Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

The goal of this clinical trial is to study the efficacy and safety of treatment with avalglucosidase alfa in patients with late onset Pompe disease that previously deteriorated on alglucosidase alfa. The main question it aims to answer is: * Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa safe? * Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa potentially more effective? Participants will switch to biweekly avalglucosidase alfa infusions (instead of alglucosidase alfa infusions) and perform assessment for: * Efficacy: muscle strength and function, pulmonary function, patient-reported outcomes. * Safety: Adverse events assessment, physical examination, clinical laboratory evaluations, vital signs, ECGs and immunogenicity assessments. * Pharmacokinetic assessments during 2 avalglucosidase alfa infusions.

NCT Number	NCT07652814
Lead Sponsor	Iris Plug Collaborators: Sanofi
Conditions	Glycogen Storage Disease Type II Late Onset, Pompe Disease (Late-onset), Pompe Disease Late-Onset, Pompe's Disease Juvenile Onset, Pompe Disease
Enrollment	6 participants
Start Date	2022-10-13
Primary Completion	2028-03-09 (estimated)
Study Completion	2028-03-09 (estimated)
Updated on ClinicalTrials.gov	2026-06-17