11 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…
This is a phase I/II pediatric dose-ranging study that will evaluate the safety, tolerability and pharmacokinetics of midostaurin in children <18 years of age and >= 3 months who have relapsed or refractory leukemias that may benefit…
To determine if the addition of midostaurin to daunorubicin/ cytarabine induction, high dose cytarabine consolidation, and continuation therapy improves overall survival (OS) in both the mutant FLT3-ITD and FLT-3 TKD AML patients.
The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
Objective of this study is to determine the efficacy of midostaurin in patients with ASM or MCL with/without an associated hematological clonal non-mast cell lineage disease.
Primary objective:To asses the safety and feasibility of post-transplant panobinostat combined with midostaurin in patients with adverse risk AML/RAEB with FLT3-ITD with high allelic ratio in terms of dose limiting toxicity.Secondary objectives:To…
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.
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…
To compare OS between gilteritinib and midostaurin in combination with induction therapy and consolidation therapy followed by one-year maintenance therapy in patients with newly diagnosed AML with a FLT3 gene mutation eligible for intensive…
Primary objectives:- To assess in a randomized comparison the effect of midostaurin added to 10-day decitabine treatment on the cumulative CR/CRi rate during 3 cycles.Secondary objectives:- To assess the safety and tolerability of midostaurin added…