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

NCT06666413 | Recruiting Phase 4

• 2 other identifiers: LPS18086U1111-1295-2947
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 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

Outcome Measures

Primary Outcomes (2)

• Incidence of AEs, SAEs, AESIs including IARs during the TE period

  From baseline to Week 56

• Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period

  From baseline to Week 56

Secondary Outcomes (7)

• Proportion of participants who are alive and free of invasive ventilation at Week 52

  At Week 52

• Change from baseline to Week 52 in LVM Z score evaluated by echocardiography

  From baseline to Week 52

• The absolute change in body growth Z scores from baseline to Week 52

  From baseline to Week 52

• The percentiles of the body growth Z scores changes from baseline to Week 52

  From baseline to Week 52

• Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)

  From baseline to Week 52

Study Arms (1)

avalglucosidase alfa

EXPERIMENTAL

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

Drug: Avalglucosidase alfa

Interventions

Avalglucosidase alfa DRUG

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

Also known as: GZ402666, Nexviazyme

avalglucosidase alfa

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (1)

Investigational Site Number: 1560001

Shanghai, 200127, China

RECRUITING

Related Links

• LPS18086 Plain Language Results Summary

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

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

CONTACT

800-633-1610; contact-us@sanofi.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2024
• First Posted: October 30, 2024
• Study Start: May 7, 2025
• Primary Completion (Estimated): May 2, 2028
• Study Completion (Estimated): May 2, 2028
• Last Updated: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)