5 results
Primary ObjectiveThe primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.Secondary ObjectivesThe secondary…
The primary objective is to compare the efficacy of plitidepsin in combination with dexamethasone vs. dexamethasone alone as measured by progression-free survival (PFS) in patients with relapsed/refractory multiple myeloma (MM). Secondary objectives…
The primary objective of this study is to evaluate whether treatment with nesiritide improves patient outcomes (as measured by reduction in the composite of HF rehospitalization and all-cause mortality through 30 days after randomization [Day 30])…
The study will look at patients with relapsed (returned after prior treatment) and refractory (not responsive to prior treatment) multiple myeloma. The research aims to compare a new drug called elotuzamab combined with standard of care (…
Primary Objective** To compare the PFS of melflufen plus dexamethasone (Arm A) versus pomalidomide plus dexamethasone (Arm B) as assessed by the Independent Review Committee (IRC) according to the International Myeloma Working Group Uniform Response…