Actively Recruiting
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Led by Genzyme, a Sanofi Company · Updated on 2026-03-23
13
Participants Needed
1
Research Sites
155 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must be <18 years of age at the time of signing informed consent
- Documented onset of Pompe disease symptoms before 12 months of age, corrected for gestation if born before 40 weeks
- Diagnosis of infantile-onset Pompe disease confirmed by GAA enzyme deficiency and GAA gene pathogenic mutations
- Documented cardiomyopathy at the time of diagnosis
- Contraceptive use consistent with local regulations
- Parent or legally authorized representative capable of providing signed informed consent
You will not qualify if you...
- Major congenital abnormalities that could prevent participation or reduce survival
- Clinically significant organic diseases unrelated to Pompe disease
- Prior enzyme replacement therapy other than alglucosidase alfa or avalglucosidase alfa
- Receipt of alglucosidase alfa or avalglucosidase alfa within one week before first dose of study drug
- Anticipated use of prohibited therapies for Pompe disease during the study
- Use of other investigational drugs within 30 days or 5 elimination half-lives before enrollment
- Any condition or risk judged by the investigator to make the participant unsuitable for the study or at risk of noncompliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Investigational Site Number: 1560001
Shanghai, China, 200127
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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