4 results
Primary: To study the efficacy and tolerability of Midostaurin in patients with indolent or smoldering systemic mastocytosis on mediator symptom reduction.Secondary: 1) To study whether Midostaurin can reduce mast cell infiltration and 2) to assess…
Assess the neurological efficacy of ambroxol in adults and children with GD3.
Primary: To determine the MTD and/or RDE of LGH447 with or without midostaurin. Secondary: 1. To characterize the safety and tolerability of LGH447 with or without midostaurin at the MTD and/or RDE.2. To assess any observed antitumor activity of…
The purpose of this study is to obtain blood samples from up to 14 subjects who previously received namilumab in the previous study M1-1188-002-EM (PRIORA, [NCT01317797]) to correlate genetic markers with clinical outcomes.