2 results
Approved WMOCompleted
Primary objective: - To evaluate the efficacy of VX-445/TEZ/IVA in CF subjects who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).Secondary objectives: - To evaluate the safety of VX-445/TEZ/IVA- To…
Approved WMORecruiting
Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.Part 2: To compare the efficacy, in terms of red blood cell (RBC)…