NCT02782741

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 101 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa. Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).

Detailed description

The duration of the study per participant will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to less than (\<) 18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

Conditions

• Glycogen Storage Disease Type II;Pompe's Disease

Interventions

Type	Name	Description
DRUG	Avalglucosidase alfa (GZ402666)	Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
DRUG	Alglucosidase alfa (GZ419829)	Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

Timeline

• Start date: 2016-11-02
• Primary completion: 2020-03-19
• Completion: 2023-05-31
• First posted: 2016-05-25
• Last updated: 2024-04-04
• Results posted: 2021-04-08

Locations

69 sites across 25 countries: United States, Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, Denmark, France, Germany, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russia, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study