- Conditions
- Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
- Interventions
- rhASM
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
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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 25–36
of 12
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Adverse Drug Events, Adverse Drug Reactions, Drug Interaction Potentiation, Drug Metabolism, Poor, CYP2D6-RELATED, Drug Metabolism, Poor, CYP2C19-RELATED, Cytochrome P450 Enzyme Deficiency, Cytochrome P450 CYP2D6 Enzyme Deficiency, Cytochrome P450 CYP2C9 Enzyme Deficiency, Cytochrome P450 CYP2C19 Enzyme Deficiency, Cytochrome P450 CYP3A Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2C9 Variant, Poor Metabolizer Due to Cytochrome p450 CYP2C19 Variant, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant
- Interventions
- Pharmacogenetic testing
- Genetic
- Lead sponsor
- Genelex Corporation
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Searcy, Arkansas
- Conditions
- Alpha1-Antitrypsin Deficiency
- Interventions
- Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
- Biological
- Lead sponsor
- Grifols Therapeutics LLC
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Miami, Florida + 2 more
- Conditions
- NGLY1 Deficiency
- Interventions
- GS-100
- Genetic
- Lead sponsor
- Grace Science, LLC
- Industry
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 2
- States / cities
- Oakland, California • Houston, Texas
- Conditions
- Immunodeficiency, Hematopoietic Stem Cell Transplantation
- Interventions
- Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF), Tacrolimus, Busulfan, Total Body Irradiation (TBI), Fludarabine, Cyclophosphamide (CY), Post-Transplant Cyclophosphamide (PTCY)
- Procedure · Drug · Radiation
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sarcoma, Kaposi, HIV Infections
- Interventions
- SU5416
- Drug
- Lead sponsor
- SUGEN
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- South San Francisco, California
- Conditions
- HIV Infection
- Interventions
- Not listed
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 12 Years to 24 Years
- Enrollment
- 440 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2007
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Interventions
- ALXN1101, Placebo
- Drug
- Lead sponsor
- Origin Biosciences
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Mucopolysaccharidosis II
- Interventions
- iduronate-2-sulfatase enzyme replacement therapy, Placebo
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years to 25 Years · Male only
- Enrollment
- 96 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 4
- States / cities
- Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
- Conditions
- Adverse Drug Events, Adverse Drug Reactions, Drug Interaction Potentiation, Drug Metabolism, Poor, CYP2D6-RELATED, Drug Metabolism, Poor, CYP2C19-RELATED, Cytochrome P450 Enzyme Deficiency, Cytochrome P450 CYP2D6 Enzyme Deficiency, Cytochrome P450 CYP2C9 Enzyme Deficiency, Cytochrome P450 CYP2C19 Enzyme Deficiency, Cytochrome P450 CYP3A Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2C9 Variant, Poor Metabolizer Due to Cytochrome P450 CYP2C19 Variant, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant
- Interventions
- Not listed
- Lead sponsor
- Genelex Corporation
- Industry
- Eligibility
- 65 Years and older
- Enrollment
- 800 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 7
- States / cities
- Garden Grove, California • Denver, Colorado • Lexington, Kentucky + 3 more
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa, Placebo
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 11
- States / cities
- Tucson, Arizona • Palo Alto, California • San Francisco, California + 8 more
- Conditions
- Adenosine Deaminase Severe Combined Immunodeficiency
- Interventions
- elapegademase-lvlr
- Biological
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 0 Months to 65 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more