4 results
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.…
Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…
Part A: Determine safety and tolerability of FTX-1821 in healthy subjects. Part B and C: Determine safety and tolerability of FTX-1821 in FSHD patients.
Main objective:The main objective is to evaluate the efficacy of two intensified consolidation strategies in very-high risk neuroblastoma (VHR-NBL) patients in terms of event-free survival from randomisation date. Thisevaluation will follow a…