Open-label Trial of Midostaurin in Aggressive Systemic Mastocytosis
Research type
Research Study
Full title
A single arm phase II, open-label study to determine the efficacy of 100 mg twice daily oral dosing of midostaurin administered to patients with Agressive Systemic Mastocytosis or Mast Cell Leukemia +/- an assoicated hematological clonal non-mast cell lineage
IRAS ID
14080
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2008-000280-42
ISRCTN Number
N/A
Research summary
Midostaurin (PKC412) is an investigational drug which is being developed for the treatment of rare types of leukaemia. Preclinical studies have shown that midostaurin has strong inhibitory activity on some types of neoplastic mast cells. Preliminary results of an ongoing phase II trial in aggressive systemic mastocytosis (ASM) revealed high overall response rates of approximately 73 % in 15 patients. This open-label study has been designed to more fully assess the safety and effectiveness of midostaurin in patients with ASM or mast cell leukaemia. Initially, 40 patients will be given 100 mg midostaurin twice-a-day in continuous 4 week cycles. Patients will continue treatment until there is disease progression, intolerable toxicity or the patient withdraws. Data on safety and the overall response to treatment will be collected after each cycle for the first 12 cycles and thereafter after every 3rd cycle. When 40 patients have completed 6 cycles (or earlier if the there is sufficient information) the overall response rate will be determined, and if response criteria are met, then a further 20 patients will be enrolled. All patients will be followed up for survival.The second stage analysis will be performed when the final 20 patients have completed 12 cycles of treatment or have discontinued treatment due to progression, intolerable toxicity or have withdrawn.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
09/H1208/3
Date of REC Opinion
9 Apr 2009
REC opinion
Further Information Favourable Opinion