Avalglucosidase alfa treatment for children with Pompe disease

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

This study is testing a new treatment called avalglucosidase alfa to see if it helps children with infantile-onset Pompe disease feel better over a year.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 13 (estimated)
• Ages: N/A to 17 Years
• Sex: All
• Sponsor: Sanofi (Industry-sponsored)
• Locations: 1 site (Shanghai)
• Trial ID: NCT06666413 on ClinicalTrials.gov

What this trial studies

This is a 52-week, open-label, single-arm study assessing the safety and efficacy of avalglucosidase alfa IV infusion in Chinese participants diagnosed with infantile-onset Pompe disease (IOPD). The study includes a screening period of up to 8 weeks, followed by a treatment phase lasting 52 weeks, and a 4-week follow-up. Participants will have a total of 30 visits, including 29 site visits and one follow-up phone call. The study aims to evaluate the effects of the treatment on participants who are either treatment-naïve or have previously received enzyme replacement therapy.

Who should consider this trial

Good fit: Ideal candidates for this study are children under 18 years old with a confirmed diagnosis of infantile-onset Pompe disease and documented cardiomyopathy.

Not a fit: Patients with major congenital abnormalities or significant organic diseases unrelated to Pompe disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the health outcomes and quality of life for children with Pompe disease.

How similar studies have performed: Previous studies on enzyme replacement therapies for Pompe disease have shown promising results, indicating potential for success with this approach.

Eligibility criteria

Inclusion Criteria:

* Participant must be \<18 years of age, at the time of signing the informed consent.
* Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
* Participants must have documented cardiomyopathy at the time of diagnosis.
* Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.

Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:

* Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
* Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
* Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
* Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
* Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
* Participants 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.

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

Where this trial is running

Shanghai

• Investigational Site Number: 1560001 — Shanghai, China (Recruiting)

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)

Conditions Glycogen Storage Disease Type II; Pompe's Disease