CompletedNCT02310321Updated Sep 2, 2025Results posted

A Study of ASP2215 in Combination With Induction and Consolidation Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia.

A Phase 1/2 interventional study of Gilteritinib and Idarubicin in Acute Myeloid Leukemia and FLT3-mutated Acute Myeloid Leukemia, sponsored by Astellas Pharma Inc. Completed at 55 sites in 3 countries. Open to participants aged 18 Years to 69 Years. Per ClinicalTrials.gov, last updated 2025-09-02.

Sponsored by Astellas Pharma Inc · Phase 1/2, Interventional, and Treatment

Phase: Phase 1/2
Study type: Interventional
Enrollment: 97
Allocation: Non-randomized
Ages: 18 Years to 69 Years
Sex: All

Study summary

The purpose of phase 1 part in this study was to determine the maximum tolerated dose (MTD) and/or recommended expansion dose (RED) of ASP2215 concomitant with cytarabine/idarubicin as induction chemotherapy based on the status of the onset of dose-limiting toxicity (DLT) in newly diagnosed Acute Myeloid Leukemia (AML) subjects. Phase 1 part also evaluated safety and tolerability and characterized the pharmacokinetic (PK) parameters of ASP2215 concomitant with induction and consolidation chemotherapy as well as evaluated the PK parameters of cytarabine concomitant with ASP2215.

The purpose of phase 2 part was to evaluate efficacy of ASP2215 in combination with induction therapy. Phase 2 cohort also evaluated safety and characterized the PK parameters of ASP2215 in combination with induction and consolidation therapy followed by maintenance therapy in newly diagnosed FLT3-mutated AML subjects.

Read the detailed description

This study was composed of Phase 1 part (the dose-evaluation part and the expansion part) and Phase 2 part.

In the dose-evaluation part of Phase 1 part, at least 3 subjects received ASP2215 at each dose (low, middle, and high) for determination of MTD and/or RED. Treatment of AML in Phase 1 part was composed of 3 periods of therapy: remission induction, consolidation, and maintenance. The decision of whether or not to proceed to the next dose was made based on the occurrence of DLT during Cycle 1 of the induction period.

In the expansion part of Phase 1 part, a maximum of 3 subjects received ASP2215 at RED that had been recommended in the dose-evaluation part and the safety was assessed based on the onset of DLTs during Cycle 1 of the induction and consolidation periods.

In Phase 2 part, Subjects received ASP2215 at the recommended dose established in Phase 1 part. The target population was limited to newly diagnosed FLT3-mutated AML.

Conditions studied

Acute Myeloid Leukemia
FLT3-mutated Acute Myeloid Leukemia

Browse trials for

Leukemia, Myeloid, Acute 2,971

Keywords

Acute Myeloid Leukemia
Cytarabine
Idarubicin
ASP2215

In context

Leukemia, Myeloid, Acute

2,971 studies on the registry are indexed under Leukemia, Myeloid, Acute; 745 are open to participants now.

This study's enrollment of 97 is above the median of 41 across 2,509 interventional studies indexed under Leukemia, Myeloid, Acute.

Browse Leukemia, Myeloid, Acute studies →

Lead sponsor

Astellas Pharma Inc is the lead sponsor of 512 studies on the registry; 4 are open to participants now.

Of its 27 completed or terminated interventional studies of FDA-regulated products, 19 (70%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years to 69 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

[Phase 1 part]

Subject is defined as having previously untreated de novo AML according to the World Health Organization (WHO) criteria (2008) within 28 days prior to study enrollment.
Subject has an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2.
Subject must meet all of the following criteria in the laboratory test at screening:
- Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels of ≤ 2.5 × institutional upper limit of normal (ULN)
- Total serum bilirubin level of ≤ 1.5 × institutional ULN
- Serum creatinine level of ≤ 1.5 × institutional ULN or an estimated glomerular filtration rate (eGFR) of > 50 mL/min
Subject is suitable for oral administration of ASP2215.
Female subject falls under the following:
- Of non-childbearing potential:
- ・Post-menopausal (defined as at least 1 year with no menses without a medical reason such as drug administration) at screening, or
- ・Documented surgically sterile or status post-hysterectomy (at least 1 month prior to screening)
- Of childbearing potential:
- ・Has a negative result for the pregnancy test at screening, and
- ・Agrees to use an appropriate contraception starting at screening and throughout the study period and for 60 days after the final study drug administration
Female subject agrees not to breastfeed starting at screening and throughout the study period and for 60 days after the final study drug administration.
Female subject agrees not to donate ova starting at screening and throughout the study period and for 60 days after the final study drug administration.
Male subject and his female spouse/partner who is of childbearing potential agrees to use an appropriate contraception starting at screening and throughout the study period and for 120 days after the final study drug administration.
Male subject agrees not to donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration.
Subject agrees not to participate in another interventional study while on study treatment.
Subject can be admitted during the induction period.

