Resumen

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).

Descripción detallada

The duration of the study per participant will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to less than (\<) 18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

Elegibilidad

Edad mínima: 3 YearsSexo: ALL

Criterios de inclusión

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.

Criterios de exclusión

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.

Intervenciones

drug

Avalglucosidase alfa (GZ402666)

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

drug

Alglucosidase alfa (GZ419829)

Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

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