Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

Summary

This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent Beta-thalassemia (NTDT).

Timeline

Start

2020-06-11

Primary completion

2021-10-11

Completion

2021-11-03

Publications

• Kattamis A, Taher A, Viprakasit V, Levin C, Hermosilla R, Szecsody P, Richard F, Cappellini MD, Porter J. Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent beta-thalassemia: results from a randomized phase 2a study. Orphanet J Rare Dis. 2025 Nov 25;20(1):608. doi: 10.1186/s13023-025-04119-y.

Drugs

Indications