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Zavesca for Anti-rhGAA Immune Response in Pompe Disease
NCT02185651Phase 1Terminated
- Sponsor
- University of Florida
- Start
- Oct 2016
- Primary completion
- Jul 2018
- Modality
- Small Molecules
01
Overview
Phase 1, open-label trial of miglustat in Pompe disease (2 arms), sponsored by University of Florida. Primary endpoint: Pharmacodynamics of ERT with pre-medication Zavesca at Week 6. Started Oct 2016; status: terminated.
02
Drugs
miglustat
03
Conditions
Pompe disease
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