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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:20 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 649–672 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Pancreatic Cancer Registry: For Any Person With a Personal or Family History

NCT02886247
View directory record Official record
Conditions
Pancreatic Cancer, ATM, BRCA, Hereditary Cancer
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
12,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2032
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Completed No phase listed Observational

Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

NCT01767779
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Day to 6 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2019 · Synced Oct 21, 2026, 1:20 AM EDT
Active, not recruiting Not applicable Interventional

Pediatric Powered Wheelchair Standing Devices: An Exploratory Study

NCT05117827
View directory record Official record
Conditions
Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida, Development Delay, Genetic Disease
Interventions
Power Wheelchair Standing Device
Device
Lead sponsor
Grand Valley State University
Other
Eligibility
5 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 21, 2026, 1:20 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 11, 2022 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360
View directory record Official record
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2020 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Phase 2 Interventional

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NCT02728388
View directory record Official record
Conditions
NEUROFIBROMATOSIS 1
Interventions
aminolevulinic acid
Drug
Lead sponsor
Donald Basel
Other
Eligibility
14 Years to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

NCT03403205
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, SoC Therapy
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2024 · Synced Oct 21, 2026, 1:20 AM EDT
Recruiting Not applicable Interventional

Stem Cell Ophthalmology Treatment Study II

NCT03011541
View directory record Official record
Conditions
Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions
Arm 1
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Recruiting No phase listed Observational

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

NCT05354622
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Month to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Change in MUNIX in Patients With CMT1A Undergoing a Home Ankle Strengthening Program Versus Standard of Care

NCT03715283
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Home Ankle Strengthing Program, Standard of Care
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 20, 2019 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 2 Interventional Results available

Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas

NCT00076102
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
15
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2018 · Synced Oct 21, 2026, 1:20 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)

NCT05269394
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
E2814, Lecanemab, Matching Placebo (E2814)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
197 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
12
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Not applicable Interventional

Aripiprazole in the Treatment of Tourette's Syndrome

NCT00282139
View directory record Official record
Conditions
Tourette's Syndrome
Interventions
Aripiprazole
Drug
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 17 Years
Enrollment
40 participants
Timeline
2004 – 2006
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 5, 2011 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 2 Interventional

Creatine Safety and Tolerability in Premanifest HD: PRECREST

NCT00592995
View directory record Official record
Conditions
Huntington Disease
Interventions
Creatine monohydrate, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
26 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Phase 2 Interventional

Phase 2 Study to Determine if RTB101 Reduces the Severity of COVID-19 in Older Adults Residing in Nursing Homes

NCT04409327
View directory record Official record
Conditions
COVID19
Interventions
RTB101, Placebo
Drug
Lead sponsor
Restorbio Inc.
Industry
Eligibility
65 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Middletown, Rhode Island
Source: ClinicalTrials.gov public record
Updated Feb 10, 2021 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer

NCT03437941
View directory record Official record
Conditions
Metastatic Castration-Resistant Prostate Cancer
Interventions
Exicorilant, Enzalutamide, Placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Scottsdale, Arizona • Detroit, Michigan • Basking Ridge, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 2 Interventional

Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3

NCT00992771
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
varenicline, placebo
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 18, 2012 · Synced Oct 21, 2026, 1:20 AM EDT
Terminated Phase 2Phase 3 Interventional

STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia

NCT05490563
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
SLS-005, Placebo
Drug
Lead sponsor
Seelos Therapeutics, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2024 · Synced Oct 21, 2026, 1:20 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 2 Interventional Results available

Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL)

NCT00920699
View directory record Official record
Conditions
Huntington's Disease
Interventions
CoQ10
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
13
States / cities
Sacramento, California • Englewood, Colorado • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2017 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 1 Interventional

Phase I Trial of Oral PX-866

NCT00726583
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
PX-866
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
2
States / cities
Aurora, Colorado • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 17, 2018 · Synced Oct 21, 2026, 1:20 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

NCT07505342
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 88 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 21, 2026, 1:20 AM EDT
Completed No phase listed Observational

Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

NCT00844129
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
6 Years to 9 Years
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 3, 2018 · Synced Oct 21, 2026, 1:20 AM EDT