A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
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: IV; Pharmaceutical form: powder for concentrate for solution for infusion,
Route of administration: IV
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 22.0
Sex: All
Minimum age: 6 Months
Maximum age: 17 Years
Healthy volunteers: No

Summary:

      Primary Objective:

      To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset
      Pompe disease previously treated with alglucosidase alfa.

      Secondary Objective:

      To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the
      preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.
    

Eligibility criteria:

        Inclusion criteria:

          -  The participants has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any
             tissue source.

          -  The participants who has reached legal age of majority as defined by local regulation,
             or the participant's legal guardian(s) must provide signed informed consent prior to
             performing any study-related procedures. If the participant is legally minor per local
             regulations, assent shall be obtained from participants, if applicable.

          -  The participants (and participant's legal guardian if participant is legally minor as
             defined by local regulation) must have the ability to comply with the clinical
             protocol.

          -  The participants is less than 18 years old.

          -  The participants, if female and of childbearing potential, must have a negative serum
             pregnancy test (beta-human chorionic gonadotropin) and must not breastfeeding at
             screening/Baseline.

          -  The participant has cardiomyopathy at the time of diagnosis: i.e., left ventricular
             mass index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations.

          -  The participant has been receiving a stable dose of alglucosidase alfa regularly for a
             minimum of 6 months immediately prior to study entry.

          -  For participants in Stage 1: The participant has documented evidence of clinical
             decline in at least 1 of the following parameters related to Pompe Disease and not
             related to intercurrent illness as assessed by the Investigator: respiratory function,
             motor skills, and/or cardiac parameters.

          -  For participants in Stage 2: The participant has documented evidence of suboptimal
             clinical response in at least 1 of the following parameters related to Pompe Disease
             and not related to intercurrent illness as assessed by the Investigator: respiratory
             function, motor skills, and/or new onset of ptosis.

        Exclusion criteria:

          -  The participant has high antibody titer to alglucosidase alfa.

          -  The participant has a high risk for a severe allergic reaction to neoGAA
             (avalglucosidase alfa).

          -  The participant requires any prohibited concomitant medications (e.g., immune
             modulatory treatment) for the duration of the study.

          -  The participant has previously participated in any ACT14132 study cohort.

          -  Female participant of childbearing potential not protected by highly effective
             contraceptive method of birth control and/or who is unwilling or unable to tested for
             pregnancy.

        The above information is not intended to contain all considerations relevant to a
        participant's potential participation in a clinical trial.