Treatment Frequency Reduction in Pompe Disease

The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.

NCT Number	NCT06575829
Lead Sponsor	Erasmus Medical Center
Conditions	Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II, Acid Maltase Deficiency
Enrollment	10 participants
Start Date	2024-10-01
Primary Completion	2026-12-31 (estimated)
Study Completion	2027-12-31 (estimated)
Updated on ClinicalTrials.gov	2024-08-28