Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients with FLT3-ITD Mutated AML

  • Study type:

    Interventional What is this?

    Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Study phase:

    II What is this?

    During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

Study IDs

  • Site IRB:

    • Rochester, Minnesota: 13-002979
  • NCT ID:

    NCT01883362
  • Sponsor Protocol Number:

    CPKC412AUS23

About this study

To determine if the addition of midostaurin (PKC412) to Standard of Care (SOC) therapy reduces relapse in FLT3-ITD mutated AML patients receiving an allogenetic hematopoietic stem cell transplant.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Patients must be between 18 and 60 years of age
  • Patients must have an ECOG Performance Status of < 2
  • Patients must have a documented Unequivocal diagnosis of AML according to WHO 2008 classification (>20% blasts in the bone marrow), excluding M3 (acute promyelocytic leukemia).
  • Patients must have a documented FLT3 ITD mutation, determined by local laboratory for eligibility (historical tissue will be requested for central analysis confirmation)
  • Patients who have undergone allogeneic HSCT in CR1 from a matched related or matched unrelated donor. All of the following criteria must also be met:
    • HLA typing to include available 8/8 or 7/8 allele HLA matched donor (at A,B,C, DRB1) Single allelic mismatch allowed
  • Patients who received a conditioning regimen which included one of the following:
    • Busulfan/Fludarabine (Bu/Flu)
      • Busulfan (16 mg/kg PO or 12.8 mg/kg IV)
      • Fludarabine (120-180 mg/m2)
    • Fludarabine / Melphalan (Flu/Mel)
      • Fludarabine (120-180 mg/m2)
      • Melphalan (≤ 150 mg/m2)
    • Busulfan/Cyclophosphamide (Bu/Cy)
      • Busulfan (16 mg/kg PO or 12.8 mg/kg IV)
      • Cyclophosphamide (120 mg/kg)
    • Cyclophosphamide/Total Body Irradiation (Cy/TBI)
      • Cyclophosphamide (120 mg/kg)
      • TBI (1200-1420 cGy)
    • Recovery of counts by day 42 and able to start midostaurin by day 60 post-HSCT (first dose of midostaurin to start no earlier than 28 days post-HSCT); ANC >1000µL, platelets ≥20,000 without platelet transfusion

Exclusion Criteria:

  • Patients whom have failed prior attempts at allogeneic HSCT
  • Patients who have received an autologous transplant
  • Patients with Acute GVHD Grade III-IV
  • Patients with a known confirmed diagnosis of HIV infection or active viral hepatitis.
  • Impaired cardiac function including any of the following:
    • Screening ECG with a QTc > 450 msec. If QTc > 450 and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient rescreened for QTc.
    • Patients with congenital long QT syndrome
    • History or presence of sustained ventricular tachycardia
    • Any history of ventricular fibrillation or torsades de pointes
    • Bradycardia defined as HR. < 50 bpm
    • Right bundle branch block + left anterior hemiblock (bifascicular block)
    • Patients with myocardial infarction or unstable angina < 6 months prior to starting study
    • Congestive Heart Failure NY Heart Association class III or IV
    • Patients with an ejection fraction < 45% assessed by MUGA or ---ECHO within 28 days prior to starting study cycle 1 (of midostaurin or control group)
  • Patients with any pulmonary infiltrate including those suspected to be of infectious origin (unless resolves to ≤ Grade 1 within screening timeframe)
  • Patient requires treatment with strong CYP3A4 inhibitors or moderate or strong CYP3A4 inducers other than those required for GVH or infection prophylaxis or treatment
  • Pregnant or nursing (lactating) women, or women of child-bearing potential, must use highly effective methods of contraception during dosing and for 30 days after treatment completion

Other protocol-defined inclusion/exclusion criteria may apply

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Mrinal Patnaik, MBBS

Contact us for the latest status

Wendy Sundt

(507)266-6004

Sundt.Wendy@mayo.edu