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ClinicalTrials.gov public records Last synced Oct 21, 2026, 6:26 PM EDT

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Showing 1–24 of 35 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Enzyme Deficiency Clear all
Completed No phase listed Observational

Prevalence of Hepatitis C Virus Infection in HIV-Infected Children

NCT00037076
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 31, 2012 · Synced Oct 21, 2026, 6:26 PM EDT
Active, not recruiting Phase 3 Interventional

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

NCT06199531
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Oct 21, 2026, 6:26 PM EDT
Completed No phase listed Observational

A Novel Method to Determine HIV Incidence Among Youth

NCT00103883
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 Mar 1, 2017 · Synced Oct 21, 2026, 6:26 PM EDT
Recruiting Phase 2 Interventional

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome

NCT05027945
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 6:26 PM EDT
Recruiting No phase listed Observational

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326
View directory record Official record
Conditions
Pompe Disease
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 21, 2026, 6:26 PM EDT
Completed No phase listed Observational

The Effect of Anti-HIV Treatment on Body Characteristics of HIV-Infected Children

NCT00006064
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 29, 2014 · Synced Oct 21, 2026, 6:26 PM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 21, 2026, 6:26 PM EDT
Terminated Phase 1 Interventional Results available

Drug-Drug Interaction Study of Vesatolimod in Adults With HIV-1 Who Have Very Low or Undetectable Virus Levels

NCT05458102
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 3, 2024 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Evaluation of Safety, Tolerability and Immune Responses of Ebola-S and Marburg Vaccines in Healthy Adults

NCT04723602
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 3, 2022 · Synced Oct 21, 2026, 6:26 PM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724
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
4
States / cities
Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1 Interventional

Intrathecal Enzyme Replacement for Hurler Syndrome

NCT00638547
View directory record Official record
Conditions
Hurler Syndrome
Interventions
IRT Laronidase
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
6 Months to 3 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2018
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 3, 2018 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1 Interventional

Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease

NCT01554800
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 14, 2019 · Synced Oct 21, 2026, 6:26 PM EDT
Terminated No phase listed Observational

Optimizing Antiretroviral Therapy in HIV-Infected Children and Adolescents

NCT00207948
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 5, 2015 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641
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 10, 2021 · Synced Oct 21, 2026, 6:26 PM EDT
Completed No phase listed Observational

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220
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 28, 2015 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Not applicable Interventional Results available

Pharmacogenetic Testing Among Home Health Patients

NCT02378220
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
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
Source: ClinicalTrials.gov public record
Updated Sep 20, 2019 · Synced Oct 21, 2026, 6:26 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

NCT04885179
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 24, 2025 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

NCT01307098
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 11, 2018 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 2 Interventional

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

NCT00051935
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 5, 2014 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994
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 4, 2019 · Synced Oct 21, 2026, 6:26 PM EDT
Completed No phase listed Observational

YouScript IMPACT Registry

NCT02191358
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 Mar 1, 2017 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 2 Interventional Results available

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

NCT01488097
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 20, 2018 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT01894165
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 19, 2019 · Synced Oct 21, 2026, 6:26 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

NCT04722887
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 21, 2026, 6:26 PM EDT