NCT01288027

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in participants with Late-Onset Pompe disease. The secondary objectives are to characterize the disease burden in participants with late-onset Pompe disease and explore imaging, histologic, and functional assessments in these participants and to explore potential plasma or urine biomarkers relative to late-onset Pompe disease and participant's response to treatment with alglucosidase alfa (Myozyme®/Lumizyme®/GZ419829).

Conditions

• Pompe Disease (Late-Onset)
• Glycogen Storage Disease Type II (GSD II)
• Glycogenesis 2 Acid Maltase Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Alglucosidase Alfa	Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.

Timeline

• Start date: 2011-06-01
• Primary completion: 2013-12-01
• Completion: 2013-12-01
• First posted: 2011-02-02
• Last updated: 2014-12-19
• Results posted: 2014-12-02

Locations

15 sites across 4 countries: United States, Germany, Netherlands, United Kingdom