Daunorubicin, Cytarabine, and Midostaurin in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

Not Recruiting

Trial ID: NCT00651261


The purpose of this study is to compare the effects, good and/or bad, of a standard chemotherapy regimen for AML that includes the drugs daunorubicin and cytarabine combined with or without midostaurin (also known as PKC412), to find out which is better. This research is being done because it is unknown whether the addition of midostaurin to chemotherapy treatment is better than chemotherapy treatment alone. Midostaurin has been tested in over 400 patients and is being studied in a number of illnesses, including AML, colon cancer, and lung cancer. Midostaurin blocks an enzyme, produced by a gene known as FLT3, that may have a role in the survival and growth of AML cells. Not all leukemia cells will have the abnormal FLT3 gene. This study will focus only on patients with leukemia cells with the abnormal FLT3 gene.

Official Title

A Phase III Randomized, Double-Blind Study of Induction (Daunorubicin/Cytarabine) and Consolidation (High-Dose Cytarabine) Chemotherapy + Midostaurin (PKC412) (IND #101261) or Placebo in Newly Diagnosed Patients < 60 Years of Age With FLT3 Mutated Acute Myeloid Leukemia (AML)

Stanford Investigator(s)

Jason Gotlib

Professor of Medicine (Hematology)

Caroline Berube
Caroline Berube

Clinical Associate Professor, Medicine - Hematology


1. Documentation of Disease:

      - Unequivocal diagnosis of AML ( > 20% blasts in the bone marrow based on the WHO
      classification), excluding M3 (acute promyelocytic leukemia). Patients with
      neurologic symptoms suggestive of CNS leukemia are recommended to have a lumbar
      puncture. Patients whose CSF is positive for AML blasts are not eligible.

      - Documented FLT3 mutation (ITD or point mutation), determined by analysis in a
      protocol- designated FLT3 screening laboratory.

   2. Age Requirement:

      - Age ≥ 18 and < 60 years

   3. Prior Therapy:

      - No prior chemotherapy for leukemia or myelodysplasia with the following

         - emergency leukapheresis

         - emergency treatment for hyperleukocytosis with hydroxyurea for ≤ 5 days

         - cranial RT for CNS leukostasis (one dose only)

         - growth factor/cytokine support

      - AML patients with a history of antecedent myelodysplasia (MDS) remain eligible
      for treatment on this trial, but must not have had prior cytotoxic therapy (e.g.,
      azacitidine or decitabine)

      - Patients who have developed therapy related AML after prior RT or chemotherapy
      for another cancer or disorder are not eligible.

   4. Cardiac Function: Patients with symptomatic congestive heart failure are not eligible.

   5. Initial Laboratory Value: Total bilirubin < 2.5 x ULN (Upper Limit of Normal)

   6. Pregnancy and Nursing Status:

      - Non-pregnant and non-nursing due to the unknown teratogenic potential of
      midostaurin in humans, pregnant or nursing patients may not be enrolled.

      - Women of childbearing potential must have a negative serum or urine pregnancy
      test within a sensitivity of at least 50 mIU/mL within 16 days prior to

      - Women of child-bearing potential must either commit to continued abstinence from
      heterosexual intercourse or commit to TWO acceptable methods of birth control:

         - one highly effective method (eg, IUD, hormonal (non-oral contraceptive),
         tubal ligation, or partner's vasectomy) and

         - one additional effective method (e.g., latex condom, diaphragm or cervical

      - The two acceptable methods of birth control must be used AT THE SAME TIME, before
      beginning midostaurin/placebo therapy and continuing for 12 weeks after
      completion of all therapy.

      - Note that oral contraceptives are not considered a high effective method because
      of the possibility of a drug interaction with midostaurin.

      - Women of childbearing potential is defined as a sexually active mature woman who
      has not undergone a hysterectomy or who has not had menses at any time in the
      preceding 24 consecutive months.

      - Men must agree not to father a child and must use a latex condom during any
      sexual contact with women of childbearing potential while taking
      midostaurin/placebo and for 12 weeks after therapy is stopped, even if they have
      undergone a successful vasectomy.


other: placebo

drug: cytarabine

drug: midostaurin

drug: daunorubicin

drug: dexamethasone acetate

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Cancer Clinical Trials Office

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