NCT03687333

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 0 Months – 12 Months
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
DRUG	ALGLUCOSIDASE ALFA (MYOZYME)	Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

Timeline

• Start date: 2018-12-04
• Primary completion: 2020-12-30
• Completion: 2020-12-30
• First posted: 2018-09-27
• Last updated: 2022-04-13

Locations

1 site across 1 country: China