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Clinical trial

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

NCT04808505 is registered in ClinicalTrials.gov with Amicus Therapeutics as lead sponsor.

NCT NCT04808505
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
Lead sponsor
Amicus Therapeutics
Phase
Phase 3
Overall status
Recruiting
Start date
July 18, 2023
Completion date
July 31, 2027
Enrollment
36 (estimated)
Sites
14

Conditions

As the registry lists them; a condition with its own page is linked.

glycogen storage disease type ii infantile onset