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ClinicalTrials.gov public records Last synced Oct 10, 2026, 1:31 PM EDT

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

Showing 73–96 of 1,145 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Enrolling by invitation No phase listed Observational

Neurofibromatosis Type 1 Tumor Early Detection Study

NCT06515860
View directory record Official record
Conditions
Neurofibromatosis Type 1, Neurofibromatosis 1, Plexiform Neurofibroma, Plexiform Neurofibromas, Malignant Peripheral Nerve Sheath Tumor, Malignant Peripheral Nerve Sheath Tumors, Atypical Neurofibroma
Interventions
Liquid biopsy for MPNST development
Diagnostic Test
Lead sponsor
David Miller
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Completed No phase listed Observational

Tourette Discrimination (TD) Stigma Scale

NCT05696769
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tic Disorder, Childhood, Tics
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
8 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 10, 2026, 1:31 PM EDT
Active, not recruiting Not applicable Interventional

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT06139172
View directory record Official record
Conditions
Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions
Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional Accepts healthy volunteers

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation. Master Protocol DIAN-TU-001

NCT01760005
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Gantenerumab, Solanezumab, Matching Placebo (Gantenerumab), Matching Placebo (Solanezumab), E2814, Lecanemab, Matching Placebo (E2814)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
490 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 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 10, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT00634270
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Sirolimus
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2017 · Synced Oct 10, 2026, 1:31 PM 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 10, 2026, 1:31 PM EDT
Recruiting No phase listed Observational

Glioma Developmental and HyperActive Ras Tumor (DHART) Board

NCT05489783
View directory record Official record
Conditions
Glioma
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 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 Aug 31, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Terminated Phase 2 Interventional Results available

SF1126 in Recurrent or Progressive SCCHN and Mutations in PIK3CA Gene and/or PI-3 Kinase Pathway Genes

NCT02644122
View directory record Official record
Conditions
Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma
Interventions
SF1126
Drug
Lead sponsor
Ezra Cohen
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 10, 2026, 1:31 PM EDT
Terminated Phase 1 Interventional

PF-07284892 in Participants With Advanced Solid Tumors

NCT04800822
View directory record Official record
Conditions
Solid Tumor
Interventions
PF-07284892, lorlatinib, binimetinib, cetuximab, encorafenib
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Detroit, Michigan + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Imaging Studies in Corticobasal Syndrome

NCT07000851
View directory record Official record
Conditions
Cortico Basal Degeneration, Corticobasal Syndrome, Corticobasal Syndrome(CBS), Corticobasal Degeneration, Corticobasal Degeneration (CBD), Corticobasal Syndrome (CBS)
Interventions
C-11 ER176 Radiotracer, C-11 PiB, AV1451 Tau
Diagnostic Test
Lead sponsor
Jennifer Whitwell
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2031
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Completed Not applicable Interventional Results available

Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

NCT01472874
View directory record Official record
Conditions
Wilson Disease
Interventions
Once a day Trientine
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 10, 2026, 1:31 PM EDT
Completed No phase listed Observational

Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

NCT00667836
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
3 Years to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Oct 10, 2026, 1:31 PM EDT
Completed Not applicable Interventional Results available

CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)

NCT04182763
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
CoA-Z, Placebo
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
3 Months to 89 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 10, 2026, 1:31 PM 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 10, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Efficacy of a Therapeutic Combination of Dronabinol and PEA for Tourette Syndrome

NCT03066193
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Dronabinol and Palmitoylethanolamide
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 60 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 24, 2020 · Synced Oct 10, 2026, 1:31 PM EDT
Completed Phase 3 Interventional Results available

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

NCT02623699
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Tofersen, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
20
States / cities
Phoenix, Arizona • La Jolla, California • San Francisco, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2023 · Synced Oct 10, 2026, 1:31 PM EDT
Enrolling by invitation Phase 2 Interventional

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

NCT06148311
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Matching placebo, Dexmedetomidine
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Withdrawn Phase 2 Interventional

A Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of SNC-102 in Subjects With Tourette Syndrome

NCT02217007
View directory record Official record
Conditions
Tourette Syndrome
Interventions
SNC-102 sustained release tablet
Drug
Lead sponsor
Synchroneuron Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Chicago, Illinois • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 10, 2026, 1:31 PM EDT
Recruiting No phase listed Observational

Gait and Bone Health in SMA

NCT07400198
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Jacqueline Montes
Other
Eligibility
8 Years to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 10, 2026, 1:31 PM 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 10, 2026, 1:31 PM EDT
Not listed No phase listed Observational

Prospective Single-Timepoint Huntington's Disease Biospecimen Collection Study

NCT05773196
View directory record Official record
Conditions
Huntington Disease, Huntington's Dementia, Huntington Disease, Late Onset, Huntington; Dementia (Etiology)
Interventions
Biospecimen Collection
Other
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
25 Years to 65 Years
Enrollment
5 participants
Timeline
2023 – 2025
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 10, 2026, 1:31 PM EDT
Completed No phase listed Observational

Measurement of Frataxin mRNA in Biofluids

NCT06496451
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Spinal tap for CSF collection, Venipuncture for blood sample
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 10, 2026, 1:31 PM EDT
Not listed No phase listed Observational

Development of Assessments for Later Stage HD

NCT04370470
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
20 Years and older
Enrollment
340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
9
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 6 more
Source: ClinicalTrials.gov public record
Updated May 1, 2020 · Synced Oct 10, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

NCT01406873
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Mexiletine, Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 19, 2018 · Synced Oct 10, 2026, 1:31 PM EDT