3 results
Approved WMOCompleted
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Approved WMOCompleted
Phase IPrimary Objective- To define the recommended phase II dose (RP2D) of selumetinib/dexamethasone combination in adult and paediatric patients with relapsed/refractory, RAS pathway mutant ALLSecondary Objectives- To evaluate safety and…
Approved WMOCompleted
Primary objectives:- To determine the PK of selumetinib after administration of the selumetinib granule formulation. - To assess the safety and tolerability of the selumetinib granule formulation.