A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Development Studies in France

  • End date
    May 31, 2023
  • participants needed
  • sponsor
    Genzyme, a Sanofi Company
Updated on 14 September 2022
beta-human chorionic gonadotropin
replacement therapy


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 May 2023, whichever comes first.

  • Study visit frequency: every 2 weeks


Treatment duration approximately 16 months

Condition Glycogen Storage Disease Type II
Treatment Avalglucosidase alfa (GZ402666)
Clinical Study IdentifierNCT05164055
SponsorGenzyme, a Sanofi Company
Last Modified on14 September 2022


Yes No Not Sure

Inclusion Criteria

Patient with Pompe Disease who has previously completed Study EFC14028, LTS13769, or ACT14132 of avalglucosidase alfa studies 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
The patient is concurrently participating in another clinical study of investigational treatment
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
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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