- Conditions
- X-Linked Adrenoleukodystrophy
- Interventions
- Not listed
- Lead sponsor
- NeuroVia, Inc.
- Industry
- Eligibility
- 2 Years to 13 Years · Male only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
Search Results
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Congenital Bleeding Disorder, Haemophilia B
- Interventions
- nonacog beta pegol
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 13 Years to 70 Years · Male only
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 7
- States / cities
- Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- (+)- Epicatechin
- Drug
- Lead sponsor
- Craig McDonald, MD
- Other
- Eligibility
- 8 Years to 17 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Sacramento, California
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
- Interventions
- SPK-8016
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Peoria, Illinois • Ann Arbor, Michigan + 7 more
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Interventions
- Ataluren, Chronic Corticosteroid Therapy
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years and older · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 5
- States / cities
- Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 2 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 1 Day to 90 Years
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
- Conditions
- X-linked Hypophosphatemia
- Interventions
- burosumab
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Fragile X Syndrome
- Interventions
- Metformin, Placebo oral tablet
- Drug
- Lead sponsor
- Rowan University
- Other
- Eligibility
- 18 Years to 50 Years · Male only
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Stratford, New Jersey
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle, Echocardiogram
- Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 5 Years to 15 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Interventions
- Lenti-D
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Not listed
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- Female only
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Male only
- Enrollment
- 270 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2026
- U.S. locations
- 29
- States / cities
- Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 25 more
- Conditions
- Fabry Disease
- Interventions
- PRX-102
- Drug
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 9
- States / cities
- Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
- Conditions
- Rett Syndrome, RTT
- Interventions
- GWP42003-P, Placebo
- Drug
- Lead sponsor
- Jazz Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • San Diego, California • Aurora, Colorado + 11 more
- Conditions
- IPEX
- Interventions
- CD4^LVFOXP3
- Biological
- Lead sponsor
- Bacchetta, Rosa, MD
- Other
- Eligibility
- 4 Months to 35 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2037
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyperimmunoglobulin M Syndrome
- Interventions
- Subgam
- Biological
- Lead sponsor
- Bio Products Laboratory
- Other
- Eligibility
- 2 Years to 75 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 16
- States / cities
- Chandler, Arizona • Irvine, California • San Diego, California + 12 more
- 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
- Pelizaeus-Merzbacher Disease, PMD
- Interventions
- HuCNS-SC transplant in the lead-in phase
- Biological
- Lead sponsor
- StemCells, Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, PLACEBO
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 360 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
- Conditions
- Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies, Idiopathic Generalised Epilepsy, Familial Dementia, X-linked Mental Retardation
- Interventions
- Not listed
- Lead sponsor
- State University of New York - Upstate Medical University
- Other
- Eligibility
- 6 Months and older
- Enrollment
- 176 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2015
- U.S. locations
- 1
- States / cities
- Syracuse, New York
- Conditions
- Fabry Disease
- Interventions
- AMT-191
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years to 50 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Interventions
- L-arginine
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 7 Years to 11 Years · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- X-linked Hypophosphatemia
- Interventions
- Placebo, KRN23
- Drug
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 4
- States / cities
- New Haven, Connecticut • Indianapolis, Indiana • Durham, North Carolina + 1 more
- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Interventions
- Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
- Drug · Radiation · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 0 Years to 70 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota