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

NCT05164055 | Active Not Recruiting Phase 4

• 4 other identifiers: PTA17333U1111-1266-54342024-514773-222021-002590-26
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 11

Brief 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

Outcome Measures

Primary Outcomes (1)

• 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 3 months

Secondary Outcomes (9)

• Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants

  From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months (at enrolment and every 6 months)

• Assessment of quick motor function test (QMFT) for LOPD participants

  From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months (at enrolment and every 6 months)

• Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants

  From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months (at enrolment and every 6 months)

• Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants

  From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months (at enrolment and every 6 months)

• Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants

  From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months (at enrolment and every 6 months)

Study Arms (1)

Avalglucosidase alfa

EXPERIMENTAL

Administered intravenously every other week

Drug: Avalglucosidase alfa (GZ402666)

Interventions

Avalglucosidase alfa (GZ402666) DRUG

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

Also known as: Nexviadyme®

Avalglucosidase alfa

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (11)

Investigational Site Number : 2500004

Bordeaux, 33000, France

Location

Investigational Site Number : 2500005

Brest, 29609, France

Location

Investigational Site Number : 2500008

Clermont-Ferrand, 63000, France

Location

Investigational Site Number : 2500009

Lille, 59037, France

Location

Investigational Site Number : 2500003

Lyon, 69003, France

Location

Investigational Site Number : 2500001

Marseille, 13385, France

Location

Investigational Site Number : 2500006

Nantes, 44093, France

Location

Investigational Site Number : 2500007

Nice, 06200, France

Location

Investigational Site Number : 2500002

Paris, 75013, France

Location

Investigational Site Number : 2500010

Paris, 75015, France

Location

Investigational Site Number : 2500011

Tours, 37044, France

Location

Related Links

• PTA17333 Plain Language Results Summary

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2021
• First Posted: December 20, 2021
• Study Start: July 11, 2022
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: October 31, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

FR (11)