[Phase 2 part]

Subject has a diagnosis of previously-untreated de novo acute myeloid leukemia (AML) according to World Health Organization (WHO) classification (2017) documented within 28 days prior to enrollment.
Subject is positive for FLT3-ITD and/or TKD mutation in bone marrow or whole blood as determined by the central lab. Registration by the local lab result is not acceptable.
Subject has an ECOG performance status (PS) 0 to 1. Subject who has an ECOG PS 2 is eligible only if the primary disease related symptoms such as pneumonia and febrile neutropenia are the cause of PS score.
Subject is suitable for oral administration of ASP2215.
Female subject is not pregnant and at least 1 of the following conditions apply:
- Not a woman of childbearing potential (WOCBP)
- WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 180 days after final study treatment administration.
Female subject must agree not to breastfeed starting at screening and throughout the study period, and for 60 days after the final study drug administration.
Female subject must not donate ova starting at screening and throughout the study period, and for 180 days after the final study drug administration.
Male subject and their female partners who are of childbearing potential must be using highly effective contraception per locally accepted standards in addition to a barrier method starting at screening and continue throughout the study period and for 120 days after the final study drug administration.
Male subject must not donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration.
Male subject with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 120 days after the final study treatment administration.
Subject agrees not to participate in another interventional study while on treatment.
Subject must meet the following criteria as indicated on the clinical laboratory tests:
- Serum creatinine ≤ 1.5 × institutional upper limit of normal (ULN), or if serum creatinine outside normal range, then glomerular filtration rate (GFR) > 50 mL/min/1.73m\^2 as calculated with the 4-parameter Modification of Diet in Renal Disease (MDRD) equation.
- Serum total bilirubin ≤ 2.5 mg/dL (43 μmol/L), except for subjects with Gilbert's syndrome.
- Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x ULN. If liver abnormality by the primary disease is suspected, subject may be pre-registered to initiate the chemotherapy. Prior to registration, AST/ALT values must meet the criteria to continue the study.
- Serum magnesium ≥ institutional lower limit of normal (LLN). Subject may pre-register without magnesium value, but subject must meet the criteria prior to the full registration on Day 8.
- Serum potassium ≥ institutional lower limit of normal (LLN).

Exclusion criteria

Exclusion Criteria:

[Phase 1 part]

Subject was diagnosed with acute promyelocytic leukemia (APL).
Subject has breakpoint cluster region-abelson (BCR-ABL)-positive leukemia (chronic myelogenous leukemia in blast crisis).
Subject has active malignant tumors other than AML or myelodysplastic syndrome (MDS).
Subject has received prior AML treatment except for the following:
- Urgent leukapheresis
- Hydroxyurea administration for emergency treatment of hyperleukocytosis (≤ 7 days)
- Administration of retinoic acid before the diagnosis to exclude APL (≤ 7 days)
- Supportive care using growth factors or cytokines
- Steroid administration to treat hypersensitivity or blood transfusion reactions
Subject has clinically active central nervous system leukemia.
Subject has disseminated intravascular coagulation (DIC).
Subject has had major surgery within 28 days prior to the first study drug administration.
Subject has had radiation therapy within 28 days prior to the first study drug administration.
Subject has congestive heart failure of New York Heart Association (NYHA) class 3 or 4, or subject with a past history of congestive heart failure of NYHA class 3 or 4 and in whom echocardiogram (ECHO) or Multiple Gate Acquisition (MUGA) scan performed within 3 months prior to screening or at screening showed a left ventricular ejection fraction (LVEF) of \< 45%.
Subject has cardiac impairment or a clinically significant cardiac disease, including any one of the following:
- Complete left bundle branch block
- Obligate use of a cardiac pacemaker
- Long QT syndrome at Screening
- Prolongation of the QTc interval (> 450 ms) on electrocardiogram (ECG) at screening
- Right bundle branch block + left anterior hemiblock (bifascicular block)
- Angina pectoris within 3 months prior to study drug administration
- Acute myocardial infarction within 3 months prior to study drug administration
Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP)3A.
Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of P glycoprotein (P-gp) with the exception of drugs that are considered absolutely essential for the care of the subject.
Subject requires treatment with concomitant drugs that target serotonin 5HT1 or 5HT2B receptors or sigma receptors, with the exception of drugs that are considered absolutely essential for treatment of the subject.
Subject has an active uncontrollable infection.
Subject is known to have human immunodeficiency virus (HIV) infection.
Subject has active hepatitis B or C or other active hepatic disorders.
Subject has any condition that, in the investigator's or sub-investigator's opinion, makes the subject unsuitable for study participation.
Potassium and magnesium levels of below institutional lower limit of normal in the laboratory test at screening.

