A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease
- Registry ID
- EUCTR2013-001768-48
- Source registry
- EUCTR
- Status
- Prematurely Ended
- Phase
- PHASE3
- Sponsor
- BioMarin Pharmaceutical Inc
- Start date
- 2013-11-27
- Completion date
- 2016-06-22
- Last update
- 2026-08-16
Conditions
Summary
The primary objective of the study is to determine if treatment with BMN 701 results in an increase in MIP measured at the mouth by the Mueller maneuver from Baseline to Week 24 in subjects with late-onset Pompe disease previously treated with rhGAA.
The primary objective of the extension period of the study is to evaluate the long-term safety and efficacy of BMN 701 in subjects with late-onset Pompe disease previously treated with rhGAA.
Locations
- , , Germany
- , , Austria
- , , Portugal
- , , Belgium
- , , Italy
- , , UK
View on source registry