Hematological Non-mast Cell Lineage Disease (AHNMD) Clinical Trial
Official title:
A Single Arm, Phase 2, Open-Label Study to Determine the Efficacy of Twice Daily Oral Dosing of PKC412 <Midostaurin> Administered to Patients With Aggressive Systemic Mastocytosis (ASM) and Mast Cell Leukemia (MCL)
The safety and efficacy of midostaurin (PKC412), a novel investigational drug, will be evaluated on the basis of response rate, when administered to patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL)
This study assesses the activity and safety profile of twice-daily oral doses of midostaurin
in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) with or
without associated clonal hematological non-mast cell lineage disease (AHNMD).
Aggressive systemic mastocytosis (ASM) and mast cell leukemia (MCL) are characterized by
excessive bone marrow production of mast cells which can can infiltrate tissues and release
harmful substances, resulting in organ damage. These diseases have very limited treatment
options and poor prognosis. Existing treatments for in advanced mast cell disease, eg,
interferon-alpha; corticosteroids; and/or cladribine, exhibit low response rates that are
usually partial in nature.
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