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

NCT04848779 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 16

Last updated 2025-05-13

No results posted yet for this study

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.

Conditions

  • Glycogen Storage Disease Type II

Interventions

DRUG

Alglucosidase alfa GZ419829

Pharmaceutical form: Lyophilized powder for solution Route of administration: intravenous

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Eligibility

Min Age
0 Days
Max Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-10
Primary Completion
2026-10-28
Completion
2026-10-28
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • France
  • Germany
  • Italy
  • Netherlands
  • Spain
  • Taiwan
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04848779 on ClinicalTrials.gov