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

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 - AgressiveSystemicMastocytosis (ASM) or MastCell Leukemia (MCL), midostaurin

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON37318

Enrollment

Registered

2008-06-10

Start date

2009-01-26

Completion date

Unknown

Last updated

2024-05-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Agressive Systemic Mastocytosis

Interventions

Patients will be treated with twice daily 100 mg midostaurin. A cycles is 28 days. Patients take their medication continuously.

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 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.

Exclusion criteria

Exclusion criteria: 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.

Design outcomes

Primary

Measure	Time frame
To determine the efficacy of midostaurin in patients with ASM or MCL with or without an AHNMD when administered orally at a dose of 100 mg b.i.d. continuously as measured by overall response rate.	—

Secondary

Measure	Time frame
To evaluate the duration of response To evaluate the time to response To evaluate overall survival To evaluate the safety and tolerability of midostaurin in patients with ASM or MCL (with or without an associated hematological clonal non-mast cell lineage disease). To characterize t he KIT mutational status at baseline and after 6 cycles of therapy and evaluate potential associations with efficacy outcomes.	—

Measure

Time frame

To evaluate the duration of response To evaluate the time to response To evaluate overall survival To evaluate the safety and tolerability of midostaurin in patients with ASM or MCL (with or without an associated hematological clonal non-mast cell lineage disease). To characterize t he KIT mutational status at baseline and after 6 cycles of therapy and evaluate potential associations with efficacy outcomes.

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)