NCT06666413RecruitingPhase 4
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)
Source: ClinicalTrials.gov · Last updated 23 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 13 (estimated)
- First submitted
- 16 Oct 2024
- Start date
- 7 May 2025
- Primary completion date
- 2 May 2028
- Lead sponsor
- Genzyme, a Sanofi Company
- Diseases
- Pompe disease
- NCT number
- NCT06666413
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 13
- From 7 May 2025 to the primary completion date, 2 May 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
- 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.
Other
- Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
Who the study is looking for
- 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.
Age in the registry: - to 17 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Shanghai | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.