6 results
Main objective: To assess the effect of CK-2127107 versus placebo on respiratory function in patients with ALS.
Primary Objective:* The primary objective is to demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST),…
Primary: • The primary objective is to determine adjudicated ORR (CR/CR with partial recovery of peripheral blood counts [CRh] + PR + CI) based on modified IWG-MRT-ECNM consensus response criteria in patients with AdvSM treated with avapritinib and…
To assess the long-term safety and tolerability of reldesemtiv in patients with ALS
Primary: Part 1 • To determine the RP2D of avapritinib in patients with ISM for use in Part 2 and Part 3 of the study.Part 2 • To determine main change in ISM-SAF TSS from baseline to C7D1, compared to placebo. Part 3 • To assess the long-term…
The purpose of this study is to evaluate whether the new drug reldesemtiv is effective and safe in patients with ALS. The primary objective is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.The secondary objectives…