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Ambroxol for Type 1 Gaucher Disease
NCT03950050Phase 2Completed
- Sponsor
- Shaare Zedek Medical Center
- Start
- Mar 2019
- Primary completion
- Nov 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ambroxol in Gaucher disease type 1 (GD1), sponsored by Shaare Zedek Medical Center. Primary endpoints: Platelets count at 12 months, Bone mineral density at 12 months and 1 more. Started Mar 2019; status: completed.
02
Drugs
ambroxol
03
Conditions
Gaucher disease type 1 (GD1)
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