Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa

• Status: Recruiting
• Trial end date: 2026-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and female participants less than or equal to 6 months of age with IOPD.

Study details include:

• Study duration: Screening - up to 4 weeks;

• Primary Analysis Period (PAP) - 52 weeks;

• Extended Treatment Period (ETP) - 52 weeks;

• Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years.

• Treatment duration: Up to 4 years

• Visit frequency: every other week and potentially every week

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sanofi

Criteria

Inclusion Criteria:

• Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates).

• Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment.

• Participants must have cardiomyopathy at the time of diagnosis: ie, LVMI equivalent to mean age specific LVMI

• 1 standard deviation for participants diagnosed by newborn screening or sibling screening;

• 2 standard deviation for participants diagnosed by clinical evaluation.

• Parents or legally authorized representative(s) must be capable of giving signed informed consent.

Exclusion Criteria:

• Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment.

• Participants with major congenital abnormality.

• Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).

• Participant received enzyme-replacement therapy (ERT) with recombinant human acid α glucosidase (rhGAA) from any source.

• Participant who has previously been treated in any clinical trial of avalglucosidase alfa.

• Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.