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Clinical Trial Summary

Primary Objective: To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset Pompe disease 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.


Clinical Trial Description

The duration of the study for each participant will be up to approximately 7 years, and consists of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations. This is followed by a 25-week treatment period and an up to 346-week treatment extension period and a 4-week post-treatment observation period. Cohort 1 and 2 (Cohort 1: avalglucosidase alfa 20 milligrams per kilogram [mg/kg], Cohort 2: avalglucosidase alfa 40 mg/kg) will be non-randomized and Cohort 3 (Cohort 3a: avalglucosidase alfa 40 mg/kg [maximum tolerated dose] and Cohort 3b: alglucosidase alfa) will be randomized. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03019406
Study type Interventional
Source Sanofi
Contact
Status Active, not recruiting
Phase Phase 2
Start date October 12, 2017
Completion date June 15, 2026