Last updated on April 2019

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


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Glycogen Storage Disease Type II-Pompe's Disease
  • Age: Between 1 - 17 Years
  • Gender: Male or Female

Inclusion criteria:

  • The patient has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source.
  • The patient who has reached legal age of majority as defined by local regulation, or the patient's legal guardian(s) must provide signed informed consent prior to performing any study-related procedures. If the patient is legally minor per local regulations, assent shall be obtained from patients, if applicable.
  • The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
  • The patient is <18 years old.
  • The patient, if female and of childbearing potential, must have a negative serum pregnancy test (beta-human chorionic gonadotropin) and must not be breastfeeding at screening/baseline.
  • The patient 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 patient 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 patient 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 patient 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 patient has high antibody titer to alglucosidase alfa.
  • The patient has a high risk for a severe allergic reaction to neoGAA.
  • The patient requires any prohibited concomitant medications (e.g., immune modulatory treatment) for the duration of the study.
  • The patient has previously participated in any ACT14132 study cohort.
  • Female patient of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to be tested for pregnancy

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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