Conditions: Leukemia
Keywords: Aggressive systemic mastocytosis mast cell leukemia C-Findings tyrosine kinase inhibitor KIT mutation AHNMD Systemic Aggressive
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 116
Healthy volunteers: no

Brief summary:
The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated Hematological clonal Non-Mast cell lineage Disease (AHNMD).

Detailed description:

Arms:
arm 1: Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.

Interventions: intervention 1: Midostaurin (PKC412)
Intervention descriptions: intervention 1: Midostaurin was provided as 25 mg soft gelatin capsules for oral administration.

Protocol document: