Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Condition: Glycogen Storage Disease Type II;Pompe's Disease
MeSH terms: Glycogen Storage Disease Type II; Glycogen Storage Disease
Intervention: Avalglucosidase alfa (GZ402666); Alglucosidase alfa (GZ419829)
Intervention description: Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous; Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 100.0
Sex: All
Minimum age: 3 Years
Healthy volunteers: No

Summary:

      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).
    

Eligibility criteria:

        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.