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ClinicalTrials.gov public records Last synced Oct 18, 2026, 9:42 AM EDT

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Showing 529–552 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Not applicable Interventional

Online Mindfulness-based Tic Reduction

NCT03525626
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Online Mindfulness-based Tic Reduction
Behavioral
Lead sponsor
Bowdoin College
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
2
States / cities
Brunswick, Maine • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 9, 2019 · Synced Oct 18, 2026, 9:42 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Inherited Diseases, Caregiving, and Social Networks

NCT01498263
View directory record Official record
Conditions
Undiagnosed Disease, Healthy Volunteer (Adult With Typically Developing Child), Inherited Neurodegenerative Disorders, Inherited Metabolic Disorders (Inborn Errors of Metabolism), Alzheimer's Disease and Related Dementias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
682 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
2
States / cities
Bethesda, Maryland • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 17, 2022 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting No phase listed Observational

Sensory Symptoms in Tourette Syndrome

NCT03851484
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Completed No phase listed Observational

Down Syndrome Biomarker Initiative (DSBI)

NCT02141971
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
Michael Rafii
Other
Eligibility
30 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated May 2, 2017 · Synced Oct 18, 2026, 9:42 AM EDT
Terminated Phase 2 Interventional Results available

Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities

NCT04572893
View directory record Official record
Conditions
Primary Familial Dilated Cardiomyopathy
Interventions
danicamtiv
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
La Jolla, California • Washington D.C., District of Columbia • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Not applicable Interventional Results available

Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome

NCT02067819
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Active Oral Orthotic Treatment, Placebo Oral Orthotic Treatment
Device
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
7 Years to 25 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2022 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Not applicable Interventional Results available

Brain Stimulation for the Treatment of Tourette Syndrome

NCT01329198
View directory record Official record
Conditions
Tourette Syndrome
Interventions
NeuroPace RNS® System Deep Brain Stimulator
Device
Lead sponsor
University of Florida
Other
Eligibility
25 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 8, 2018 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Enrolling by invitation No phase listed Observational

GABA and GSH in FRDA

NCT07635030
View directory record Official record
Conditions
Friedreich's Ataxia, FRDA
Interventions
MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy)
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
8 Years to 16 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting Phase 2 Interventional

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

NCT07237100
View directory record Official record
Conditions
Advanced Unresectable Melanoma, Metastatic Melanoma
Interventions
Mirdametinib
Drug
Lead sponsor
Kevin Kim, MD
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Oct 18, 2026, 9:42 AM EDT
Completed No phase listed Observational

Memory Disorders Registry

NCT00654563
View directory record Official record
Conditions
Dementia, Alzheimer's Disease, Parkinson Disease, Huntington Disease
Interventions
Not listed
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 27, 2012 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting Not applicable Interventional

Neuromodulation to Enhance Motor Function in HSP

NCT07417943
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Completed No phase listed Observational

A Pre-Cellular Therapy Observational Study in Early Huntington's Disease

NCT01937923
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 1, 2016 · Synced Oct 18, 2026, 9:42 AM EDT
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Not yet recruiting No phase listed Observational

Nemaline Myopathy Clinical Research Network (NM-CTRN)

NCT06774703
View directory record Official record
Conditions
Nemaline Myopathy
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
0 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Palo Alto, California • Bethesda, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Oct 18, 2026, 9:42 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 5, 2014 · Synced Oct 18, 2026, 9:42 AM EDT
Completed No phase listed Observational

Studies in Patients With Tuberous Sclerosis Complex

NCT03276195
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Translational Genomics Research Institute
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 29, 2023 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

NCT00411619
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Phase 1 Interventional

A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer

NCT03423628
View directory record Official record
Conditions
Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme, Brain Neoplasms, Malignant, Leptomeningeal Disease (LMD)
Interventions
Radiation Therapy, AZD1390
Radiation · Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting No phase listed Observational

TSC Biosample Repository and Natural History Database

NCT05676099
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Phlebotomy, Buccal (cheek) swab, Genetic Testing, Tissue donation after routine clinical procedure
Procedure · Genetic · Other
Lead sponsor
National Tuberous Sclerosis Association
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
24
States / cities
Birmingham, Alabama • Loma Linda, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Recruiting Not applicable Interventional

Deep Brain Stimulation for Spinocerebellar Ataxia

NCT07288437
View directory record Official record
Conditions
Spinocerebellar Ataxia (SCA), Spinocerebellar Ataxia Type 6
Interventions
Deep Brain Stimulation (DBS)
Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
21 Years to 89 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 18, 2026, 9:42 AM EDT
Completed Phase 1 Interventional

Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma

NCT00352495
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Neurofibromatosis Type 1
Interventions
carboplatin, vinblastine sulfate
Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
19
States / cities
Birmingham, Alabama • Orange, California • Washington D.C., District of Columbia + 16 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2014 · Synced Oct 18, 2026, 9:42 AM EDT