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ClinicalTrials.gov public records Last synced May 22, 2026, 4:55 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–36 of 12 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Enzyme Deficiency Clear all
Completed No phase listed Observational Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced May 22, 2026, 4:55 AM EDT
Recruiting Phase 2 Interventional
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 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced May 22, 2026, 4:55 AM EDT
Active, not recruiting No phase listed Observational
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 7, 2026 · Synced May 22, 2026, 4:55 AM EDT
Terminated Phase 4 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 9, 2024 · Synced May 22, 2026, 4:55 AM EDT
Completed Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 22, 2026, 4:55 AM EDT
Completed Phase 1 Interventional
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 2, 2018 · Synced May 22, 2026, 4:55 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 28, 2020 · Synced May 22, 2026, 4:55 AM EDT
Completed Not applicable Interventional Results available
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 19, 2019 · Synced May 22, 2026, 4:55 AM EDT
Not yet recruiting Phase 3 Interventional
View directory record Official record
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
U.S. locations
10
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced May 22, 2026, 4:55 AM EDT
Active, not recruiting Phase 3 Interventional
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 3, 2026 · Synced May 22, 2026, 4:55 AM EDT
Recruiting No phase listed Observational
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 7, 2026 · Synced May 22, 2026, 4:55 AM EDT
Completed Phase 1Phase 2 Interventional
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 Dec 11, 2025 · Synced May 22, 2026, 4:55 AM EDT