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

Genzyme

Status and phase

Enrolling

Phase 4

Conditions

Pompe's Disease

Glycogen Storage Disease Type II

Treatments

Drug: Avalglucosidase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT06666413

U1111-1295-2947 (Registry Identifier)

LPS18086

Details and patient eligibility

About

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.

Study details include:

The study duration: total study duration is approximately 64 weeks.
- Screening period of up to 8 weeks
- Treatment period of 52 weeks
- Follow-up period of 4 weeks.
The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.

Enrollment

13 estimated patients

Sex

All

Ages

Under 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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.