Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant

Not Recruiting

Trial ID: NCT02723435


This phase 2 trial studies the side effects and how well midostaurin works in treating older patients with acute myeloid leukemia with change in genetic material post-hematopoietic cell transplantation. Midostaruin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving midostaruin post-transplant may improve patient outcomes.

Official Title

An Open-Label Extension Study of Post-Transplant Maintenance Midostaurin (PKC412) in Elderly Patients (Age ≥ 60 Years) With FLT3-ITD/TKD Mutated AML Who Previously Received Midostaurin and Decitabine as Part of Study HEMAML0022 / CPKC412AUS27T

Stanford Investigator(s)

David Iberri
David Iberri

Clinical Assistant Professor, Medicine - Hematology



   - Elderly patients with FLT3-mutated acute myeloid leukemia (AML)

   - Prior enrollment in Stanford study IRB-25737

   - In continued complete remission

   - ≥ 30 days but ≤ 90 days post allogeneic hematopoietic cell transplant (HCT); treatment
   on this study protocol must begin before day 90 post-HCT

   - Absolute neutrophil count (ANC) ≥ 1000 cells/uL

   - Hemoglobin ≥ 8.0 g/dL and not requiring regular transfusions

   - Platelets ≥ 50,000 cells/uL and not requiring regular transfusions

   - Aspartate aminotransferase (AST) ≤ 2.5 times upper limit of normal (ULN)

   - Alanine aminotransferase (ALT) ≤ 2.5 X ULN

   - Serum bilirubin ≤ 2.5 times ULN

   - Ability to give written informed consent, including via legally authorized

   - Corrected QT (QTc) ≤ 450 msec

   - Ejection fraction (EF) ≥ 45% by 2-dimensional transthoracic echocardiography (TTE) or
   multiple-gated acquisition (MUGA)

   - Sexually active males, including vasectomized males, must agree via informed consent
   to use a condom during vaginal, anal, or oral intercourse, while taking midostaurin
   and for 5 months after stopping midostaurin

   - Females must have or be:

      - Negative pregnancy test, within 21 days of the first dose of midostaurin OR

      - Not of childbearing potential as follows:

         - Has undergone a hysterectomy or bilateral oophorectomy;

         - Has not had menses at any time in the preceding 24 consecutive months


   - Uncontrolled acute graft-vs-host disease (GVHD) grade 3 to 4

   - Uncontrolled active infection

   - Evidence of active AML (eg, circulating peripheral blasts on complete blood count)

   - Known confirmed diagnosis of human immunodeficiency virus (HIV) infection

   - Known confirmed diagnosis of active viral hepatitis

   - QTc > 450 msec

   - Congenital long QT syndrome

   - History of presence of sustained ventricular tachycardia, history of ventricular
   fibrillation or torsades de pointes

   - Bradycardia defined as heart rate (HR) < 50 beats per minute (bpm)

   - Bifascicular block (right bundle branch block plus left anterior hemiblock)

   - Congestive heart failure (CHF) New York Heart Association (NYHA) class 3 or 4

   - Cardiac ejection fraction (EF) < 45% within 28 days prior to starting cycle 1

   - Other known malignancy (except carcinoma in situ)

   - Other concurrent severe and/or uncontrolled medical condition which could compromise
   participation in the study, eg:

      - Uncontrolled diabetes

      - Chronic active pancreatitis

      - Myocardial infarction within 6 months

      - Poorly-controlled hypertension

      - Chronic kidney disease

   - Received any investigational agent within 30 days prior to day 1

   - Antineoplastic chemotherapy or radiotherapy within 28 days prior to cycle 1

   - No plans for concurrent chemotherapy while on study (exception: antineoplastic drugs
   used as part of GVHD prophylaxis or treatment)

   - Any surgical procedure, excluding central venous catheter placement, bone marrow
   biopsy or other minor procedures (eg, skin biopsy) within 14 days of day 1

   - Unwillingness or inability to comply with the protocol

   - Known malignant disease of the central nervous system

   - History of allergic reactions attributed to compounds of similar chemical or biologic
   composition to midostaurin

   - Concomitant use of strong inhibitors of cytochrome P450 family 3 subfamily A member 4

   - Pregnant or lactating

   - Women of child-bearing potential


drug: Midostaurin

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Jack Taw