- Conditions
- Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
- Interventions
- anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
- Drug · Procedure
- Lead sponsor
- Fairview University Medical Center
- Other
- Eligibility
- 0 Years to 55 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
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 995
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- fitusiran, factor concentrates
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 12 Years and older · Male only
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 8
- States / cities
- Little Rock, Arkansas • Gainesville, Florida • Tampa, Florida + 5 more
- Conditions
- Fragile X Syndrome
- Interventions
- Gaboxadol, Placebo
- Drug
- Lead sponsor
- Craig Erickson
- Other
- Eligibility
- 18 Years to 40 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- Other: Assessments
- Other
- Lead sponsor
- NightstaRx Ltd, a Biogen Company
- Other
- Eligibility
- 7 Years and older
- Enrollment
- 201 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Los Angeles, California • San Francisco, California + 7 more
- Conditions
- Fabry Disease
- Interventions
- AT1001 150 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 4
- States / cities
- Costa Mesa, California • Miami, Florida • Orlando, Florida
- Conditions
- Choroideremia, CHM (Choroideremia) Gene Mutations
- Interventions
- AAV2-hCHM
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Philadelphia, Pennsylvania
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- exercise training
- Behavioral
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 21 Years to 70 Years · Female only
- Enrollment
- 31 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP, XLPP, X-Linked Dominant Erythropoietic Protoporphyria, XLEPP, XLDP
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- 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
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Optical Coherence Tomography (OCT) of the Retina
- Interventions
- OCT imaging
- Device
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- Muscular Dystrophy, Duchenne, Ambulatory Care
- Interventions
- Placebo, Idebenone
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 10 Years to 18 Years · Male only
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 9
- States / cities
- Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
- Conditions
- Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
- Interventions
- VK0214, Placebo
- Drug · Other
- Lead sponsor
- Viking Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Gainesville, Florida • Baltimore, Maryland + 1 more
- Conditions
- Fragile X Syndrome
- Interventions
- Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose), RO4917523
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 5
- States / cities
- Sacramento, California • Decatur, Georgia • Chicago, Illinois + 2 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
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- SAT-3247
- Drug
- Lead sponsor
- Satellos Bioscience, Inc.
- Industry
- Eligibility
- 16 Years to 40 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 4
- States / cities
- Los Angeles, California • St Louis, Missouri • Columbus, Ohio + 1 more
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hemophilia B, Hemophilia B, Moderately Severe or Severe
- Interventions
- BE-101
- Drug
- Lead sponsor
- Be Biopharma
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 5
- States / cities
- Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 3
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York
- Conditions
- Hemophilia B, Allergic Reactions
- Interventions
- Not listed
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
- Industry
- Eligibility
- Not listed
- Enrollment
- 166 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Orange, California • Atlanta, Georgia + 12 more
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 6 Years and older · Male only
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 9
- States / cities
- Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more
- Conditions
- Brain Disorders, Aicardi Syndrome
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Not listed
- Enrollment
- 2,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2027
- U.S. locations
- 1
- States / cities
- San Francisco, California