Overview

Treatment Frequency Reduction in Pompe Disease

Status:
NOT_YET_RECRUITING
Trial end date:
2027-12-31
Target enrollment:
Participant gender:
Summary
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.
Phase:
PHASE4
Details
Lead Sponsor:
Erasmus Medical Center