Last updated on August 2019

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Brief description of study

Primary Objective:

To evaluate the safety profile of avalglucosidase alfa in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa.

Secondary Objective:

To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.

Detailed Study Description

The duration of the study per patient will be up to 3 years that will consist of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations, a 25-week treatment period, a 120-week extension period, and a 4-week post-treatment observation period.

Clinical Study Identifier: NCT03019406

Recruitment Status: Closed

Brief Description Eligibility Contact Research Team

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