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ClinicalTrials.gov public records Last synced Oct 15, 2026, 10:35 AM EDT

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

Showing 265–288 of 1,146 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 4 Interventional

N-of-1 for Beta-Blockers in Cardiac Amyloidosis

NCT05019027
View directory record Official record
Conditions
Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions
Beta blocker
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
65 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 15, 2026, 10:35 AM EDT
Recruiting No phase listed Observational

Molecular and Genetic Studies of Congenital Myopathies

NCT00272883
View directory record Official record
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 15, 2026, 10:35 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 15, 2026, 10:35 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 15, 2026, 10:35 AM EDT
Completed Phase 3 Interventional Results available

Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease

NCT04102579
View directory record Official record
Conditions
Chorea, Huntington
Interventions
Valbenazine, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
42
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2023 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

NCT01140360
View directory record Official record
Conditions
Neurofibromatosis, Neurofibromas
Interventions
Gleevec
Drug
Lead sponsor
Kent Robertson
Other
Eligibility
3 Years to 65 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 10:35 AM EDT
Terminated Phase 2 Interventional

Safety and Efficacy of OMS643762 in Subjects With Huntington's Disease

NCT02074410
View directory record Official record
Conditions
Huntington's Disease
Interventions
OMS643762, Placebo
Drug
Lead sponsor
Omeros Corporation
Industry
Eligibility
18 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
9
States / cities
San Diego, California • Englewood, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2018 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 4 Interventional

A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex

NCT05864846
View directory record Official record
Conditions
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
Interventions
Cannabidiol Oral Solution [Epidiolex]
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
12
States / cities
Aurora, Colorado • New Haven, Connecticut • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 15, 2026, 10:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Brain Perfusion in Pantothenate Kinase-associated Neurodegeneration (PKAN)

NCT01838018
View directory record Official record
Conditions
Pantothenate Kinase-associated Neurodegeneration (PKAN)
Interventions
Not listed
Lead sponsor
Susan J. Hayflick
Other
Eligibility
7 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 29, 2018 · Synced Oct 15, 2026, 10:35 AM EDT
Completed No phase listed Observational

Evaluation and Follow-up of Individuals With Obsessive-Compulsive Disorder and Related Conditions

NCT00004550
View directory record Official record
Conditions
Anorexia Nervosa, Obsessive Compulsive Disorder, Tourette Syndrome, Trichotillomania
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 10:35 AM EDT
Terminated Phase 1 Interventional

FAAH Inhibitor Trial for Adults With Tourette Syndrome

NCT02134080
View directory record Official record
Conditions
Tourette Syndrome
Interventions
PF-04457845, Placebo
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 60 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Oct 15, 2026, 10:35 AM EDT
Terminated Phase 1 Interventional

Study of ASN003 in Subjects With Advanced Solid Tumors

NCT02961283
View directory record Official record
Conditions
Neoplasms, Melanoma, Colorectal Neoplasm, Carcinoma, Non-small Cell Lung
Interventions
ASN003 ascending doses, ASN003 MTD
Drug
Lead sponsor
Asana BioSciences
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 9, 2023 · Synced Oct 15, 2026, 10:35 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 15, 2026, 10:35 AM EDT
Recruiting Not applicable Interventional

CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study

NCT06096675
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
• Cardiac Rehabilitation
Behavioral
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 1 Interventional

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

NCT04095273
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Oct 15, 2026, 10:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Reliability and Validity of the MINI International Neuropsychiatric Interview for Children and Adolescents (MINI-KID)

NCT00579267
View directory record Official record
Conditions
Major Depression, Mania, Anxiety Disorders, Psychotic Disorder, Alcohol Dependence, Drug Dependence, Eating Disorders, Suicidality, Dysthymia, ADHD, Tourettes Disorder, Conduct Disorder, Oppositional Defiant Disorder, Pervasive Developmental Disorder
Interventions
MINI-KID
Other
Lead sponsor
University of South Florida
Other
Eligibility
6 Years to 17 Years
Enrollment
230 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2008
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 16, 2009 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 1 Interventional

A Study To Assess The Tolerability And Clinical Activity Of Gedatolisib In Combination With Palbociclib/Letrozole Or Palbociclib/Fulvestrant In Women With Metastatic Breast Cancer

NCT02684032
View directory record Official record
Conditions
Breast Cancer
Interventions
Gedatolisib, Palbociclib, Letrozole, Fulvestrant
Drug
Lead sponsor
Celcuity Inc
Industry
Eligibility
18 Years and older · Female only
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
41
States / cities
Birmingham, Alabama • Corona, California • Fountain Valley, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2022 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 1Phase 2 Interventional

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

NCT01369901
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
A functional exercise program, A stretching exercise program
Behavioral
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 90 Years · Male only
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 3 Interventional

TREND-HD - A Trial of Ethyl-EPA (Miraxion™) in Treating Mild to Moderate Huntington's Disease

NCT00146211
View directory record Official record
Conditions
Huntington Disease
Interventions
Ethyl-EPA (Miraxion™)
Drug
Lead sponsor
Amarin Neuroscience Ltd
Industry
Eligibility
35 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
35
States / cities
Birmingham, Alabama • Scottsdale, Arizona • La Jolla, California + 31 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2007 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resiliency Training for Adolescent Neurofibromatosis Patients Via Videoconferencing With Skype

NCT02387177
View directory record Official record
Conditions
Neurofibromatoses
Interventions
Stress Management Group 1, Stress Management Group 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
12 Years to 17 Years
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 17, 2018 · Synced Oct 15, 2026, 10:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Nicotine Treatment of Cognitive Decline in Down Syndrome

NCT01778946
View directory record Official record
Conditions
Down Syndrome, Mild Cognitive Impairment
Interventions
Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg)
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
25 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated May 10, 2022 · Synced Oct 15, 2026, 10:35 AM EDT
Recruiting Not applicable Interventional

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

NCT00912041
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophy
Interventions
Placement of the BrainGate2 sensor(s) into the motor-related cortex
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2038
U.S. locations
6
States / cities
Sacramento, California • Stanford, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 15, 2026, 10:35 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Extracellular RNA Biomarkers of Myotonic Dystrophy

NCT05020002
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
215 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 15, 2026, 10:35 AM EDT