- 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
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 come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 118
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Observational study
- Other
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 269 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 3
- States / cities
- Sacramento, California • Cincinnati, Ohio • Columbus, Ohio
- 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
- Sickle Cell Disease
- Interventions
- Not listed
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Interventions
- Natural History Observation
- Other
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 4
- States / cities
- Stanford, California • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 6
- States / cities
- Palo Alto, California • Aurora, Colorado • Boston, Massachusetts + 3 more
- 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
- Homocystinuria Due to CBS Deficiency
- Interventions
- Not listed
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 1 Year to 65 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 7
- States / cities
- Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
- Conditions
- Stargardt Disease
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 259 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 6
- States / cities
- Baltimore, Maryland • Cleveland, Ohio • Philadelphia, Pennsylvania + 2 more
- Conditions
- KSHV Inflammatory Cytokine Syndrome (KICS), KSHV, HHV-8
- Interventions
- Zidovudine, Liposomal Doxorubicin, Valganiclovir, Rituximab, Standard Therapies, Cohort 1
- Drug · Other
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 140 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2008
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Cambridge, Massachusetts
- Conditions
- Primary Hyperoxaluria Type 3
- Interventions
- Not listed
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • New York, New York
- 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
- 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
- Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Neurologic Manifestations, Neurologic Disorder, Neurodevelopmental Disorders, Nervous System Diseases
- Interventions
- Not listed
- Lead sponsor
- International Rett Syndrome Foundation
- Other
- Eligibility
- 0 Years to 99 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more
- Conditions
- HIV Infections
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2033
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- COVID-19
- Interventions
- Not listed
- Lead sponsor
- National Institute on Aging (NIA)
- NIH
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 78 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Sanfilippo Syndrome Type B
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Fibrodysplasia Ossificans Progressiva (FOP)
- Interventions
- Not listed
- Lead sponsor
- The International FOP Association
- Other
- Eligibility
- Not listed
- Enrollment
- 800 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2040
- U.S. locations
- 1
- States / cities
- North Kansas City, Missouri
- Conditions
- Pyruvate Dehydrogenase Complex Deficiency Disease
- Interventions
- No intervention
- Other
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2030
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Leigh Syndrome
- Interventions
- None (Observational study)
- Other
- Lead sponsor
- Taysha Gene Therapies, Inc.
- Industry
- Eligibility
- Up to 18 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Post Influenza
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fucosidosis
- Interventions
- Not listed
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 2
- States / cities
- Orange, California • Minneapolis, Minnesota
- Conditions
- SMN Complex Proteins, GEMIN5 Protein, Human, Neurodevelopmental Disorders
- Interventions
- GEMIN5-Related Neurodevelopmental Disorder
- Other
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2050
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania