Patients with newly diagnosed c-KIT or FLT3-ITD mutated t(8
Conditions
Interventions
Trade Name: Rydapt
Product Name: Midostaurin
Product Code: PKC412
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Midostaurin
Sponsors
Technische Universität Dresden
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Diagnosis of c-KIT and/or FLT3-ITD mutated t(8;21) AML i.e. o >20% myeloid blasts in bone marrow and/or peripheral blood at initial diagnosis o Plus cytogenetic diagnosis of aberration t(8;21)/AML1-ETO o Plus mutation of c-KIT gene (mut-KIT17 or mut-KIT8) or FLT3-ITD mutation or both c-KIT and FLT3-ITD mutations • Chemo-responsive disease* as determined by early bone marrow assessment on day 14-16 after first cycle of standard induction therapy with seven-day continuous infusion of 100-200 mg/m2 cytarabine per day in combination with three doses of daunorubicine, or idarubicine, or mitoxantrone • Age 18 – 65 years • Fit for intensive chemotherapy as assessed by o Adequate liver and renal function as assessed by the following laboratory requirements to be conducted within 7 days prior to screening: ? Total bilirubin = 1.5 times the upper limit of normal ? ALT and AST = 2.5 times upper limit of normal ? Creatinine = 1.5 times upper limit of normal o Adequate cardiac function, i.e. left ventricular ejection fraction (LVEF) of >= 50% as assessed by transthoracal twodimensional echocardiography (“M Mode”) or MUGA scan • ECOG performance status of 0-2 • Life expectancy of at least 12 weeks • Subject's written informed consent has been obtained • Legal capacity (see also exclusion criterion) Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 18
Exclusion criteria
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 as defined by left ventricular ejection fraction (LVEF) of =50% as assessed by transthoracal twodimensional echocardiography (“M Mode”) or MUGA scan - Subject without legal capacity who is unable to understand the nature, significance and consequences of the study - Investigational drug therapy outside of this trial during or within 4 weeks of study entry - Known or persistent abuse of medication, drugs or alcohol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML ;Secondary Objective: Not applicable;Primary end point(s): 2-year event-free Survival;Timepoint(s) of evaluation of this end point: evaluation during study and in follow-up visits up to 2 year after study entry | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Time to relapse (TTR), Cumulative incidence of relapse (CIR), Overall survival (OS), Relapse-free survival (RFS), morphologic and molecular CR rate, incidence of AEs/SAEs, MRD kinetics;Timepoint(s) of evaluation of this end point: evaluation during study and in follow-up visits up to 2 year after study entry | — |
Countries
Germany
Contacts
Public ContactHead of Clinical Trial Dept.
Technische Universität Dresden
Outcome results
None listed