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Active Not Recruiting
NCT05164055
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Conditions: Glycogen Storage Disease Type II
Sex: All
Ages: 6 Months – N/A
Healthy volunteers: No
Phase: PHASE4
Enrollment: 17
Sponsor: Genzyme, a Sanofi Company
Location: Investigational Site Number : 2500004 Bordeaux
Summary
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until the conclusion of negotiations of avalglucosidase alfa in France under the standard reimbursement framework on each indication independently, infantile (IOPD) and late (LOPD), or until June 2027, whichever comes first.
\- Study visit frequency: every 2 weeks
Eligibility Criteria
Inclusion Criteria:
* Patient with LOPD or IOPD who has previously completed Study EFC14028, LTS13769, or ACT14132 in France, and reimbursement for avalglucosidase alfa is not yet granted in France.
* The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if \
Source: ClinicalTrials.gov (NCT05164055). StuddyBuddy aggregates publicly available trial information.
