9 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.…
The primary objective of this study is to assess the negative predictive value of a Skin Prick Test protocol in subjects with clinical CMA and/or RA.
The primary objective of this exploratory study is to test if prophylactic use of conestat alfa decreases the frequency of angioedema attacks in patients with InH-AAE. As secondary objectives, effects on disease severity, quality of life and drug…
Primary Objective:• To evaluate the proportion of patients with a response of very good partial response (VGPR) or better to IDd treatment.Secondary Objectives:• To measure progression-free survival (PFS), time to progression (TTP), and overall…
Primary: To evaluate the tolerability and safety of the treatment with rhC1INH (conestat alfa) on top of Standard Care, for patients with pre-eclampsia.Secondary: To evaluate the efficacy of treatment with rhC1INH (conestat alfa) on top of Standard…
Primary objective:To compare the effect of ixa+dex versus pom+dex on progression-free survival (PFS) in patients with relapsed and/or refractory multiple myeloma (RRMM) who have received at least 2 prior lines of therapy, including lenalidomide and…
Primary objective* To determine the efficacy, defined as overall response rate (ORR; >= partial response (PR)), of 9 cycles of ixazomib, daratumumab and low dose dexamethasoneSecondary objectives* To determine the tolerability, defined as…
Primary objective: To evaluate the effect of navitoclax in combination with ruxolitinib on splenomegaly response when compared to ruxolitinib in subjects with myelofibrosis.Secondary objectives:• To evaluate the effect of navitoclax in combination…
Study ObjectivesPrimary:• To determine the effect of ixazomib maintenance therapy on progression-free survival (PFS), compared to placebo, in patients with NDMM who have had a response (complete response [CR], very good partial response [VGPR], or…