5 results
Aim of this project is to diagnose hearing impairment, and underlying disorders in speech perception and production, and to develop and implement adequate intervention techniques, which are subsequently evaluated. The ultimate goal is to prevent…
Primary:To evaluate the efficacy of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN) after 12 weeks of treatment.Secondary:• To evaluate…
The objectives of the study are to assess safety and tolerability, pharmacokinetics (PK), and biological activity (including efficacy and pharmacodynamics [PD]) of multiple subcutaneous doses of pegcetacoplan in pediatric patients with paroxysmal…
Objectives:Primary: To evaluate the long-term safety of IVT injected pegcetacoplanSecondary:1. To assess changes in the total area of geographic atrophy (GA) lesion in the study eye measured by fundus autofluorescence (FAF).2. To assess changes in…
The Primary Objectives of the Phase 1b are: • To assess the safety and tolerability of carfilzomib, alone and in combination with induction chemotherapy, for the treatment of children with relapsed or refractory ALL • To determine the maximum…