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ClinicalTrials.gov public records Last synced May 21, 2026, 11:09 PM EDT

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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Pompe Disease Infantile-Onset Clear all
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months to 36 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
3
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 24 Months
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
3
States / cities
Gainesville, Florida • Decatur, Georgia • Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 25, 2022 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Alglucosidase Alfa, Methotrexate, Rituximab
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2014 · Synced May 21, 2026, 11:09 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
6
States / cities
Birmingham, Alabama • Gainesville, Florida • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced May 21, 2026, 11:09 PM EDT
Terminated Phase 4 Interventional Results available
View directory record Official record
Conditions
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 12 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
14
States / cities
Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 17, 2016 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Glycogen Storage Disease Type II-Pompe's Disease
Interventions
Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months to 17 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2027
U.S. locations
3
States / cities
Valhalla, New York • Durham, North Carolina • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Alglucosidase alfa GZ419829
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 6 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced May 21, 2026, 11:09 PM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II Infantile Onset
Interventions
Cipaglucosidase alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Up to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Gainesville, Florida • Atlanta, Georgia • Durham, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2014 · Synced May 21, 2026, 11:09 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions
Aldurazyme (laronidase)
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 21, 2026, 11:09 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced May 21, 2026, 11:09 PM EDT