Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 14, 2026, 11:21 PM EDT

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

Showing 145–168 of 1,146 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

NCT04007991
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 14, 2026, 11:21 PM 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 14, 2026, 11:21 PM EDT
Completed Phase 1 Interventional

Developing Cognitive Training for Tourette Syndrome

NCT01440023
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorders
Interventions
Comprehensive Behavioral Intervention for Tics (CBIT), Computerized Response Inhibition Training, Computerized Placebo Cognitive Training
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
9 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

NCT03325010
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Valbenazine, Placebo oral capsule
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 17 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
34
States / cities
Sun City, Arizona • Rogers, Arkansas • Anaheim, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 14, 2026, 11:21 PM EDT
Not yet recruiting Phase 1 Interventional

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

NCT07531940
View directory record Official record
Conditions
Down Syndrome, Intellectual Disability, Alzheimer Dementia (AD)
Interventions
Memantine
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years to 32 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 Brain Tumor Genetic Risk

NCT01707836
View directory record Official record
Conditions
Neurofibromatosis Type 1, Pediatric Brain Tumor
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 18 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 9, 2017 · Synced Oct 14, 2026, 11:21 PM EDT
Recruiting Not applicable Interventional

Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence

NCT04763109
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Whole-body Magnetic Resonance Imaging
Other
Lead sponsor
Nicole Baca
Other
Eligibility
5 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Completed No phase listed Observational

Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II Patients

NCT01222728
View directory record Official record
Conditions
Neoplasms, Nervous System Disease, Vestibular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 14, 2026, 11:21 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Terminated No phase listed Observational

Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products

NCT03886753
View directory record Official record
Conditions
ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle, Dyskinetic Syndrome, Epilepsy, Glaucoma, Huntington Disease, Inflammatory Bowel Disease (IBD), Multiple Sclerosis, Neuropathy, Opioid Use, Parkinson Disease, HIV/AIDS, Ptsd, Intractable Pain, Sickle Cell Disease, Terminal Illness
Interventions
Registry, PK microsampling of blood
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 14, 2026, 11:21 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Tracking Neurodegeneration in Early Wolfram Syndrome

NCT02455414
View directory record Official record
Conditions
Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day and older
Enrollment
101 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190, Intracerebroventricular (ICV) access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 16 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 24, 2022 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2021 · Synced Oct 14, 2026, 11:21 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

TANDem-2: Closing the Gap to Interventions for TAND

NCT06879665
View directory record Official record
Conditions
Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC)
Interventions
Well-Beans for Caregivers Program
Behavioral
Lead sponsor
Vrije Universiteit Brussel
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Oct 14, 2026, 11:21 PM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

NCT07630389
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Recruiting No phase listed Observational

A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

NCT06628687
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Morrisville, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Completed No phase listed Observational

Apathy in Tourette Syndrome and Changes in Quality of Life

NCT02765178
View directory record Official record
Conditions
Tourette Syndrome, Apathy
Interventions
Children's Motivation Scale, Yale Global Tic Severity Scale (YGTSS), Studies Depression Scale for Children (CES-DC), Gilles de la Tourette Syndrome Quality Of Life scale, Demographic Data
Other
Lead sponsor
University of Florida
Other
Eligibility
12 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 6, 2017 · Synced Oct 14, 2026, 11:21 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Doxycycline in Cutaneous Schwannoma (NF2)

NCT05521048
View directory record Official record
Conditions
Neurofibromatosis Type 2
Interventions
Doxycycline Injection [Doxy]
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
8 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 14, 2026, 11:21 PM EDT
Completed No phase listed Observational

Tuberous Sclerosis Complex Natural History Study: Renal Manifestations

NCT00598455
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
7 Years to 65 Years
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 4, 2017 · Synced Oct 14, 2026, 11:21 PM 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 Sep 24, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants

NCT03649165
View directory record Official record
Conditions
Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Healthy Participants
Interventions
Treatment A, Treatment B, Treatment C, Treatment D, Acetaminophen
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 8, 2018 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 2 Interventional Results available

An Open Label Study of ANX005 in Participants With, or at Risk for, Manifest Huntington's Disease

NCT04514367
View directory record Official record
Conditions
Huntington Disease
Interventions
ANX005
Drug
Lead sponsor
Annexon, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
6
States / cities
Birmingham, Alabama • Englewood, Colorado • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 14, 2026, 11:21 PM EDT
Completed Phase 1 Interventional

A Phase I Study of BKM120 and Everolimus in Advanced Solid Malignancies

NCT01470209
View directory record Official record
Conditions
Solid Tumors, Lung Cancer
Interventions
BKM120, Everolimus
Drug
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Mar 19, 2018 · Synced Oct 14, 2026, 11:21 PM EDT