- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
- Interventions
- Hematopoietic Stem Cell Transplant
- Biological
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 4 Months to 45 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
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.
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Showing 1–24
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- Conditions
- Hemophilia A
- Interventions
- SPK-8011
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 11
- States / cities
- Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 more
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Jerry Vockley, MD, PhD
- Other
- Eligibility
- 1 Month and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Fragile X Syndrome
- Interventions
- Baclofen, Memantine, Roflumilast, Placebo
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Interventions
- FLT190
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 4 Years and older · Male only
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 8
- States / cities
- Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Edasalonexent, Placebo
- Drug
- Lead sponsor
- Catabasis Pharmaceuticals
- Industry
- Eligibility
- 4 Years to 7 Years · Male only
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Orlando, Florida + 2 more
- Conditions
- Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
- Interventions
- Not listed
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2031
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- PF-06939926, Placebo
- Genetic · Other
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 4 Years to 7 Years · Male only
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2039
- U.S. locations
- 17
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
- Conditions
- Autism Spectrum Disorder, Fragile X Syndrome
- Interventions
- Mismatch negativity
- Behavioral
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 15 Years to 35 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
- Interventions
- ALD-101
- Biological
- Lead sponsor
- Aldagen
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 3
- States / cities
- Los Angeles, California • New York, New York • Durham, North Carolina
- Conditions
- Hemophilia B
- Interventions
- HEMGENIX, Factor IX (FIX)
- Genetic · Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2043
- U.S. locations
- 1
- States / cities
- Rochester, New York
- 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
- Genetic Disorder, Asperger Disorder, Autism Spectrum Disorder, Fragile X Syndrome, Developmental Delay
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Year to 99 Years
- Enrollment
- 96 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- OTC Deficiency
- Interventions
- DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2031
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl 150 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 5
- States / cities
- Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, agalsidase
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Prednisone
- Drug
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 4 Years to 10 Years · Male only
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2008
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Mucopolysaccharidosis II, MPS II
- Interventions
- SB-913
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Fabry Disease
- Interventions
- migalastat
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Wiskott-Aldrich Syndrome
- Interventions
- OTL-103
- Genetic
- Lead sponsor
- Fondazione Telethon
- Other
- Eligibility
- Up to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Behavioral Manifestations of Lesch-Nyhan Disease
- Interventions
- sapropterin
- Drug
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Rett Syndrome
- Interventions
- Drug: Bionetide, Placebo
- Drug
- Lead sponsor
- Biomed Industries, Inc.
- Industry
- Eligibility
- 5 Years to 20 Years · Female only
- Enrollment
- 187 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 12
- States / cities
- La Jolla, California • Los Angeles, California • Sacramento, California + 9 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- Not listed
- Lead sponsor
- MeiraGTx UK II Ltd
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 140 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 6
- States / cities
- La Jolla, California • Stanford, California • Atlanta, Georgia + 3 more