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

NCT ID: NCT06666413

Last Updated: 2025-05-28

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-07
• Study Completion Date: 2028-05-02

Brief Summary

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.

Conditions

Glycogen Storage Disease Type II; Pompe's Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

avalglucosidase alfa

Avalglucosidase alfa will be administered by IV infusion following reconstitution and dilution at a dose of 20 mg/kg body weight qow

Group Type: EXPERIMENTAL

Avalglucosidase alfa

Intervention Type: DRUG

Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion

Interventions

Avalglucosidase alfa

Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion

Intervention Type: DRUG

Other Intervention Names

GZ402666; Nexviazyme

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 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.

• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigational Site Number: 1560001

Shanghai, , China

Site Status: RECRUITING

Countries

China

Central Contacts

Trial Transparency email recommended (Toll free for US & Canada)

Role: CONTACT

contact-us@sanofi.com

800-633-1610 ext. option 6

Other Identifiers

U1111-1295-2947

Identifier Type: REGISTRY

Identifier Source: secondary_id

LPS18086

Identifier Type: -

Identifier Source: org_study_id