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…
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 primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) in participants with the neuronopathic form of mucopolysaccharidosis type II (nMPS…
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…