New hope for kids with pompe disease: experimental drug shows promise

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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: Participants are excluded from the study if any of the following criteria apply: * 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.