6 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.…
Primary Objective:To evaluate the effect of INS1007 compared with placebo on time to first pulmonary exacerbation over the 24-week treatment period.Secondary Objectives:1. To evaluate the effect of INS1007 compared with placebo on quality of life (…
• To assess feasibility and safety of a sequential treatment regime in which standard intensive chemotherapy (fludarabin-amsacrin-cytarabin) is directly followed by standard allogeneic stem cell transplantation (T cell depleted RIC alloSCT with…
Main objective of the trial is to determine the effect of individualizing the immunosuppressive therapy based on baseline immune-risk stratification according to 2 new biomarkers (d-sp ELISPOT IFN-γ and donor/recipient HLA Eplet Mismatch), in a…
Primary* To evaluate the effect of EDP1066 in multiple formulations on the systemic immune system.Secondary* To evaluate the safety and tolerability of EDP1066 in multiple formulations.
1. To evaluate the effect of brensocatib at 10 mg and 25 mg compared with placebo on the rate of pulmonary exacerbations (PEs) over the 52-week treatment period