Phase 3 N=15 Randomized Treatment

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

Acute Myeloid Leukemia (AML) · Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation

Bottom Line

View on ClinicalTrials.gov: NCT02752035 ↗

Enrolled (actual)

Serious AEs

83.2%

Results posted

Sep 2025

Primary outcome: Primary: Overall Survival (OS) — 9.82; 9.23; 5.24 months — p=0.523

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: gilteritinib (Drug); azacitidine (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Astellas Pharma Global Development, Inc.
Primary completion: Mar 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Overall Survival (OS)	9.82; 9.23; 5.24	0.523
SECONDARY Event-free Survival (EFS)	0.03; 0.03; 0.03	0.787
SECONDARY Percentage of Participants With Best Response	25.8; 17.5; 9.1; 6.7; 0; 18.2	—
SECONDARY Completed Remission (CR) Rate	25.8; 17.5; 9.1	0.249
SECONDARY CRc Rate	61.8; 28.1; 54.5	<0.001 sig
SECONDARY Complete Remission With Partial Hematologic Recovery (CRh)	10.1; 1.8; 22.7	0.049 sig
SECONDARY CR/CRh Rate	36.0; 19.3; 31.8	0.032 sig
SECONDARY Percentage of Participants With Transfusion Conversion Rate	35.6; 44.7; 61.1	0.340
SECONDARY Percentage of Participants With Transfusion Maintenance Rate	100.0; 50.0; 50.0	0.221
SECONDARY Leukemia-free Survival (LFS)	7.16; 8.08; 3.20	0.592
SECONDARY Duration of Remission	19.94; 8.08; 2.83; 25.10; 8.25; NA	0.300
SECONDARY Change From Baseline in Brief Fatigue Inventory (BFI)	0.77; 0.87; 1.37	0.560
SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs)	15; 88; 52; 22	—
SECONDARY Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores	1; 15; 9; 3; 4; 34	—

Summary

This was a clinical study for adult participants who were recently 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 participants 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 participants with AML who could not receive standard chemotherapy, azacitidine (also known as Vidaza®) was a current standard of care treatment option in the United States. This clinical study tested an experimental medicine called ASP2215, also known as gilteritinib. Gilteritinib worked by stopping the leukemia cells from making the FLT3 protein. This helped stop the leukemia cells from growing faster. This study compared two different treatments. Participants were 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 was 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.

Eligibility Criteria

Inclusion Criteria

Subject is considered an adult according to local regulation at the time of obtaining informed consent.
Subject has a diagnosis of previously-untreated AML according to World Health Organization (WHO) classification [Swerdlow et al, 2008] as determined by pathology review at the treating institution.
Subject is positive for FLT3 mutation (internal tandem duplication [ITD] or tyrosine kinase domain [TKD] [D835/I836] mutation) (or for Korea only: ITD alone or ITD with concurrent TKD activating mutation) in bone marrow or whole blood as determined by central laboratory. Note: Only requirement of FLT3 mutation assessment by central laboratory is only applicable to the randomization portion of the study.
Subject is ineligible for intensive induction chemotherapy by meeting at least 1 of the following criteria:
Subject is ≥ 65 years of age and ineligible for intensive induction chemotherapy.
Subject is ≥ 18 to 64 years of age and has any of the following comorbidities: [Ex-US Only]: Congestive heart failure (New York Heart Association {NYHA} class ≤ 3) or ejection fraction (Ef) ≤ 50%; [US Only]: Severe cardiac disorder e.g. congestive heart failure (New York Heart Association [NYHA] class ≤ 3) requiring treatment, ejection fraction ≤ 50%, or chronic stable angina; [Ex-US Only]: Creatinine > 2 mg/dL (177 µmol/L), dialysis or prior renal transplant; [US Only]: Creatinine clearance 480 ms at screening based on central reading.
Subject with a history of Long QT Syndrome at screening.
[Ex-US Only]: Subject has known pulmonary function tests with diffusion capacity of lung for carbon monoxide (DLCO) ≤ 50%, forced expiratory volume in the first second (FEV1) ≤ 60%, dyspnea at rest or requiring oxygen or any pleural neoplasm (Transient use of supplemental oxygen is allowed.)
Subject has active hepatitis B or C or other active hepatic disorder.
Subjects with positive hepatitis B surface antigen (HBsAg) or detectable hepatitis B DNA are not eligible.
Subjects with negative HBsAg, positive hepatitis B core antibody and negative hepatitis B surface antibody will be eligible if hepatitis B DNA is undetectable.
Subjects with antibodies to hepatitis C virus will be eligible if hepatitis C RNA is undetectable
Subject has any condition which makes the subject unsuitable for study participation, including any contraindications of azacitidine.
Subject has a known or suspected hypersensitivity to ASP2215, azacitidine or any components of the formulations used.
[US Only]: Subject is ≥ 65 to 74 years of age, suitable for and willing to receive intensive induction chemotherapy.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02752035). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.