A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease

EFC14028

Primary Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa. Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).

2016-11-02

2020-03-19

2023-05-31

Completed

Early phase I

101

Inclusion criteria : * The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations. * The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable. Exclusion criteria: * The participant is \<3 years of age. * The participant has known Pompe specific cardiac hypertrophy. * The participant is wheelchair dependent. * The participant is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device. * The participant requires invasive-ventilation (non-invasive ventilation is allowed). * The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted and less than or equal to 85% predicted. * The participant (and participant's legal guardian if participant is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol. * The participant has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease. * The participant has prior or current use of immune tolerance induction therapy. * The participant, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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2024-04-04