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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 AM EDT
Approved For Marketing No phase listed Expanded access

3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)

NCT00872950
View directory record Official record
Conditions
Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndrome
Interventions
3,4-DIAMINOPYRIDINE
Drug
Lead sponsor
Lahey Clinic
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Burlington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 10, 2022 · Synced Jul 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 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 23, 2026, 5:05 AM EDT
Completed Phase 2 Interventional Results available

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

NCT02561702
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Placebo, Mexiletine
Other · Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced Jul 23, 2026, 5:05 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Prospective Huntington At Risk Observational Study

NCT00052143
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
26 Years to 55 Years
Enrollment
1,001 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2009
U.S. locations
37
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 32 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2024 · Synced Jul 23, 2026, 5:05 AM EDT
Completed Phase 2 Interventional Results available

Everolimus for Cancer With TSC1 or TSC2 Mutation

NCT02201212
View directory record Official record
Conditions
TSC1, TSC2, Tuberous Sclerosis Complex, MTOR
Interventions
Everolimus
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 15, 2020 · Synced Jul 23, 2026, 5:05 AM EDT
Completed No phase listed Observational

Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities

NCT01966757
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis, Batten's Disease
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 25, 2016 · Synced Jul 23, 2026, 5:05 AM EDT
Recruiting Not applicable Interventional

Home-based TDCS (Transcranial Direct Current Stimulation) for Cognitive and Behavioral Symptoms in Huntington's Disease

NCT06843252
View directory record Official record
Conditions
Huntington Disease
Interventions
Transcranial direct current stimulation (tDCS), Sham tDCS
Device
Lead sponsor
The University of Texas Health Science Center at San Antonio
Other
Eligibility
18 Years to 85 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Jul 23, 2026, 5:05 AM EDT
Completed No phase listed Observational

Transient Receptor Potential Vanilloid One (TRPV1) Receptor Expression in Children With Retrosternal Pain

NCT00677378
View directory record Official record
Conditions
Epigastric Pain, Retrosternal Pain, Esophagitis, Gastrointestinal Symptoms
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
5 Years to 17 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 21, 2015 · Synced Jul 23, 2026, 5:05 AM EDT
Completed Phase 2 Interventional Results available

Medical Treatment of "High-Risk" Neurofibromas

NCT00846430
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar), Vincristine Sulfate (Oncovin)
Drug
Lead sponsor
Corewell Health West
Other
Eligibility
2 Years to 30 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Jul 23, 2026, 5:05 AM EDT
Completed Phase 2 Interventional Results available

Combination Study of RMC-4630 and Sotorasib for NSCLC Subjects With KRASG12C Mutation After Failure of Prior Standard Therapies

NCT05054725
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
RMC-4630, Sotorasib
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Fort Myers, Florida • Plantation, Florida • Saint Augustine, Florida + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2026 · Synced Jul 23, 2026, 5:05 AM EDT
Not listed Phase 3 Interventional

OnabotulinumtoxinA (onaBoNT-A) Versus Oral Oxybutynin ER

NCT01050114
View directory record Official record
Conditions
Overactive Bladder
Interventions
onaBoNT-A, Oxybutynin ER
Drug
Lead sponsor
Christopher Patrick Smith
Other
Eligibility
18 Years to 80 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 29, 2016 · Synced Jul 23, 2026, 5:05 AM EDT