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

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 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.

Study Overview

• Status: Recruiting
• Conditions: Glycogen Storage Disease Type II; Pompe's Disease
• Intervention / Treatment: Drug: Avalglucosidase alfa

• Study Type: Interventional
• Enrollment (Estimated): 13
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Trial Transparency email recommended (Toll free for US & Canada); Phone Number: option 6 800-633-1610; Email: Contact-US@sanofi.com

Study Locations

• China
  • Shanghai, China, 200127
    • Recruiting
    • Investigational Site Number: 1560001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: avalglucosidase alfa; 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; Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion; Other Names: GZ402666; Nexviazyme

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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
Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)	From baseline to Week 52
Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52	At Week 52

Collaborators and Investigators

• Sponsor: Genzyme, a Sanofi Company

Publications and helpful links

Helpful Links

• LPS18086 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): May 7, 2025
• Primary Completion (Estimated): May 2, 2028
• Study Completion (Estimated): May 2, 2028

Study Registration Dates

• First Submitted: October 16, 2024
• First Submitted That Met QC Criteria: October 29, 2024
• First Posted (Actual): October 30, 2024

Study Record Updates

• Last Update Posted (Actual): March 23, 2026
• Last Update Submitted That Met QC Criteria: March 19, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Glycogen Storage Disease Type II; GAA protein, human
• Other Study ID Numbers: LPS18086; U1111-1295-2947 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No