NCT04848779

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 16 (estimated)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 0 Days – 6 Months
• Healthy volunteers: Not accepted

Summary

Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.

Detailed description

The planned duration of observation for each participant will be 104 weeks after enrollment, to determine secondary outcomes at 18 months (approximately 78 weeks) of age.

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
DRUG	Alglucosidase alfa GZ419829	Pharmaceutical form: Lyophilized powder for solution Route of administration: intravenous

Timeline

• Start date: 2021-06-10
• Primary completion: 2026-10-28
• Completion: 2026-10-28
• First posted: 2021-04-19
• Last updated: 2025-05-13

Locations

15 sites across 9 countries: United States, Belgium, France, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study