Search | Clinical Trials Finder

ClinicalTrials.gov public records Last synced Jul 21, 2026, 4:29 PM EDT

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

Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.

Local D1 index available.

Filters: Condition: Pompe Disease Infantile-Onset Clear all

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

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

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

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

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

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

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

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

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

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

Conditions: Pompe Disease Infantile-Onset
Interventions: ATB200, AT2221; Biological · Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 0 Years to 17 Years
U.S. locations: 2
States / cities: Oakland, California • Gainesville, Florida

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

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

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

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

Search by objective public record fields.

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Growth and Development Study of Alglucosidase Alfa

Registry of Patients Diagnosed With Lysosomal Storage Diseases

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Pompe Telemedicine Developmental Study

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies