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. * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion
Investigational Site Number: 1560001
Shanghai, China
RECRUITINGIncidence of AEs, SAEs, AESIs including IARs during the TE period
Time frame: From baseline to Week 56
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
Time frame: From baseline to Week 56
Proportion of participants who are alive and free of invasive ventilation at Week 52
Time frame: At Week 52
Change from baseline to Week 52 in LVM Z score evaluated by echocardiography
Time frame: From baseline to Week 52
The absolute change in body growth Z scores from baseline to Week 52
Time frame: From baseline to Week 52
The percentiles of the body growth Z scores changes from baseline to Week 52
Time frame: From baseline to Week 52
Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)
Time frame: From baseline to Week 52
Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)
Time frame: From baseline to Week 52
Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52
Time frame: At Week 52
Trial Transparency email recommended (Toll free for US & Canada)
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