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

Phase 4

Completed

• Conditions: Glycogen Storage Disease Type II
• Interventions: Drug: ALGLUCOSIDASE ALFA (MYOZYME)

• Registration Number: NCT03687333
• Lead Sponsor: Genzyme, a Sanofi Company

Brief 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.

Study Timeline

• Study First Submitted: 2018-09-24
• Study First Submitted QC: 2018-09-25
• Study First Posted: 2018-09-27
• Study Start: 2018-12-04
• Primary Completion: 2020-12-30
• Study Completion: 2020-12-30
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-05
• Last Update Posted: 2022-04-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Alglucosidase Alfa therapy	ALGLUCOSIDASE ALFA (MYOZYME)	Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.

Primary Outcome Measures

Name	Time	Method
Survival	at week 52	The proportion of patients alive at the end of study
Left Ventricular Mass Index (LVMI)	at week 52	Change from baseline in LVMI

Secondary Outcome Measures

Name	Time	Method
Growth in body weight and length	at week 52	Physical growth: Change from baseline at Week 52 with regards to length and weight
Motor development milestones	at week 52	Number of motor development milestones achieved at Week 52 and change from baseline
Any ventilation-free survival	at week 52	Survival free of any ventilator use at 52-week treatment
GESELL Development Scale	at week 52	Change from baseline at Week 52 on GESELL Developmental Scale
Cardiac failure	at week 52	Proportion of patients with signs and/or symptoms of cardiac failure at Week 52
Invasive ventilation-free survival	at week 52	Survival free of invasive ventilator use at 52-week treatment

Trial Locations

Locations (1)

Investigational site number; 🇨🇳 Shanghai, China