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Miglustat for Infantile Sandhoff and Tay-Sachs Diseases
NCT03822013Phase 3Terminated
- Sponsor
- Tehran University of Medical Sciences
- Start
- Jan 2019
- Primary completion
- Sep 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label trial of miglustat in Sandhoff disease and Tay-Sachs disease (TSD) (2 arms), sponsored by Tehran University of Medical Sciences. Primary endpoints: Hospitalization frequency change at 1 year, Pneumonia aspiration frequency change at 1 year and 3 more. Started Jan 2019; status: terminated.
02
Drugs
miglustat
03
Conditions
Sandhoff diseaseTay-Sachs disease (TSD)
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