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 range17 Years
SexALL
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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…
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)