drugset / Trial / NCT04817670
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
RandomizedParallel-groupDouble-blindTreatment
Summary
The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD). The safety, tolerability and clinical beneficial effects of VIT-2763 for the treatment of SCD are also explored.
Timeline
- Start
- 2021-11-18
- Primary completion
- 2024-03-07
- Completion
- 2024-03-07
Publications
- Background Nyffenegger N, Zennadi R, Kalleda N, Flace A, Ingoglia G, Buzzi RM, Doucerain C, Buehler PW, Schaer DJ, Durrenberger F, Manolova V. The oral ferroportin inhibitor vamifeport improves hemodynamics in a mouse model of sickle cell disease. Blood. 2022 Aug 18;140(7):769-781. doi: 10.1182/blood.2021014716.
- Parrow NL, Doherty JM, Conrey A, Thein SL, Fleming RE. Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease. Adv Hematol. 2024 Dec 3;2024:9872440. doi: 10.1155/ah/9872440. eCollection 2024.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Vamifeport | Small molecule | 60 mg | Oral |
| Subject | Vamifeport | Small molecule | 120 mg | Oral |