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Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2025-10-31

No results posted yet for this study

Summary

This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to enzyme replacement therapy (ERT) with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first.

\- Study visit frequency: every 2 weeks

Conditions

  • Glycogen Storage Disease Type II

Interventions

DRUG

Avalglucosidase alfa (GZ402666)

Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-11
Primary Completion
2026-09-30
Completion
2026-09-30

Countries

  • France

Study Locations

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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 NCT05164055 on ClinicalTrials.gov