A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

Official Title

A Phase 3 Multicentre, Open-label, Randomized Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia With FLT3 Mutation in Patients Not Eligible for Intensive Induction Chemotherapy


This is a clinical study for adult patients who have recently been diagnosed with acute myeloid leukemia or AML. AML is a type of cancer. It is when bone marrow makes white blood cells that are not normal. These are called leukemia cells. Some patients with AML have a mutation, or change, in the FLT3 gene. This gene helps leukemia cells make a protein called FLT3. This protein causes the leukemia cells to grow faster. For patients with AML who cannot receive standard chemotherapy, azacitidine (also known as Vidaza®) is a current standard of care treatment option in the United States. This clinical study is testing an experimental medicine called ASP2215, also known as gilteritinib. Gilteritinib works by stopping the leukemia cells from making the FLT3 protein. This can help stop the leukemia cells from growing faster. This study will compare two different treatments. Patients are assigned to one of these two groups by chance: a medicine called azacitidine, also known as Vidaza®, or an experimental medicine gilteritinib in combination with azacitidine. There is a twice as much chance to receive both medicines combined than azacitidine alone. The clinical study may help show which treatment helps patients live longer.

Trial Description

Primary Outcome:

  • Overall survival (OS)
Secondary Outcome:
  • Event free survival (EFS)
  • Best response
  • Complete remission (CR) rate
  • Composite complete remission (CRc) rate
  • Complete remission with partial hematologic recovery (CRh) rate
  • Complete remission and complete remission with partial hematological recovery (CR/CRh) rate
  • Transfusion conversion rate
  • Transfusion maintenance rate
  • Leukemia free survival (LFS)
  • Duration of remission
  • Participant reported fatigue from Brief Fatigue Inventory (BFI)
  • Safety assessed by adverse events (AEs)
  • Number of participants with abnormal laboratory values and/or adverse events related to treatment
  • Number of participants with abnormal vital signs and/or adverse events related to treatment
  • Number of participants with Physical Exam abnormalities and/or adverse events
  • Safety assessed by electrocardiograms (ECGs)
  • Eastern Cooperative Oncology Group (ECOG) performance status score
Patients considered an adult according to local regulation at the time of obtaining informed consent may participate in the study. Safety Cohort Prior to initiation of the randomized trial, 8 to 12 patients will be enrolled to evaluate the safety and tolerability of ASP2215 given with azacitidine therapy in the study population. Randomized Trial Approximately 250 patients will be randomized in a 2:1 ratio to receive ASP2215 plus azacitidine (Arm AC) or azacitidine only (Arm C). Patients will enter the screening period up to 14 days prior to the start of treatment. Patients will be administered treatment over 28-day cycles. Earlier protocol versions included a 1:1:1 randomization ratio to receive Arm A: ASP2215, Arm AC: ASP2215 + azacitidine or Arm C: azacitidine. Patients previously randomized to Arm A should continue following treatment and assessments as outlined in the protocol.

View this trial on ClinicalTrials.gov

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