Clinical Trials Directory

Trials / Completed

CompletedNCT00233454

Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia

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)

Status
Completed
Phase
Phase 2
Study type
Interventional
Enrollment
26 (actual)
Sponsor
Jason Robert Gotlib · Academic / Other
Sex
All
Age
18 Years
Healthy volunteers
Not accepted

Summary

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)

Detailed description

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.

Conditions

Interventions

TypeNameDescription
DRUGMidostaurinMidostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR

Timeline

Start date
2005-03-01
Primary completion
2010-06-18
Completion
2011-04-16
First posted
2005-10-05
Last updated
2018-09-20
Results posted
2015-07-09

Locations

3 sites across 1 country: United States

Source: ClinicalTrials.gov record NCT00233454. Inclusion in this directory is not an endorsement.