Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:12 PM EDT

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Showing 25–35 of 35 matching trials from the live ClinicalTrials.gov search.
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
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 Infections
Interventions
Nevirapine, Zidovudine
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day and older
Enrollment
1,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Ends 2004
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Alpha-1 15%, Liquid Alpha1-PI
Drug · Other
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
10
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 7 more
Conditions
Cytochrome P450 CYP2D6 Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant, Ultrarapid Metabolizer Due to Cytochrome P450 CYP2D6 Variant, Extensive Metabolizer Due to Cytochrome P450 CYP2D6 Variant, Cytochrome P450 CYP2C9 Enzyme Deficiency, Cytochrome P450 CYP2C19 Enzyme Deficiency, Drug Metabolism, Poor, CYP2D6-RELATED, Drug Metabolism, Poor, CYP2C19-RELATED, CYP2D6 Polymorphism
Interventions
Pharmacogenetic testing, Software-based drug & gene interaction risk analysis, MTM
Genetic · Other
Lead sponsor
Genelex Corporation
Industry
Eligibility
65 Years and older
Enrollment
341 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Salt Lake City, Utah
Completed No phase listed Observational Results available

Registry Study of Revcovi Treatment in Patients With ADA-SCID

NCT03878069
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
Completed Not applicable Interventional Accepts healthy volunteers

Drug Metabolizing Enzyme and Transporter Function in Chronic Kidney Disease

NCT02360644
Conditions
Chronic Kidney Diseases, Deficiency, Vitamin D
Interventions
Cholecalciferol
Dietary Supplement
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 70 Years
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2019
U.S. locations
2
States / cities
Aurora, Colorado • Pittsburgh, Pennsylvania
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
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Interventions
Ravicti
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Fabry Disease, Alpha Galactosidase A Deficiency
Interventions
agalsidase beta
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2024
U.S. locations
3
States / cities
Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota