Actively Recruiting
A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Children With Infantile-onset Pompe Disease
Led by Genzyme, a Sanofi Company · Updated on 2026-03-23
13
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety and effects of avalglucosidase alfa given by intravenous infusion in male and female Chinese children with infantile-onset Pompe disease (IOPD). Participants may be new to treatment or have been treated before with enzyme replacement therapy (ERT). This is a Phase 4, open-label, single-arm study lasting about 64 weeks, sponsored by Genzyme, a Sanofi Company. Avalglucosidase alfa is given as an IV infusion at a dose of 20 mg per kilogram of body weight every other week. The study includes a screening period of up to 8 weeks, followed by a 52-week treatment period, and then a 4-week follow-up period, which may be shortened to 2 weeks if participants join another study or begin commercial ERT. Participants will have 30 visits in total, including 29 site visits and one follow-up phone call. During the study, participants will be monitored for adverse events, lab test abnormalities, vital signs, and heart function via ECG from baseline to Week 56. Researchers will also assess survival free of invasive ventilation, heart muscle thickness, body growth, walking distance in a six-minute walk test, motor function, and motor milestone achievements at Week 52. Safety and efficacy evaluations will help understand the treatment's impact over the course of the study.
CONDITIONS
Brief 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 under 18 years of age at the time of signing informed consent
- Symptoms of Pompe disease began before 12 months of age (adjusted for gestation if born early)
- Confirmed diagnosis of infantile-onset Pompe disease by enzyme deficiency and gene mutations
- Documented cardiomyopathy at diagnosis
- Parent or legally authorized representative must be able to provide signed informed consent
- Contraceptive use consistent with local regulations
You will not qualify if you...
- Major congenital abnormalities that could prevent study participation or reduce survival
- Clinically significant organic diseases other than Pompe disease symptoms
- Prior treatment with enzyme replacement therapies other than alglucosidase alfa or avalglucosidase alfa
- Treatment with alglucosidase alfa or avalglucosidase alfa less than one week before starting study drug
- Anticipated use of prohibited therapies for Pompe disease during the study
- Use of other investigational drugs within 30 days or 5 half-lives before enrollment
- Any condition judged unsuitable by the investigator, including risk of noncompliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 56 weeks
Participants receive Avalglucosidase alfa by intravenous infusion every other week at a dose of 20 mg/kg body weight.
Regular visits for IV infusions every other week with additional assessments up to Week 56
Trial Site Locations
Total: 1 location
1
Investigational Site Number: 1560001
Shanghai, China, 200127
Actively Recruiting
Research Team
T
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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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