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Sirolimus pilot trial in transfusion-dependent beta-thalassemia
2018-001942-33Phase 2Completed
- Sponsor
- Rare Partners
- Start
- Aug 2018
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sirolimus in beta thalassemia, sponsored by Rare Partners. Primary endpoint: HbF level in peripheral blood at day 360 compared to day 0. Started Aug 2018; status: completed.
02
Drugs
sirolimus
03
Conditions
beta thalassemia
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