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

Phase 4Active Not RecruitingNCT05164055

Genzyme, a Sanofi Company17 enrolled

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Glycogen Storage Disease Type II

Interventions

Avalglucosidase alfa (GZ402666)DRUG

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

Eligibility

Sex: ALLMin age: 6 Months

Medical Language ↔ Plain English

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.

Locations (11)

Investigational Site Number : 2500004

Bordeaux, France

Investigational Site Number : 2500005

Brest, France

Investigational Site Number : 2500008

Clermont-Ferrand, France

Outcomes

Primary Outcomes

Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death

Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 3 months

Secondary Outcomes

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

The primary measurement is the distance in meters walked by the participant on a flat, hard surface in 6 minutes. The distance walked in meters will be recorded and the corresponding percent predicted value will be calculated. The greater the distance (that a participant could walk in 6 minutes), the greater the endurance.

Time frame: 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

The QMFT is an observer administered test to evaluate changes in motor function. QMFT comprises of 16 items specifically difficult for participants with Pompe disease. Each item is scored separately on a 5-point ordinal scale (ranged from 0 to 4, higher score indicated better outcome). Total QMFT score is obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), higher score represented better outcome.

Time frame: 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

FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position, also tested in supine position. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) \* 100. Maximum Inspiratory Pressure (MIP) is a quick and non-invasive test to measure strength of inspiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MIP refers to how much air pressure force an individual creates by inhaling through the mouth as hard as possible. Maximum Expiratory Pressure (MEP) is a quick and non-invasive test to measure strength of expiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MEP is the greater pressure generated during maximal expiration.

Data from ClinicalTrials.gov

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