A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa.
- Registry ID
- EUCTR2010-020611-36
- Source registry
- EUCTR
- Status
- Completed
- Phase
- PHASE4
- Sponsor
- Genzyme Europe B.V.
- Start date
- 2011-06-01
- Completion date
- 2013-12-19
- Last update
- 2026-08-16
Conditions
- Pompe disease (acid alpha-glucosidase deficiency)
Summary
The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.
Locations
- , , Germany
- , , Netherlands
- , , UK
View on source registry