Overview

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

Status:
Active, not recruiting
Trial end date:
2024-12-01
Target enrollment:
Participant gender:
Summary
Primary Objective: To evaluate the safety profile of avalglucosidase alfa in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa. Secondary Objective: To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.
Phase:
Phase 2
Details
Lead Sponsor:
Genzyme, a Sanofi Company