This study is currently not recruiting participants.

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

Investigating Long-term Access to Enzyme Replacement Therapy for Pompe Disease

Not Recruiting
6 months or above
All
Phase 4
17 participants needed

Study Overview

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

Study Details

Treatment duration approximately 4 years and 3 months: until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Glycogen Storage Disease Type II
  • Age: 6 months or above
  • Gender: All

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.

This study investigates Pompe disease. It follows up with participants who have completed previous studies and provides ongoing access to enzyme replacement therapy.

The focus of this study is to evaluate the long-term safety and effectiveness of the enzyme replacement therapy. It will help understand how well the treatment works over an extended period.

  • Who can participate: Participants must have completed specific previous studies related to Pompe disease in France. They must be able to provide informed consent and comply with study requirements. Female participants of childbearing potential must have a negative pregnancy test.
  • Study details: Participants will receive enzyme replacement therapy every two weeks. A placebo is not used in this study. The study will provide access to the therapy until it becomes reimbursed in France or until September 2026.
  • Study timelines and visits: The study will last approximately 4 years and 3 months. The study requires visits every 2 weeks.
Updated on 31 Oct 2025. Study ID: NCT05164055