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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 25, 2026, 1:38 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Heredodegenerative Disorders, Nervous System Clear all
Completed Phase 3 Interventional Results available

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

NCT00790400
View directory record Official record
Conditions
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Interventions
Everolimus (RAD001), Everolimus Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
6
States / cities
Birmingham, Alabama • Phoenix, Arizona • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2017 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Phase 2 Interventional Results available

Risperidone for the Treatment of Huntington's Disease Involuntary Movements

NCT04201834
View directory record Official record
Conditions
Huntington Disease, Chorea
Interventions
Risperidone, BioStamp nPoint device
Drug · Device
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Sep 25, 2026, 1:38 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

NCT02269865
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Phase 1 Interventional

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

NCT02211768
View directory record Official record
Conditions
Neurofibromatosis, MPNST
Interventions
MRI, FDG-PET/CT scans, [18F]-FLT-PET/CT scans
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
10 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Terminated Not applicable Interventional

Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

NCT02584413
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Magnetic resonance imaging, Gadolinium contrast
Device · Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Year to 7 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 2, 2019 · Synced Sep 25, 2026, 1:38 PM 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 12, 2026 · Synced Sep 25, 2026, 1:38 PM 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 19, 2019 · Synced Sep 25, 2026, 1:38 PM EDT
Completed No phase listed Observational

Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I

NCT00684398
View directory record Official record
Conditions
Neurofibromatosis Type 1, Von Recklinghausen Disease, NF1
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 15, 2019 · Synced Sep 25, 2026, 1:38 PM EDT
Recruiting No phase listed Observational

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

NCT06075693
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
88 participants
Timeline
2022 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Sep 25, 2026, 1:38 PM 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 20, 2025 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Phase 3 Interventional Results available

RESCUE and REVERSE Long-term Follow-up

NCT03406104
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Sham Intravitreal Injection
Genetic · Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up on Menkes Disease Patients

NCT04337684
View directory record Official record
Conditions
Menkes Disease
Interventions
Long Term Follow-Up
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
Not listed
Enrollment
50 participants
Timeline
2019 – 2026
U.S. locations
1
States / cities
Solana Beach, California
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Not applicable Interventional

Respiratory Training in Friedreich's Ataxia

NCT06539598
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Respiratory Strength Training (RST)
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Neurobiology and Treatment of Reading Disability in NF-1

NCT00624234
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced Sep 25, 2026, 1:38 PM EDT
Terminated No phase listed Observational

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

NCT02204904
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Interventions
Allo-HSCT
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
6
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated May 20, 2020 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Phase 2 Interventional Results available

A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome

NCT03569631
View directory record Official record
Conditions
Fragile X Syndrome, FXS, Fra(X) Syndrome
Interventions
BPN14770, Placebo
Drug
Lead sponsor
Tetra Discovery Partners
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Sep 25, 2026, 1:38 PM EDT
Not yet recruiting Phase 2 Interventional

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

NCT07521657
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1)
Interventions
mirdametinib with radiation, mirdametinib
Combination Product · Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
12 Years to 89 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Phase 1 Interventional

Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT06132165
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser, 755nm Alexandrite Laser
Drug · Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Sep 25, 2026, 1:38 PM EDT
Withdrawn Phase 2Phase 3 Interventional

Trial of Kuvan in Lesch-Nyhan Disease

NCT00935753
View directory record Official record
Conditions
Behavioral Manifestations of Lesch-Nyhan Disease
Interventions
sapropterin
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 1, 2019 · Synced Sep 25, 2026, 1:38 PM EDT
Recruiting Phase 2 Interventional

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

NCT05010031
View directory record Official record
Conditions
Metastatic Cancer
Interventions
Palliative radiotherapy
Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Suspended Not applicable Interventional

Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)

NCT06934928
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1), Neurofibromatosis Type I
Interventions
GentleMax Pro (with skin cooling), GentleMax Pro (without skin cooling)
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Child to Adult Neurodevelopment in Gene Expanded Huntington's Disease

NCT01860339
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
Peggy C Nopoulos
Other
Eligibility
6 Years to 30 Years
Enrollment
488 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2026
U.S. locations
6
States / cities
Sacramento, California • Iowa City, Iowa • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Sep 25, 2026, 1:38 PM EDT
Completed Not applicable Interventional

Behavioral Therapy for Tourette Syndrome

NCT03019731
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Therapist-directed Behavioral Therapy, Home-based DVD Therapy
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
7 Years to 13 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2021 · Synced Sep 25, 2026, 1:38 PM EDT
Terminated Phase 1 Interventional

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

NCT05160558
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
BIIB132, BIIB132-Matching Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2023 · Synced Sep 25, 2026, 1:38 PM EDT