Trial to Assess the Efficacy of Midostaurin (PKC412) in Patients With c-KIT or FLT3-ITD Mutated t(8;21) AML

• Status: Completed
• Trial end date: 2019-10-30
• Target enrollment: 0
• Participant gender: All

Summary

To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML. To assess the efficacy of midostaurin depending on the type of c-KIT mutation

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Technische Universität Dresden
• Collaborator: Novartis Pharmaceuticals
• Treatments: 4'-N-benzoylstaurosporine; Midostaurin; Staurosporine

Criteria

Inclusion Criteria:

• Diagnosis of c-KIT mutated t(8;21) AML i.e.

1. >20% myeloid blasts in bone marrow and/or peripheral blood at initial diagnosis

2. Plus cytogenetic diagnosis of aberration t(8;21)/AML1-ETO

3. Plus mutation of c-KIT gene (mut-KIT17 or mut-KIT8) or FLT3-ITD mutation or both c-KIT and FLT3-ITD mutations

• Chemoresponsive disease as determined by early bone marrow assessment on day 14-16 after first cycle of induction therapy with cytarabine in combination with daunorubicine or idarubicine, or mitoxantrone- Fit for further intensive chemotherapy

• Age 18-65 years

• ECOG performance status of 0-2

• Life expectancy of at least 12 weeks

Exclusion Criteria:

• Primary refractory or previously relapsed AML

• Non-eligibility for high-dose cytarabine based consolidation, e.g. intolerance to cytarabine

• Inability to swallow oral medications

• Symptomatic congestive heart failure

• Bilirubin >2.5 x upper limit of normal