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

Running, not enrolling · Phase 2

Conditions studied: Glycogen Storage Disease Type II-Pompe's Disease

In brief

This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.

Key facts

Study ID

NCT03019406

Run by

Genzyme, a Sanofi Company

People needed

22

Starts

2017-10-12

Expected to finish

2027-08-10

Last updated by the study team

2026-01-30

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Regional Medical Genetics Center of New York Site Number : 8400002 — Valhalla, New York, United States
• Duke University Medical Center Site Number : 8400001 — Durham, North Carolina, United States
• Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005 — Seattle, Washington, United States
• Investigational Site Number : 2500004 — Nantes, France
• Investigational Site Number : 2500003 — Paris, France
• Investigational Site Number : 2500002 — Paris, France
• Investigational Site Number : 2500001 — Tours, France
• Investigational Site Number : 3920001 — Fuchu-shi, Tokyo, Japan
• Investigational Site Number : 3920002 — Fuchu-shi, Tokyo, Japan
• Investigational Site Number : 1580001 — Hsinchu, Taiwan
• Investigational Site Number : 8260001 — London, London, City of, United Kingdom
• Investigational Site Number : 8260002 — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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