Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 10, 2026, 4:42 PM EDT

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Showing 1–24 of 1,262 matching trials from the live ClinicalTrials.gov search.
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Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, Spinocerebellar Ataxia Type 17, ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1
Interventions
CAD-1883, Placebos
Drug
Lead sponsor
Cadent Therapeutics
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Aurora, Colorado + 3 more
Conditions
Huntington's Disease
Interventions
Lithium, Divalproex
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Terminated Phase 3 Interventional Results available

Tanezumab In Osteoarthritis Of The Hip Or Knee

NCT00985621
Conditions
Osteoarthritis
Interventions
tanezumab 10 mg, tanezumab 5 mg, oxycodone, placebo
Biological · Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
614 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
66
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 54 more
Conditions
Irritable Mood, Huntington's Disease
Interventions
SRX246, Placebo
Drug
Lead sponsor
Azevan Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
22
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 19 more
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Dichloroacetate (DCA), Placebo, Genotype
Drug · Other · Genetic
Lead sponsor
Saol Therapeutics Inc
Industry
Eligibility
6 Months to 17 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
10
States / cities
Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more
Conditions
Congenital Myotonic Dystrophy, CDM
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
Up to 59 Months
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
Los Angeles, California • Fairway, Kansas • Rochester, New York + 1 more
Conditions
Neurofibromatosis Type 1, Legius Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
313 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Not yet recruiting Phase 1Phase 2 Interventional

Gene Therapy Trial for CLN6 Batten Disease

NCT07582484
Conditions
CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
Interventions
scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
Drug
Lead sponsor
The Charlotte and Gwenyth Gray Foundation
Other
Eligibility
4 Months and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
La Jolla, California
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Conditions
Tourette Syndrome
Interventions
NBI-98854, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 64 Years
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
30
States / cities
Long Beach, California • National City, California • San Bernardino, California + 27 more
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
Not listed
Lead sponsor
Novartis
Industry
Eligibility
Up to 14 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
East Hanover, New Jersey
Terminated No phase listed Observational

MPS II Immunophenotyping

NCT04976231
Conditions
MPS II, Mucopolysaccharidosis II, Hunter Syndrome
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Myotonic Dystrophy
Interventions
SomatoKine/IPLEX
Drug
Lead sponsor
University of Rochester
Other
Eligibility
21 Years to 60 Years
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
1
States / cities
Rochester, New York
Conditions
Solid Tumors
Interventions
thioureidobutyronitrile
Drug
Lead sponsor
Cellceutix Corporation
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Giant Axonal Neuropathy, Gene Transfer
Interventions
scAAv9/JeT-GAN
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
3 Years to 99 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Endometriosis
Interventions
Tanezumab, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 49 Years · Female only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
30
States / cities
Mobile, Alabama • Tucson, Arizona • San Diego, California + 20 more
Conditions
Mild Cognitive Impairment (MCI), Alzheimer Disease (AD), Dementia With Lewy Bodies (DLB), Frontotemporal Lobar Degeneration (FTLD), Parkinsons Disease With Dementia (PDD), Transient Epileptic Amnesia (TEA), Temporal Lobe Epilepsy (TLE), Spinocerebellar Ataxias (SCA), HIV Associated Neurocognitive Disorder (HAND), Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS)
Interventions
Neuraceq
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Timeline
2019 – 2025
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Neurofibromatosis Type 1, Neurofibroma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
20 Years to 99 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Huntington Disease, Parkinson Disease
Interventions
MindTrails-Movement, Waitlist Control
Behavioral · Other
Lead sponsor
University of Virginia
Other
Eligibility
21 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Charlottesville, Virginia
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 1 more
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

NCT05854433
Conditions
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Interventions
Non-interventional study
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
30 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Winston-Salem, North Carolina