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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Nervous System Heredodegenerative Disorder Clear all
Recruiting No phase listed Observational

Neurofibromatosis Type 1 Tumor Early Detection Study

NCT06515860
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibromatosis 1, Plexiform Neurofibroma, Plexiform Neurofibromas, Malignant Peripheral Nerve Sheath Tumor, Malignant Peripheral Nerve Sheath Tumors, Atypical Neurofibroma
Interventions
Liquid biopsy for MPNST development
Diagnostic Test
Lead sponsor
David Miller
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Recruiting Not applicable Interventional

Trajectories of Change in Tourette Syndrome

NCT06679790
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Comprehensive Behavioral Intervention for Tics
Behavioral
Lead sponsor
University of Minnesota
Other
Eligibility
12 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Recruiting No phase listed Observational

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 1 Interventional

Open-Trial of EPI-743 for Adults With Tourette Syndrome

NCT01719523
View directory record Official record
Conditions
Tourette Syndrome
Interventions
EPI-743
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Not applicable Interventional

Treadmill Walking in Individuals With Dementia With Lewy Bodies and Huntington's Disease

NCT02268617
View directory record Official record
Conditions
Huntington Disease, Lewy Body Disease
Interventions
Treadmill Walking
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 90 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 26, 2016 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

NCT02544750
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 13, 2022 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Rett Syndrome With Recombinant Human IGF-1

NCT01777542
View directory record Official record
Conditions
Rett Syndrome
Interventions
Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 10 Years · Female only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced Jul 22, 2026, 9:03 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

NCT02895789
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2022 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain

NCT01633008
View directory record Official record
Conditions
Neurofibromatosis
Interventions
ACT Workshop
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2019 · Synced Jul 22, 2026, 9:03 AM EDT
Completed No phase listed Observational

Protein Synthesis in the Brain of Patients With Fragile X Syndrome

NCT00362843
View directory record Official record
Conditions
Fragile X Syndrome, Healthy Volunteers
Interventions
[15-O]water
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years to 24 Years · Male only
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 2Phase 3 Interventional

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

NCT06849973
View directory record Official record
Conditions
Rett Syndrome
Interventions
Drug: Bionetide, Placebo
Drug
Lead sponsor
Biomed Industries, Inc.
Industry
Eligibility
5 Years to 20 Years · Female only
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
12
States / cities
La Jolla, California • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Not applicable Interventional

Teaching Math Skills to Individuals With Fragile X Syndrome

NCT01204151
View directory record Official record
Conditions
Developmental Disabilities, Fragile X Syndrome
Interventions
discrete-trial training
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
10 Years to 23 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 8, 2019 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 2 Interventional Results available

Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma

NCT01553149
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
Interventions
Lenalidomide, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
113
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 90 more
Source: ClinicalTrials.gov public record
Updated Jan 6, 2025 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Not applicable Interventional

Online Mindfulness-based Tic Reduction (Phase Two)

NCT03771235
View directory record Official record
Conditions
Tourette Syndrome, Persistent Tic Disorder
Interventions
Mindfulness-based Intervention for Tics, Tic Information and Coping Strategies
Behavioral
Lead sponsor
Bowdoin College
Other
Eligibility
18 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
2
States / cities
Brunswick, Maine • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 28, 2024 · Synced Jul 22, 2026, 9:03 AM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease

NCT07451613
View directory record Official record
Conditions
Huntington Disease
Interventions
hNSC-01
Biological
Lead sponsor
Leslie Thompson
Other
Eligibility
18 Years to 65 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Completed No phase listed Observational

Pediatric Patients With Metabolic or Other Genetic Disorders

NCT02769949
View directory record Official record
Conditions
Genetic Disorder, Asperger Disorder, Autism Spectrum Disorder, Fragile X Syndrome, Developmental Delay
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Year to 99 Years
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Jul 22, 2026, 9:03 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

NCT04886518
View directory record Official record
Conditions
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Interventions
Pitolisant Oral Tablet, Placebo oral tablet
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced Jul 22, 2026, 9:03 AM EDT
Terminated Not applicable Interventional Results available

Thalamic Deep Brain Stimulation for the Treatment of Refractory Tourette Syndrome

NCT01817517
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Medtronic Activa Deep Brain Stimulation System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
15 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
View directory record Official record
Conditions
Friedreich Ataxia, Neuro-Degenerative Disease
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 80 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2030
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2024 · Synced Jul 22, 2026, 9:03 AM EDT
Active, not recruiting Phase 3 Interventional

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
18
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced Jul 22, 2026, 9:03 AM EDT
Completed No phase listed Observational

Assessment of Children With Tic Onset in the Past 6 Months

NCT01177774
View directory record Official record
Conditions
Tourette Syndrome, Tourette's Disorder, Chronic Motor or Vocal Tic Disorder, Transient Tic Disorder, Tic Disorders, Provisional Tic Disorder
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
5 Years to 10 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 26, 2023 · Synced Jul 22, 2026, 9:03 AM EDT
Active, not recruiting Phase 2 Interventional

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

NCT02171104
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jul 22, 2026, 9:03 AM EDT
Not listed No phase listed Observational

NBIAready: Online Collection of Natural History Patient-reported Outcome Measures

NCT02587858
View directory record Official record
Conditions
Neurodegeneration With Brain Iron Accumulation (NBIA)
Interventions
Not listed
Lead sponsor
Susan J. Hayflick
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 7, 2020 · Synced Jul 22, 2026, 9:03 AM EDT
Terminated Early Phase 1 Interventional Results available

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

NCT02282917
View directory record Official record
Conditions
Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions
AR-42
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Jul 22, 2026, 9:03 AM EDT