2 results
Approved WMOCompleted
We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…
Approved WMOCompleted
Primary Objective:Part 1:- To determine the MTD and recommended phase 2 dose (RP2D) of avapritinib.- To determine the safety and tolerability of avapritinib.Part 2:* To determine the overall response rate (ORR) by RECIST criteria at the MTD/RP2D of…