- Conditions
- Mitochondrial Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Months to 115 Years
- Enrollment
- 400 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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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.
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.
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- Conditions
- Dystonia
- Interventions
- Not listed
- Lead sponsor
- Emory University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 3,265 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2020
- U.S. locations
- 34
- States / cities
- Birmingham, Alabama • Scottsdale, Arizona • Fountain Valley, California + 26 more
- Conditions
- Depression, Depression, Bipolar
- Interventions
- Observational Study
- Other
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 10 Years and older
- Enrollment
- 2,500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2036
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Genetic Diseases
- Interventions
- Not listed
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Durham, North Carolina
- Conditions
- Amyotrophic Lateral Sclerosis Type 4, Inherited Neurological Disorders of RNA Processing
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 5 Years to 120 Years
- Enrollment
- 330 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- Conditions
- Mood Disorders, Sleep Disorders, Migraine
- Interventions
- Not listed
- Lead sponsor
- National Institute of Mental Health (NIMH)
- NIH
- Eligibility
- 7 Years to 120 Years
- Enrollment
- 1,944 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease, GSD4, GSD IV, APBD
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2035
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Methylmalonic Acidemia, Propionic Acidemia
- Interventions
- Registry
- Other
- Lead sponsor
- HemoShear Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
- Interventions
- Not listed
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- Up to 5 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Stanford, California • Chicago, Illinois + 11 more
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 18 Years to 120 Years · Male only
- Enrollment
- 70 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Choroideremia
- Interventions
- Observation
- Other
- Lead sponsor
- 4D Molecular Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 3
- States / cities
- Dallas, Texas • Houston, Texas • Salt Lake City, Utah
- Conditions
- Recurrent Bacterial Infection, Autoimmune Disorder
- Interventions
- Natural History Observation
- Other
- Lead sponsor
- Catalyst Biosciences
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Huntsville, Alabama
- Conditions
- Head and Neck Neoplasms, Hearing Disorder, Hyperlipidemia
- Interventions
- Not listed
- Lead sponsor
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland
- Conditions
- Danon Disease
- Interventions
- No intervention
- Other
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • La Jolla, California • Aurora, Colorado + 6 more
- Conditions
- Alpha1-Antitrypsin Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • Charleston, South Carolina • Nashville, Tennessee
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency, Ceramidase Deficiency, N-Laurylsphingosine Deacylase Deficiency, ASAH1 Mutation
- Interventions
- Not listed
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 2
- States / cities
- Oakland, California • Washington D.C., District of Columbia
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM), Distal Myopathy With Rimmed Vacuoles (DMRV)
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 319 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Influenza
- Interventions
- Not listed
- Lead sponsor
- Henry M. Jackson Foundation for the Advancement of Military Medicine
- Other
- Eligibility
- 17 Years to 55 Years
- Enrollment
- 2,018 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2023
- U.S. locations
- 5
- States / cities
- San Diego, California • Bethesda, Maryland • San Antonio, Texas + 2 more
- Conditions
- Cherubism
- Interventions
- no interventions
- Other
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Cystic Fibrosis
- Interventions
- Not listed
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- Up to 6 Months
- Enrollment
- 79 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 22
- States / cities
- St. Petersburg, Florida • Boise, Idaho • Chicago, Illinois + 19 more
- Conditions
- COVID-19
- Interventions
- Not listed
- Lead sponsor
- National Institute on Aging (NIA)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Sanfilippo Syndrome Type C
- Interventions
- Not listed
- Lead sponsor
- Phoenix Nest
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Chronic Pain, Fatigue, Substance Use Disorders
- Interventions
- Not listed
- Lead sponsor
- National Institute on Drug Abuse (NIDA)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 396 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland