A listing of Thalassemia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
This is a Phase 2 open-label, single-arm design with dose escalation by subject cohort and with the potential for individual titration (dose increase or decrease) within each cohort. Five PTG-300 dose levels/regimens are planned to be tested for each subpopulation of beta-thalassemia (NTD and TD) on separate arms: Cohort 1: …
To compare the effect of mitapivat versus placebo on transfusion burden in subjects with alpha- or beta-transfusiondependent thalassemia (TDT) Transfusion reduction response (TRR), defined as a 50% reduction in transfused red blood cell (RBC) units with a reduction of greater that or equal to 2 units of transfused RBCs in …
The purpose of this research study is to: 1) evaluate how well IMR-687 is tolerated in subjects with β-thalassemia and how safe it is, 2) measure the amount of IMR-687 in your blood and 3) evaluate what the drug does to the red blood cells and other blood markers associated …