclonal antibody. Monoclonal antibodies act like your bodys immune system andattach to certain cells in order to attack germs and other illnesses in your body.Ocrelizumab attaches…
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ANG1005 in Leptomeningeal Disease From Breast Cancer (ANGLeD)

Condition: Neurological Disorders / Brain Tumors
Investigator: Aya Haggiagi, MD
Status: Closed
This research study compares the effects (good and/or bad), of the study drug (ANG1005) with standard treatments currently available for leptomeningeal metastases to find out which is better. In this study, you will get either ANG1005 or standard treatment available for leptomeningeal metastases. You will not get both.
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Trial of Combination TTF(Optune), Nivolumab Plus/Minus Ipilimumab for Recurrent Glioblastoma

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
In this research study, we are looking for Information on how well nivolumab with or without ipilimumab works in combination with TTFields in adult patients with glioblastoma. You will receive the Nivolumab and/or Ipilimumab study treatment until you decide to stop study treatment or your study doctor decides to remove you from the study. This means that…
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A study for patients with glioblastoma using study drug ICT-107

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
This is a research study to test the effectiveness of ICT-107, an experimental drug, with the standard of care (SOC) for treatment of glioblastoma compared to enriched blood cells (PBMC) with the SOC. The study will enroll 400 subjects age 18 years and older with newly diagnosed glioblastoma. Following an initial SOC radiation treatment, patients will be…
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Study of Nivolumab (Study Drug) for Recurrent or Progressive IDH Mutant Gliomas

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test how well the study drug works in recurrent or isocitratedehydrogenase (IDH) mutant high grade gliomas. The study drug is called Nivolumab. Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow your own body's immune system to work against tumor cells. Nivolumab is not…
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Study of Weekly MOD-4023 Compared to Daily Genotropin Therapy In Pre-Pubertal Children With Growth Hormone Deficiency

Condition: Pediatrics
Investigator: Ilene Fennoy, MD
Status: Closed
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…
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Study of MK-1775 in Combination with Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumor

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This is a phase 1/2 multicenter study of AZD1775 (MK-1775) that will be given in combination with irinotecan. 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 solid tumors which have come back or have not responded to standard…
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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 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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Study of Drug in Patients with Recurrent Brain Tumors (Glioma)

Condition: Neurological Disorders / Brain Tumors
Investigator: Jeffrey Bruce, MD
Status: Closed
The purpose of this study is to determine the safety of directly injecting a tumor chemotherapy drug called Topotecan and a contrast agent called Gadolinium into malignant brain tumors.
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A study for patients with malignant glioblastoma or glioma using study drug PF-06840003

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this research study is to learn about the effects of the study drug, PF-06840003,and to find the best dose for treating research subjects/participants with malignant glioblastoma or anaplastic glioma. PF-068400003 is a new investigational drug. A new investigational drug is one that is currently not approved for sale in this country. PF-…
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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 Combination Therapies in Children with Relapsed Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkins Lymphoma (NHL)

Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
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…
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A study for patients with recurrent Glioblastoma using study drug VB-111

Condition: Neurological Disorders / Brain Tumors
Investigator: Teri Kreisl, MD
Status: Closed
This is a research study to test the effects of VB-111 combined with Bevacizumab versus Bevacizumab alone on glioblastoma . This study will enroll 252 subjects age 18 years and older who have recurrent glioblastoma. All subjects will be randomly assigned in a 1:1 ratio to receive either VB-111 combined with Bevacizumab or Bevacizumab alone. Survival, tumor…
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Effects of Dysport (Botulinum toxin) Injections on Energy Consumption and Walking Efficiency in Children with Cerebral Palsy

Condition: Neurological Disorders
Investigator: Heakyung Kim, MD
Status: Closed
This study aims to evaluate the efficacy of single event multi level chemoneurolysis with the a botulinum toxin A drug, Dysport, on walking energy expenditure and gait in children with spastic diplegia Cerebral Palsy.
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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 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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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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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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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 elderly individuals with malignant brain tumors using experimental drug, bevacizumab combined with temozolomide

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to find out whether an experimental drug, bevacizumab with temozolomide is safe when given to elderly patients with brain tumors and investigate the side effects of the drug combination and how often they occur. This study will also explore whether bevacizumab and temozolomide can be effective in shrinking tumors when given to…
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Study of the efficacy and safety of Selinexor (KPT-330) in patients with recurrent gliomas

