A single arm, phase II, open-label study to determine the efficacy of 100mg twice daily oral dosing of Midostaurin administered to patients with Agressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease
- Conditions
- Agressive Systemic Mastocytosis10024324
- Registration Number
- NL-OMON37318
- Lead Sponsor
- ovartis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 3
Adult patients with diagnosis of one major plus at least one minor criterion, or the presence of at least 3 minor criteria according to WHO criteria for Systemic Mastocytosis (Valent et al. 2001);Patient must present with at least one measurable C-Finding.;For patients with MCL: bone marrow aspirate smears must show 20% or more immature mast cells.
Patients who have demonstrated relapse to more than two prior regimen of SM treatment-regardless of treatment regimen for supportive care (e.g. symptom limiting therapies);Patients who have aggressive systemic mastocytosis with eosinophilia and known positivity for the FIP1L1-PDGFR fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy;Patients on imatinib therapy and known to be KIT D816V negative unless they have demonstrated relapse, resistance or intolerance to imatinib.;Patients with any pulmonary infiltrate including those suspected to be of infectious origin.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To determine the efficacy of midostaurin in patients with ASM or MCL with or<br /><br>without an AHNMD when administered orally at a dose of 100 mg b.i.d.<br /><br>continuously as measured by overall response rate.</p><br>
- Secondary Outcome Measures
Name Time Method <p>To evaluate the duration of response<br /><br>To evaluate the time to response<br /><br>To evaluate overall survival<br /><br>To evaluate the safety and tolerability of midostaurin in patients with ASM or<br /><br>MCL (with or without an associated hematological clonal non-mast cell lineage<br /><br>disease).<br /><br>To characterize t he KIT mutational status at baseline and after 6 cycles of<br /><br>therapy and evaluate potential associations with efficacy outcomes.</p><br>