NCT02032524

Avalglucosidase Alfa Extension Study

An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety and Pharmacokinetics of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) in Patients With Pompe Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Primary Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic variables

Detailed description

The planned duration of the study for each participant was initially 6 years. Each participant continued with the study until the participant withdrew, the Investigator withdrew the participant, or the Sponsor terminated the study. An additional follow-up phase began after the participant has completed the 6-year study period, and lasted until avalglucosidase alfa was approved in the participant's country, except in the United Kingdom (UK), Germany and Denmark, where the duration of the additional follow-up phase was up to the approval in the country or limited to a maximum of 2 years, whichever occurred first (ie, for participants in the UK, Germany and Denmark, the total study duration per participant was 8 years at the maximum including the initial 6-year period and the additional 2-year follow-up).

Conditions

• Glycogen Storage Disease Type II Pompe Disease

Interventions

Type	Name	Description
DRUG	Avalglucosidase Alfa	Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous

Timeline

• Start date: 2014-02-27
• Primary completion: 2022-12-12
• Completion: 2022-12-12
• First posted: 2014-01-10
• Last updated: 2024-03-01
• Results posted: 2024-03-01

Locations

17 sites across 7 countries: United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom