4 results
- To investigate the biochemical response of ITF2984, defined as a reduction in (random) GH < 1.0 mcg/l and/or normalization of IGF-1.- To investigate the biochemical response of ITF2984, defined as a reduction of GH to no more than 2.5 mcg/l…
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: To investigate the efficacy and safety of octreotide treatment (a somatostatin analogue) in decreasing the transfusion requirements (IV iron infusions and / or red blood cell transfusions) in patients with GI bleeding caused by…
Currently, there are no curative treatment options for patients with Waldenstrom*s macroglobulinemia (WM) and there is no consensus on standard first line or relapse treatment. The proteasome inhibitor bortezomib has shown efficacy in WM, but is…