- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019
- U.S. locations
- 1
- States / cities
- Los Angeles, California
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 25–48
of 934
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fragile X Syndrome
- Interventions
- placebo, oxytocin 24IU, oxytocin 48IU
- Drug
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 13 Years to 29 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Chronic Granulomatous Disease, Inflammatory Bowel Disease
- Interventions
- Neocate Splash
- Other
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Muscular Dystrophy, Muscular Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 5 Years to 17 Years · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ketamine, Dexmedetomidine
- Drug
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- Male only
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Shriners Hospitals for Children
- Other
- Eligibility
- 4 Years to 21 Years · Male only
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Sacramento, California • Portland, Oregon
- Conditions
- Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
- Interventions
- No treatment given
- Other
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 12 Years and older · Male only
- Enrollment
- 231 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 29
- States / cities
- Los Angeles, California • Sacramento, California • Aurora, Colorado + 24 more
- Conditions
- Fabry Disease
- Interventions
- pegunigalsidase alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- AVI-4658 (Eteplirsen)
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 7 Years to 13 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 11
- States / cities
- Long Beach, California • Gainesville, Florida • Chicago, Illinois + 8 more
- Conditions
- Fabry Disease
- Interventions
- DRX005B
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fragile X Syndrome
- Interventions
- Aripiprazole
- Drug
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 5 Years to 35 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- delandistrogene moxeparvovec, Plasmapheresis
- Genetic · Procedure
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 8 Years · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
- Conditions
- Gene Therapy, Hemophilia B
- Interventions
- Interview
- Behavioral
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 150 participants
- Timeline
- 2022 – 2025
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Developmental Dysphasia, Epileptic Encephalopathy, Childhood-Onset, X-Linked Intellectual Disability
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 6 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
- Interventions
- Carbaglu, Placebo
- Drug
- Lead sponsor
- Mendel Tuchman
- Other
- Eligibility
- 1 Week to 99 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2020
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more
- Conditions
- Choroideremia
- Interventions
- BIIB111
- Drug
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 4
- States / cities
- Miami, Florida • Boston, Massachusetts • Cincinnati, Ohio + 1 more
- Conditions
- Cystic Fibrosis, Pseudohypoaldosteronism, Nontuberculous Mycobacterial Infection, Chronic Granulomatous Disease, Primary Ciliary Dyskinesia
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 65 Years
- Enrollment
- 87 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Month to 65 Years · Male only
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 5
- States / cities
- San Diego, California • Saint Paul, Minnesota • Philadelphia, Pennsylvania + 2 more
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- rAAV2tYF-GRK1-RPGR
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 6 Years to 50 Years · Male only
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 4
- States / cities
- Durham, North Carolina • Cincinnati, Ohio • Portland, Oregon + 1 more
- Conditions
- Fabry Disease
- Interventions
- Venglustat (GZ402671), Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 12
- States / cities
- Birmingham, Alabama • Los Angeles, California • Orange, California + 9 more
- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Rett Syndrome
- Interventions
- Bionetide, Placebo
- Drug
- Lead sponsor
- Biomed Industries, Inc.
- Industry
- Eligibility
- 5 Years to 20 Years · Female only
- Enrollment
- 210 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 14
- States / cities
- Phoenix, Arizona • La Jolla, California • Los Angeles, California + 11 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Idebenone
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 8 Years and older
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • New Haven, Connecticut • Chicago, Illinois + 9 more
- Conditions
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 18 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2013
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia • Charlotte, North Carolina