RecruitingPhase 4NCT06666413

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)


Sponsor

Genzyme, a Sanofi Company

Enrollment

13 participants

Start Date

May 7, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include: * The study duration: total study duration is approximately 64 weeks. * Screening period of up to 8 weeks * Treatment period of 52 weeks * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks) * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.


Eligibility

Max Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Avalglucosidase alfa for people with glycogen storage disease type ii and pompe's disease. The study is currently recruiting participants at 1 location.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGAvalglucosidase alfa

Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion


Locations(1)

Investigational Site Number: 1560001

Shanghai, China

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06666413


Related Trials