6 results
Primary Objective• To compare the anti-tumor efficacy of oral single-agent rociletinib, as measured by investigator assessment of the progression-free survival (PFS), with that of single-agent cytotoxic chemotherapy in patients with EGFR mutated,…
Primary Objective:To compare the efficacy of pacritinib with that of Best Available Therapy (BAT) in patients with PMF, PPV-MF, or PET-MF; the efficacy measure for this analysis is the proportion of patients achieving a * 35% reduction in spleen…
The primary objective is to compare the efficacy of two dose-schedule arms(s) of pacritinib (pooled once daily [QD] and twice-daily [BID] dosing arms) with that of best available therapy (BAT) in patients with thrombocytopenia and primary…
Improve allo-SCT transplant outcome using a uniform conditioning regimen and pacritinib pretreatment by means of the proportion of patients with a failure within 6 months post-transplant. Events that are considered a failure are: primary graft…
To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…
The Primary objective is to confirm the maintenance of vigilance with SENS-111.Secondary objectives are:* to confirm the maintenance of working memory, and cognitive function; * to confirm the pharmacodynamics effect of SENS-111 on symptoms…