Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 17, 2026, 3:00 AM EDT

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Showing 337–360 of 1,147 matching trials from the live ClinicalTrials.gov search.
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
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
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Cardiac Amyloidosis
Interventions
18F-Florbetaben (Neuraceq®) PET/MRI
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Stanford, California
Conditions
Epilepsy, Tuberous Sclerosis Complex
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
2
States / cities
Cincinnati, Ohio • Houston, Texas
Active, not recruiting Phase 3 Interventional

Open Pilot Trial of BHV-4157

NCT03408080
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia 3, Spinocerebellar Ataxia Type 6, MSA-C
Interventions
BHV-4157
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Giant Axonal Neuropathy (GAN)
Interventions
scAAV9/JeT-GAN
Genetic
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
10 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Dallas, Texas
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
Conditions
Friedreich Ataxia, Neuro-Degenerative Disease
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 80 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2030
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
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
Conditions
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Interventions
Emavusertib, Venetoclax
Drug
Lead sponsor
Curis, Inc.
Industry
Eligibility
18 Years and older
Enrollment
366 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
12
States / cities
Tampa, Florida • Atlanta, Georgia • Chicago, Illinois + 8 more
Conditions
Small-cell Lung Cancer
Interventions
Berzosertib, Topotecan
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Santa Rosa, California • Topeka, Kansas • Bethesda, Maryland + 8 more
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)
Interventions
alpelisib
Other
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Ryanodine Receptor 1-Related Myopathy, Ryanodine Receptor 1 Related Disorders
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
7 Years to 100 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Huntington's Disease
Interventions
Creatine
Drug
Lead sponsor
National Center for Complementary and Integrative Health (NCCIH)
NIH
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
4
States / cities
Boston, Massachusetts • Long Island City, New York • New York, New York + 1 more
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
15
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 12 more
Conditions
Advanced Solid Tumors
Interventions
BMS-986484, Nivolumab, Oxaliplatin, Capecitabine, Fluorouracil, Calcium folinate
Drug · Biological
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Tucson, Arizona • Los Angeles, California • Grand Rapids, Michigan + 2 more
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Completed No phase listed Observational Accepts healthy volunteers

Study of Electrical Impedance Myography (EIM) in ALS

NCT02011204
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
Interventions
Electrical impedance myography (EIM)
Device
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
35 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Boston, Massachusetts + 2 more
Conditions
Neurofibromatosis 1
Interventions
Lovastatin, placebo pill
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
10 Years to 50 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Huntington's Disease
Interventions
20 mg BID of PF-02545920
Drug
Lead sponsor
Pfizer
Industry
Eligibility
30 Years to 66 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
17
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Irvine, California + 9 more
Conditions
Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease, Lip and Oral Cavity Carcinoma, Lymphangioleiomyomatosis, Malignant Endocrine Neoplasm, Malignant Female Reproductive System Neoplasm, Malignant Male Reproductive System Neoplasm, Malignant Neoplasm, Malignant Respiratory Tract Neoplasm, Malignant Thoracic Neoplasm, Malignant Urinary System Neoplasm, Mesothelial Neoplasm, Metastatic Malignant Neoplasm, Metastatic Urothelial Carcinoma, Neurofibromatosis Type 2, Recurrent Adult Soft Tissue Sarcoma, Recurrent Breast Carcinoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Digestive System Carcinoma, Recurrent Female Reproductive System Carcinoma, Recurrent Male Reproductive System Carcinoma, Recurrent Malignant Neoplasm, Recurrent Pharyngeal Carcinoma, Recurrent Thyroid Gland Carcinoma, Refractory Malignant Neoplasm, Soft Tissue Neoplasm, Stage III Breast Cancer AJCC v7, Stage III Pharyngeal Cancer, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Pharyngeal Cancer, Stage IVA Pharyngeal Cancer, Stage IVB Pharyngeal Cancer, Stage IVC Pharyngeal Cancer, Thyroid Gland Neoplasm
Interventions
Bevacizumab, Cetuximab, Laboratory Biomarker Analysis, Pharmacological Study, Temsirolimus, Valproic Acid
Biological · Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Houston, Texas
Terminated No phase listed Observational Accepts healthy volunteers

Biomechanics of Optic Neuropathy

NCT02982499
Conditions
Optic Neuropathy
Interventions
magnetic resonance image (MRI), Optical Coherence Tomography (OCT)
Device
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Miami, Florida
Conditions
Cancer, Cardiovascular Disease, Neurologic Dysfunction, Congenital Abnormalities, Hearing Loss
Interventions
Experimental
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chapel Hill, North Carolina