Primary Objective: To evaluate effect of 52-week treatment with Alglucosidase Alfa in the extension of survival and improvement of cardiomyopathy measured by Left Ventricular Mass Index in Chinese patients with infantile-onset Pompe Disease. Secondary Objectives: * To observe the improvement of physical growth, motor and cognitive development of 52-week treatment with Alglucosidase Alfa in infantile-onset Pompe Disease from the baseline. * To observe the efficacy on survival free of invasive ventilation, use of any ventilation support of 52- week treatment with Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease. * To evaluate the safety and tolerability of Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.
Total of 56 weeks in the study period, including an up to 28-day screening period and 52-week treatment period, followed by 30-day post-treatment observation period. After the end of 52-week treatment, patients' guardians could choose to participate in a patient assistance program (PAP) sponsored by Sanofi and launched before first patient out (FPO) or reimbursement from social insurance for continued treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion
Investigational site number
Shanghai, China
Survival
The proportion of patients alive at the end of study
Time frame: at week 52
Left Ventricular Mass Index (LVMI)
Change from baseline in LVMI
Time frame: at week 52
Invasive ventilation-free survival
Survival free of invasive ventilator use at 52-week treatment
Time frame: at week 52
Any ventilation-free survival
Survival free of any ventilator use at 52-week treatment
Time frame: at week 52
Growth in body weight and length
Physical growth: Change from baseline at Week 52 with regards to length and weight
Time frame: at week 52
Motor development milestones
Number of motor development milestones achieved at Week 52 and change from baseline
Time frame: at week 52
GESELL Development Scale
Change from baseline at Week 52 on GESELL Developmental Scale
Time frame: at week 52
Cardiac failure
Proportion of patients with signs and/or symptoms of cardiac failure at Week 52
Time frame: at week 52
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