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Active, not recruiting Phase 4

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

NCT05164055 · tracked via the Priya Life Science France tracker
Phase
Phase 4
Started
2022-07-11
Last updated
2026-09-09

Condition(s) studied

Glycogen Storage Disease Type II

Investigational drug(s) / intervention(s)

Avalglucosidase alfa (GZ402666) →

Avalglucosidase alfa (GZ402666): Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Study 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

Sex
ALL
Min age
6 Months
Max age
—
Healthy volunteers
No
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 \<18 years of age, is willing to provide assent if deemed able to do so. * The patient (and patient's legal guardian if patient is \<18 years of age) must have the ability to comply with the clinical protocol. * The patient, if female and of childbearing potential, must have a negative pregnancy test result \[urine beta-human chorionic gonadotropin (β-HCG)\] at enrollment. * Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception. Exclusion Criteria: * Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients. * Patient who permanently discontinued avalglucosidase alfa in a previous clinical study * Pregnant or breastfeeding female patient * The patient is concurrently participating in another clinical study of investigational treatment. * The patient, in opinion of the Investigator, is unable to comply with the requirements of the study. * The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatobiliary, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival. * Individuals accommodated in an institution because of regulatory or legal order; prisoners, or patients who are legally institutionalized. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Primary outcome measure(s)

  • Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death — From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months

Trial sites (11)

FacilityCityRegionStatus
Investigational Site Number : 2500004 Bordeaux France
Investigational Site Number : 2500005 Brest France
Investigational Site Number : 2500008 Clermont-Ferrand France
Investigational Site Number : 2500009 Lille France
Investigational Site Number : 2500003 Lyon France
Investigational Site Number : 2500001 Marseille France
Investigational Site Number : 2500006 Nantes France
Investigational Site Number : 2500007 Nice France
Investigational Site Number : 2500002 Paris France
Investigational Site Number : 2500010 Paris France
Investigational Site Number : 2500011 Tours France
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05164055 on ClinicalTrials.gov ↗ ← All trials in France