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ClinicalTrials.gov public records Last synced Oct 19, 2026, 3:45 AM EDT

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Showing 553–576 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Recruiting No phase listed Observational

Long-Term Development of Muscular Dystrophy Outcome Assessments

NCT05989620
View directory record Official record
Conditions
LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 50 Years
Enrollment
1,000 participants
Timeline
2023 – 2029
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 19, 2026, 3:45 AM EDT
Completed No phase listed Observational

Individuals Patterns of Disclosure About Huntington s Disease (HD) and the Association With Adaptation to HD

NCT00491842
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 19, 2026, 3:45 AM EDT
Completed No phase listed Observational

Biomarker Testing and Treatment Patterns Among Patients With PIK3CA Mutation in Advanced Breast Cancer

NCT05982886
View directory record Official record
Conditions
Breast Cancer
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
1,093 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 9, 2023 · Synced Oct 19, 2026, 3:45 AM EDT
Active, not recruiting No phase listed Observational

Natural History of Spinocerebellar Ataxia Type 7 (SCA7)

NCT02741440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 19, 2026, 3:45 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Feasibility and Clinically Application of Magnetic Resonance Fingerprinting

NCT02387840
View directory record Official record
Conditions
Neurofibromatosis Type 1, Brain Tumor, Glioma
Interventions
Magnetic Resonance Imaging, Magnetic Resonance Fingerprinting
Device
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 35 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 12, 2021 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resiliency Training in Adolescents With NF1 and NF2

NCT03873610
View directory record Official record
Conditions
Neurofibromatoses
Interventions
Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
12 Years to 17 Years
Enrollment
196 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 19, 2026, 3:45 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 13, 2026 · Synced Oct 19, 2026, 3:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial

NCT04879160
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
7 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 22, 2024 · Synced Oct 19, 2026, 3:45 AM EDT
Completed No phase listed Observational Results available

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215
View directory record Official record
Conditions
Wilson Disease
Interventions
Standard of Care Medications
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2020 · Synced Oct 19, 2026, 3:45 AM EDT
Recruiting No phase listed Observational

Clinical and Neuropsychological Investigations in Batten Disease

NCT01873924
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2035
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2025 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1

NCT00853580
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Lovastatin ™, placebo
Drug · Device
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
8 Years to 15 Years
Enrollment
146 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2018 · Synced Oct 19, 2026, 3:45 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

NCT07748403
View directory record Official record
Conditions
Wilson Disease, Wilson's Disease, Wilsons Disease
Interventions
PM577a
Drug
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

NCT01350440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
IVIG
Biological
Lead sponsor
University of South Florida
Other
Eligibility
10 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 23, 2013 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Early Phase 1 Interventional

Pimavanserin Treatment in TS

NCT04794413
View directory record Official record
Conditions
Tourette Syndrome, Tardive Dyskinesia
Interventions
Pimavanserin
Drug
Lead sponsor
Joseph Jankovic
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 12, 2021 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Phase 2 Interventional

Effects of Lithium and Divalproex'on Brain-Derived Neurotrophic Factor in Huntington's Disease

NCT00095355
View directory record Official record
Conditions
Huntington's Disease
Interventions
Lithium, Divalproex
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 19, 2026, 3:45 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors

NCT04657068
View directory record Official record
Conditions
Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Endometrial Cancer, Metastatic Colorectal Cancer, Pancreatic Ductal Adenocarcinoma, Acinar Cell Carcinoma
Interventions
ART0380, Gemcitabine, Irinotecan
Drug
Lead sponsor
Artios Pharma Ltd
Industry
Eligibility
18 Years and older
Enrollment
542 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
41
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 33 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 19, 2026, 3:45 AM EDT
Not listed Not applicable Interventional

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder

NCT06385535
View directory record Official record
Conditions
Tics, Tic Disorders, Vocal Tic, Motor Tic, Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tics/Tremor
Interventions
Habit Reversal Training (HRT)
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 12 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 26, 2024 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Phase 3 Interventional Results available

Study Evaluating the Safety and Efficacy of Fixed-dose Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT01727700
View directory record Official record
Conditions
Tourette's Disorder, Tic Disorder
Interventions
Aripiprazole, Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 17 Years
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
55
States / cities
Dothan, Alabama • Goodyear, Arizona • Tucson, Arizona + 52 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2015 · Synced Oct 19, 2026, 3:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Preparing for Prevention of Huntington's Disease (PREVENT-HD)

NCT04818060
View directory record Official record
Conditions
Huntington Disease
Interventions
Clinical Assessments, MRI Scan, Lumber Puncture (LP)
Other · Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 80 Years
Enrollment
258 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 19, 2026, 3:45 AM EDT
Completed Not applicable Interventional

Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1

NCT05377008
View directory record Official record
Conditions
Neurocognitive Deficit, Mental Health Impairment, Neurofibromatosis 1
Interventions
OPTIMAL-SSI
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 19, 2026, 3:45 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 29, 2024 · Synced Oct 19, 2026, 3:45 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 27, 2016 · Synced Oct 19, 2026, 3:45 AM EDT
Not listed Phase 3 Interventional

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
20
States / cities
Los Angeles, California • Sacramento, California • New Britain, Connecticut + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 19, 2026, 3:45 AM EDT