China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Launched by GENZYME, A SANOFI COMPANY · Oct 29, 2024

Keywords

ClinConnect Summary

The China Post-approval Commitment (PAC) Study is a clinical trial designed to evaluate the safety and effectiveness of a new treatment called avalglucosidase alfa for patients with infantile-onset Pompe disease (IOPD), a rare genetic condition that affects muscle function. This study will include both boys and girls under 18 years of age who have shown symptoms of Pompe disease before they turned one and have a confirmed diagnosis through specific genetic testing. To participate, candidates must also have signs of heart problems related to the disease.

The trial will last about 64 weeks, including a screening phase of up to 8 weeks, a treatment phase of 52 weeks, and a 4-week follow-up period. Participants will have around 30 visits, mostly at the study site, and will receive the treatment through an intravenous (IV) infusion. It's important to note that this study is not yet recruiting participants. If you think you or someone you know might qualify, it’s a good idea to discuss it with a healthcare provider who can provide more detailed information and guidance.

Gender

ALL

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.