5 results
To assess the anti-tumor activity of LGX818/MEK162 in combination with third targeted agents after progression on LGX818/MEK162 combination therapy.
Primary objective:To asses the safety and feasibility of post-transplant panobinostat combined with midostaurin in patients with adverse risk AML/RAEB with FLT3-ITD with high allelic ratio in terms of dose limiting toxicity.Secondary objectives:To…
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…
Primary: To assess best overall response rate (ORR) up to 8 cycles. Secondary: ORR, complete response (CR), very good partial response (VGPR), progression free survival (PFS), overall survival (OS), safety, PK, exposure-response (efficacy and safety…
Primary objective* To determine the efficacy of panobinostat maintenance therapy versus standard of care administered to patients with high-risk MDS or AML in complete hematologic remission after an allogeneic hematologic stem cell transplantation (…