- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- delandistrogene moxeparvovec, placebo
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 148 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 14
- States / cities
- Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
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 73–96
of 934
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- PF-06741086
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 1 Year to 74 Years · Male only
- Enrollment
- 245 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2030
- U.S. locations
- 4
- States / cities
- Iowa City, Iowa • New Hyde Park, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 8 Years to 18 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia • Pittsburgh, Pennsylvania
- Conditions
- Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder), Cerebral Palsy (CP), Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment, Cerebral Palsy Infantile, Cerebral Palsy Spastic Hemiplegic, Cerebral Palsy, Dyskinetic, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE), Traumatic Brain Injury (TBI), Sensory Processing Disorder, Chromosomal Abnormalities, Genetic Disorders, Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS), RETT Syndrome With Proven MECP2 Mutation, Williams Syndrome, 22q11.2 Deletion Syndrome, Sensorimotor Integration
- Interventions
- Not listed
- Lead sponsor
- Healing Hope International
- Other
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2036
- U.S. locations
- 1
- States / cities
- The Woodlands, Texas
- Conditions
- Fabry Disease
- Interventions
- Replagal (agalsidase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
- Conditions
- Menkes Disease
- Interventions
- copper histidinate
- Drug
- Lead sponsor
- Sentynl Therapeutics, Inc.
- Industry
- Eligibility
- 0 Years to 6 Years
- U.S. locations
- 20
- States / cities
- Phoenix, Arizona • Sacramento, California • San Diego, California + 17 more
- Conditions
- Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
- Interventions
- Not listed
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Oakland, California • San Diego, California + 8 more
- Conditions
- Fragile X Syndrome
- Interventions
- Placebo, RO4917523
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 5 Years to 13 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2014
- U.S. locations
- 20
- States / cities
- Long Beach, California • Sacramento, California • San Diego, California + 17 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
- Drug
- Lead sponsor
- ReveraGen BioPharma, Inc.
- Industry
- Eligibility
- 4 Years to 7 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 6
- States / cities
- Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- Conditions
- Becker Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 4 Years and older · Male only
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 13
- States / cities
- Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 10 more
- Conditions
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Akron, Ohio
- Conditions
- Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
- Interventions
- Dried blood spot test for MPS
- Other
- Lead sponsor
- National MPS Society
- Other
- Eligibility
- 6 Months to 18 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 2
- States / cities
- New Brunswick, New Jersey • New York, New York
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Interventions
- Human Placental Derived Stem Cell
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 0 Years to 55 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2022
- U.S. locations
- 3
- States / cities
- Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
- Conditions
- Rett Syndrome
- Interventions
- TSHA-102
- Genetic
- Lead sponsor
- Taysha Gene Therapies, Inc.
- Industry
- Eligibility
- 2 Years to 3 Years · Female only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 3
- States / cities
- Birmingham, Alabama • Chicago, Illinois • Boston, Massachusetts
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Eplerenone, Spironolactone
- Drug
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 7 Years and older · Male only
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Aurora, Colorado • Kansas City, Kansas + 3 more
- Conditions
- Adrenoleukodystrophy, Restless Legs Syndrome
- Interventions
- Pramipexole, Placebo
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 75 Years · Female only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Pelizaeus-Merzbacher Disease
- Interventions
- HuCNS-SC cells
- Biological
- Lead sponsor
- StemCells, Inc.
- Industry
- Eligibility
- 6 Months to 5 Years · Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Chronic Granulomatous Disease
- Interventions
- Sirolimus, Donor peripheral blood stem cells., Cyclophosphamide post transplant, Total body 200cGy, Cyclophosphamide, Fludarabine, Busulfan
- Drug · Biological · Radiation
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 2 Years to 65 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hemophilia B
- Interventions
- PF-06838435/ fidanacogene elaparvovec
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2031
- U.S. locations
- 17
- States / cities
- San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more
- Conditions
- Duchenne Muscular Dystrophy, Exon 44
- Interventions
- AOC 1044, Placebo
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 7 Years to 55 Years · Male only
- Enrollment
- 70 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 10
- States / cities
- Little Rock, Arkansas • La Jolla, California • Palo Alto, California + 7 more
- Conditions
- Fabry Disease
- Interventions
- This is a non-interventional study
- Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Eagan, Minnesota
- Conditions
- Fabry Disease, Proteinuria
- Interventions
- Not listed
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 14 Years to 95 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 1
- States / cities
- Birmingham, Alabama
- Conditions
- Hemophilia B with Inhibitor, Hemophilia B
- Interventions
- SerpinPC
- Drug
- Lead sponsor
- ApcinteX Ltd
- Industry
- Eligibility
- 12 Years to 65 Years · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 3
- States / cities
- Aurora, Colorado • Tampa, Florida • Greenville, North Carolina