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ClinicalTrials.gov public records Last synced May 22, 2026, 4:00 AM EDT

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 36 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Enzyme Deficiency Clear all
Completed Phase 1 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 24, 2017 · Synced May 22, 2026, 4:00 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
HIV Infections
Interventions
Dose adjustment of Kaletra
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
4 Years to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Aug 4, 2015 · Synced May 22, 2026, 4:00 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced May 22, 2026, 4:00 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Sphingolipidoses, Enzyme Deficiency
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Coronary Artery Disease, Atherosclerosis
Interventions
ACP-501
Drug
Lead sponsor
MedImmune LLC
Industry
Eligibility
30 Years to 85 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced May 22, 2026, 4:00 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Homocystinuria
Interventions
Pegtibatinase, Placebo
Drug
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
8
States / cities
Culver City, California • Aurora, Colorado • Miami, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced May 22, 2026, 4:00 AM EDT
Completed No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 28, 2017 · Synced May 22, 2026, 4:00 AM EDT
Completed No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 28, 2017 · Synced May 22, 2026, 4:00 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
HIV Infections, Hepatitis C
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Year to 20 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2003
U.S. locations
37
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 29 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced May 22, 2026, 4:00 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
HIV Infections, HIV Wasting Syndrome, Lipodystrophy
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Month to 12 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
2
States / cities
The Bronx, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
8 Months and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced May 22, 2026, 4:00 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 29, 2021 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced May 22, 2026, 4:00 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 18, 2015 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
sebelipase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
5
States / cities
Eureka, California • Sacramento, California • San Francisco, California + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2018 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Ebola Virus Disease, Marburg Virus Disease
Interventions
cAd3-Marburg, cAd3-EBO-S
Biological
Lead sponsor
Albert B. Sabin Vaccine Institute
Other
Eligibility
18 Years to 50 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced May 22, 2026, 4:00 AM EDT
Completed Not applicable Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 22, 2017 · Synced May 22, 2026, 4:00 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Interventions
There are no interventions in this study.
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Stanford, California • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2018 · Synced May 22, 2026, 4:00 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced May 22, 2026, 4:00 AM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Late-onset Pompe Disease
Interventions
DNL952
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 22, 2026, 4:00 AM EDT
Terminated Phase 1 Interventional Results available
View directory record Official record
Conditions
Human Immunodeficiency Virus Type 1 (HIV-1) Infection
Interventions
Vesatolimod, Cobicistat, Voriconazole, Rifabutin
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
5
States / cities
Long Beach, California • Miami, Florida • West Palm Beach, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2024 · Synced May 22, 2026, 4:00 AM EDT