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this research study is to see if Selinexor (KPT-330) has any effects against glioblastoma, a type of brain cancer. The study will be conducted at 3-5 sites in the United States and Europe and plan to enroll 30 patients with brain cancer. Patients will be screened prior to study participation. If their brain tumor is evaluated to require…
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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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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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Study for Management of Eye Side Effects in Patients with EGFR-Amplified Glioblastoma Receiving ABT-414

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The main purpose of this study is to improve treatment of ocular side effects that result from depatuxizumab mafodotin (Depatux-M) use in patients with glioblastoma (GBM). Most people treated with Depatux-M will experience eye side effects. When these side effects are not tolerated, Depatux-M is usually interrupted until symptoms improve and then restarted…
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Study of Combination Treatment to Reduce Occurrence and Severity of Chronic Graft Versus Host Disease (GVHD)

Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
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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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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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Study of Memantine and Radiotherapy in Patients with Brain Tumors

Condition: Neurological Disorders / Brain Tumors
Investigator: Tony J. Wang, MD
Status: Closed
The purpose of this study is to compare any good and bad effects of avoiding the hippocampus during whole-brain radiation plus memantine to using the usual whole-brain radiation plus memantine. The hippocampus is a brain structure that is important for memory. The addition of the hippocampal avoidance technique to the usual whole-brain radiation plus…
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Flex CMT Muscle Cramps Study

Condition: Pediatrics / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
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A study for patients with Glioblastoma using study drug Tesevatinib monotherapy

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The reason for this study is to find out if the experimental study drug, tesevatinib, is effective as a possible treatment for recurrent glioblastoma. Certain genetic information about your tumor will also be collected, by drawing blood, to help better understand why tesevatinib did or did not work in treating your recurrent glioblastoma. Although…
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A Study for Charcot-Marie-Tooth type 1A Patients to Compare 2 doses of PXT3003 vs. a Placebo

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Charcot-Marie-Tooth type 1A (CMT1A) disease is a rare, slowly progressing and debilitating hereditary condition commonly causing weakness of muscles in the feet and lower parts of the legs. There is currently no approved drug to cure CMT1A disease, and the available treatments commonly target the pain and inflammation associated with the disease. In order…
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REGAL- Recurrent Glioblastoma Treated with Selinexor and Temozolomide

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of the research study is to see if selinexor has any effects against your cancer and toevaluate the side-effects of selinexor or selinexor in combination with temozolomide (TMZ ). One specific way cancer cells continue to grow is by getting rid of certain proteins called tumor suppressor proteins that would normally cause cancer cells to die.…
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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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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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Study of Drug in Patients with Progressive Supranuclear Palsy

Condition: Neurological Disorders / Neuromuscular
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a study for persons with a diagnosis of Progressive Supranuclear Palsy, involving an experimental treatment.
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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 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 mild to moderate Alzheimer's Disease using study drug T-817MA

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
The purpose of this study is to determine whether an investigational drug, T-817MA, is safe and beneficial in delaying or altering the decline in memory and daily functioning when given to people with Alzheimer's disease. Participants will be given either an active study drug (T-817) or placebo (no active medication).
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Magnetic Resonance Spectroscopy (MRS) to assess progression of Alzheimer Disease (AD)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
THE PURPOSE OF THIS STUDY is to find out whether a type of brain imaging (called "Magnetic Resonance Spectroscopy Imaging" of "MRS") might be useful to help assess the presence of Alzheimer's disease (AD) and assess progression of the disease. MRS uses magnetic resonance imaging (MRI) methods to image the chemicals in the brain.…
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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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A study for patients with progressive meningioma using study device NovoTTF-100A

Condition: Neurological Disorders / Brain Tumors
Investigator: Teri Kreisl, MD
Status: Closed
The purpose of this study is to find out what effects, good or bad, the experimental device, NovoTTF-100A has on patients with meningioma. This study is being done because currently there are no proveneffective medical treatments for a progressive meningioma that has failed surgery and/or radiation. The NovoTTF-100A System is a portable medical device. It…
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Study Treatment (INO-5401 and INO-9012 delivered by electoporation device in combination with REGN2810) in Subjects with Glioblastoma

Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
The purpose of this clinical study is to evaluate safety of the study treatment, its effects on the immune system and on GBM. The study treatment includes several experimental therapies: INO-5401 + INO-9012 delivered by an electroporation device called CELLECTRA 2000 used for delivery of INO-5401 + INO-9012 into your muscle. You will also receive another…
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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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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 infants with pre-symptomatic Spinal Muscular Atrophy (SMA)

