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ClinicalTrials.gov public records Last synced Sep 6, 2026, 8:03 AM EDT

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Showing 1–24 of 1,261 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Nervous System Heredodegenerative Disorder Clear all
Completed No phase listed Observational Results available

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Neurobehavioral testing, Visual and auditory assessments
Behavioral · Other
Lead sponsor
Shire
Industry
Eligibility
32 Months to 18 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

NCT02006472
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine, Placebo
Drug · Other
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
408 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
16
States / cities
La Jolla, California • Los Angeles, California • Englewood, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2021 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional

A Study of RO4917523 in Patients With Fragile X Syndrome

NCT01517698
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Placebo, RO4917523 0.5 mg, RO4917523 1.5 mg
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
14 Years to 50 Years
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
20
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2016 · Synced Sep 6, 2026, 8:03 AM EDT
Active, not recruiting Not applicable Interventional

Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1

NCT03820778
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Atypical Neurofibroma, Atypical Neurofibromatosis, Plexiform Neurofibroma, Von Recklinghausen Disease
Interventions
Whole Body MRI
Diagnostic Test
Lead sponsor
Children's National Research Institute
Other
Eligibility
8 Years to 30 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 1, 2021 · Synced Sep 6, 2026, 8:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease

NCT02855476
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
11 Years to 75 Years
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2017
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Active, not recruiting Phase 1 Interventional

A Study of BMS-986484 Alone and Combination Therapy in Participants With Advanced Solid Tumors

NCT06544655
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
BMS-986484, Nivolumab, Oxaliplatin, Capecitabine, Fluorouracil, Calcium folinate
Drug · Biological
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Tucson, Arizona • Los Angeles, California • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Recruiting No phase listed Observational

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

NCT05224778
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, CDM
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
Up to 59 Months
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
Los Angeles, California • Fairway, Kansas • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

NCT04007991
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

NCT03325010
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Valbenazine, Placebo oral capsule
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 17 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
34
States / cities
Sun City, Arizona • Rogers, Arkansas • Anaheim, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2021 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2Phase 3 Interventional

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT03231878
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
MIN-102, Placebos
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 17, 2025 · Synced Sep 6, 2026, 8:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Recruiting No phase listed Observational

Longitudinal Impact of Stressors in Adults With Tourette Syndrome

NCT04851678
View directory record Official record
Conditions
Tourette Syndrome
Interventions
None - observational study
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Terminated No phase listed Observational

Giant Axonal Neuropathy Natural History Study

NCT01503125
View directory record Official record
Conditions
Giant Axonal Neuropathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 26, 2017 · Synced Sep 6, 2026, 8:03 AM EDT
Terminated Phase 2 Interventional Results available

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00004418
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
glyceryl trierucate/glyceryl trioleate
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Months to 8 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

NCT01399047
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis
Interventions
Mycophenolate mofetil, Liquid Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
6 Years to 25 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated May 20, 2019 · Synced Sep 6, 2026, 8:03 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 Sep 6, 2026, 8:03 AM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2023 · Synced Sep 6, 2026, 8:03 AM EDT
Completed No phase listed Observational

Natural History Study of FDXR Mutation-related Mitochondriopathy

NCT04580979
View directory record Official record
Conditions
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
Interventions
Mutation analysis
Genetic
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
0 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2024 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

NCT02839720
View directory record Official record
Conditions
Cutaneous Neurofibroma, Neurofibromatosis Type 1, Optic Nerve Glioma
Interventions
Laboratory Biomarker Analysis, Selumetinib Sulfate
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
2
States / cities
Birmingham, Alabama • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 5, 2023 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 1 Interventional

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

NCT01853423
View directory record Official record
Conditions
Facial Angiofibroma
Interventions
Rapamune
Drug
Lead sponsor
Le Bonheur Children's Hospital
Other
Eligibility
3 Years to 45 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 18, 2016 · Synced Sep 6, 2026, 8:03 AM EDT
Terminated No phase listed Observational

NT-3 Levels and Function in Individuals With CMT

NCT05011006
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
Interventions
NT3 blood draw
Diagnostic Test
Lead sponsor
Zarife Sahenk
Other
Eligibility
7 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Results available

Randomized Controlled Study of Donepezil in Fragile X Syndrome

NCT01120626
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
donepezil, sugar pill
Drug
Lead sponsor
Stanford University
Other
Eligibility
12 Years to 29 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 16, 2016 · Synced Sep 6, 2026, 8:03 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1

NCT04481048
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-Acetyl cysteine, Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Sep 6, 2026, 8:03 AM EDT
Recruiting Phase 1 Interventional

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355
View directory record Official record
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 6, 2026, 8:03 AM EDT