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

NCT03687333 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2022-04-13

No results posted yet for this study

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.

Conditions

  • Glycogen Storage Disease Type II

Interventions

DRUG

ALGLUCOSIDASE ALFA (MYOZYME)

Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Months
Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-04
Primary Completion
2020-12-30
Completion
2020-12-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03687333 on ClinicalTrials.gov