2 results
Approved WMOCompleted
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…
Approved WMORecruiting
Phase 1Primary Objective• To determine the RP2D and, if applicable, the maximum tolerated dose (MTD) of NVL-520 in patients with advanced ROS1-positive solid tumorPhase 2Primary Objective• To evaluate the ORR of NVL-520 at the RP2D in patients with…