3 results
Establishment of treatment area dose at which 90% technical success is achieved. Technical success will be defined as * 120Gy calculated radiation absorbed dose to the target area, i.e. the hyperaemic zone surrounding the area of post-RFA…
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…
Primary Objective:* The primary objective is to demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST),…