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
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…
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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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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 glioblastoma using study drub nivolumab

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558). Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the bodys immune system to work against tumor cells. The effectiveness of BMS-936558…
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Nivolumab and Multi-fraction Stereotactic Radiosurgery (Non-Surgical Radiation Therapy) in Treating Participants With Meningioma

Condition: Neurological Disorders / Brain Tumors
Investigator: Aya Haggiagi, MD
Status: Closed
This trial studies the side effects and best dose of nivolumab when given together with radiosurgery and to see how well they work with or without ipilimumab in treating participants with grade II-III meningioma that has come back. Nivolumab and ipilimumab (two different types of immunotherapy drugs), may interfere with the ability of tumor cells to grow…
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MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (1 month to 17 years old)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
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.
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A study for patients with ALS using study drug NP001

Condition: Neurological Disorders / Neuromuscular
Investigator: Jinsy Andrews, MD, MSc
Status: Closed
The purpose of this research study is to test an investigational drug called NP001. The study will evaluate if NP001 has an effect on slowing the signs and symptoms of your ALS, if there are any side effects, and if there are any medical problems with giving the drug as a solution into your vein (intravenous IV administration). An investigational drug is…
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A Study for Patients with Neuropathy Associated Pain to Compare study drugs Nortriptyline, Duloxetine, Pregabalin and Mexiletine

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
Peripheral Neuropathy is a result of nerve damage, which may cause weakness, numbness, tingling, and pain in your hands, feet, and other areas of your body. Over 20 million Americans are affected by peripheral neuropathy, most commonly caused by diabetes. In approximately 25-50% of all people with peripheral neuropathy, the cause is unknown. These cases…
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Study of Armodafinil (Nuvigil) to Reduce Cancer-Related Fatigue in Patients with High-Grade Glioma (HGG)

Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
The purpose of this study is to: See if taking the study agent, armodafinil, at a dose of 150mg or 250mg, will improve problems with fatigue in patients who have been diagnosed with cancer and are experiencing fatigue. See the effects (good and bad) of taking Armodafinil compared to placebo (an inactive agent) on cancer-related fatigue
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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 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 with refractory solid tumors, including CNS tumors using study drug ramucirumab

Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
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…
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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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Study of Treatment Effects in Subjects with Glioblastoma Multiforme (GBM) - Glioma, Brain Tumor

Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
New data from the study of ABT-414 and ABBV-221 in patients with recurrent glioblastoma multiforme (GBM) have shown several examples where it seems evidence of disease progression (cancer that continues to grow and spread) was seen in MRI images, but examination of surgically removed tissue under the microscope did not agree with the MRI.
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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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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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Study for children with recurrent or refractory solid tumors using study drug axitinib

Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
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).
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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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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 recurrent glioblastoma or anaplastic astrocytoma using study drug Toca 511

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
This is a research study to test the effectiveness of Toca 511/Toca FC, an experimental drug, for treatment of recurring brain tumors compared to current standard of care. The study will enroll 370 subjects age 18-85 years who choose to undergo removal of the tumor a second time. At the time of surgery patients will be randomly assigned in a 1:1 ratio to…
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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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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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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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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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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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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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Testing the benefits of the vitamin supplement Benfotiamine in patients with Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Yaakov Stern, PhD
Status: Closed
The goal of this study is to investigate whether treatment with the vitamin supplement benfotiamine for one year will slow the memory problems in patients who meet the definition of Alzheimer's disease or amnestic mild cognitive impairment, an early manifestation of Alzheimer's disease.
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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 MGMT-Methylated Glioblastoma using study drug temozolomide

Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558). Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the body's immune system to work against tumor cells.
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A Study of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (MyCIDPchoice)

Condition: Neurological Disorders / Neuropathy
Investigator: Thomas Brannagan, MD
Status: Closed
We are doing this study to find out if the research drug known as Rozanolixizumab can have additional benefit for people who are already receiving IVIG( Intravenous Immune Globulin) treatment for CIDP, and to have further safety evaluation of the research drug as well. If you choose to be part of this research study, the following procedures will occur:…
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Decitabine and Vorinostat Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) in Children & Young Adults with Acute Myeloid Leukemia

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). 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 myeloid leukemia (AML)…
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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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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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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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