China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD (NCT06666413) | Clinical Trial Compass
RecruitingPhase 4
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
China13 participantsStarted 2025-05-07
Plain-language 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.
Who can participate
Age range
17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 oth…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of AEs, SAEs, AESIs including IARs during the TE period
Timeframe: From baseline to Week 56
2
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
Timeframe: From baseline to Week 56
Trial details
NCT IDNCT06666413
SponsorGenzyme, a Sanofi Company
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2028-05-02
Contact for this trial
Trial Transparency email recommended (Toll free for US & Canada)