[Phase 2 part]

Subject was diagnosed with acute promyelocytic leukemia (APL).
Subject has known BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis).
Subject has therapy-related AML.
Subject has active malignant tumors other than AML.
Subject has received previous therapy for AML, with the exception of the following:
- Emergency leukapheresis
- Emergency treatment for hyperleukocytosis with hydroxyurea for ≤ 10 days
- Preemptive treatment with retinoic acid prior to exclusion of APL ≤ 7 days
- Growth factor or cytokine support
- Steroids for the treatment of hypersensitivity or transfusion reactions.
Subject has QTcF interval > 450 ms (average of triplicate determinations based on central reading).
Subject with long QT syndrome.
Subject has clinically active central nervous system leukemia.
Subject has had major surgery within 4 weeks prior to the first study dose.
Subject has radiation therapy within 4 weeks prior to the first study dose.
Subject has immediate life-threatening, severe complications of leukemia such as severe uncontrolled bleeding and/or severe disseminated intravascular coagulation
Subject is known to have human immunodeficiency virus infection.
Subject has active hepatitis B or C.
Subject has an uncontrolled infection. An infection controlled with an approved or closely monitored antibiotic/antiviral/antifungal treatment is allowed.
Subject has uncontrolled angina, severe uncontrolled ventricular arrhythmias, electrocardiographic evidence of acute ischemia, congestive heart failure New York Heart Association (NYHA) class 3 or 4 or subject has a history of congestive heart failure of NYHA class 3 or 4 and echocardiogram (ECHO) or Multiple Gate Acquisition (MUGA) scan performed within 3 months prior to screening or at screening showed a left ventricular ejection fraction (LVEF) of \< 45%.
Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP) 3A.
Subject requires treatment with concomitant drugs that target serotonin 5HT2B receptors or sigma nonspecific receptors, with the exception of drugs that are considered absolutely essential for treatment of the subject.
Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of P glycoprotein (P-gp) with the exception of drugs that are considered absolutely essential for the care of the subject.
Subject has prior malignancies, except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent ≥ 5 years previously will be allowed. Cancer treated with curative intent \< 5 years previously will not be allowed.
Subject has any condition which makes the subject unsuitable for study participation.

Study design

Phase: Phase 1 / Phase 2
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Sequential assignment
Masking: None (open label)
Enrollment: 97 participants (actual)

Study arms

Experimental
Phase 1: Dose Evaluation (DEv)
Induction period: Participants received 120 milligrams (mg) gilteritinib (3 tablets of 40 mg) orally, once daily from day 4 to 17 combined with chemotherapy (idarubicin: 12 mg per square meters per day \[mg/m\^2/day\] on days 1 to 3, cytarabine: 100 mg/m\^2/day on days 1 to 7) via intravenous (IV) infusion; in cycles (C) 1 and 2 (1 cycle= 42 days). Consolidation period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once daily from day 1 to 14 combined with chemotherapy (cytarabine: 1500 mg/m\^2, twice daily on days 1, 3, and 5) via IV infusion; in the consolidation period in C1 to 3 (1 cycle= 28 days). Duration of infusion was determined by site clinical practice and local package insert in both induction and consolidation periods.. Maintenance period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once daily from days 1 to 28 in the maintenance period in C1 to 26 or untill discontinuation criterion is met (1 cycle= 28 days).
Drug: Gilteritinib · Drug: Idarubicin · Drug: Cytarabine
Experimental
Phase 1: Dose Expansion (DEx)
Induction period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day from days 4 to 17 in combination with chemotherapy (idarubicin: 12 mg/m\^2/day on days 1 to 3, cytarabine: 100 mg/m\^2/day on days 1 to 7) via IV infusion; in the induction period in C1 and 2 (1 cycle= 42 days). Consolidation period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day from days 1 to 14 in combination with chemotherapy (cytarabine: 1500 mg/m\^2, twice daily on days 1, 3, and 5) via IV infusion; in the consolidation period in C1 to 3 (1 cycle= 28 days). Duration of infusion was determined based on site clinical practice and the local package insert in both induction and consolidation periods. Maintenance period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day from days 1 to 28 in the maintenance period in C1 to 26 or a discontinuation criterion is met (1 cycle= 28 days).
Drug: Gilteritinib · Drug: Idarubicin · Drug: Cytarabine
Experimental
Phase 2: FLT3-mutated AML
Induction period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once daily from days 8 until blood recovery combined with chemotherapy (idarubicin: 12 mg/m\^2/day on days 1 to 3, cytarabine: 100 mg/m\^2/day on days 1 to 7) via IV infusion; in C1 and 2. Each cycle was extended until blood recovery was observed. Consolidation period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once daily from days 1 until blood recovery combined with chemotherapy (cytarabine: 1500 mg/m\^2, twice daily on days 1, 3, and 5) via IV infusion; in C1 to 3. Each cycle was extended until blood recovery was observed. Duration of infusion was based on site clinical practice and the local package insert in both induction and consolidation periods. Maintenance period: Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once daily from days 1 to 28 in the maintenance period in C1 to 26 or a discontinuation criterion is met (1 cycle= 28 days).
Drug: Gilteritinib · Drug: Idarubicin · Drug: Cytarabine

Interventions

DrugGilteritinib
Once-daily oral administration on 14 consecutive days in every cycle in each period.
Also known as: Xospata, ASP2215
DrugIdarubicin
Induction period: Once-daily intravenous injection of 12 mg/m\^2 idarubicin on 3 consecutive days.
DrugCytarabine
Induction period: Once-daily intravenous injection of 100 mg/m\^2 cytarabine on 7 consecutive days. Consolidation period: Twice-daily intravenous injection of 1.5 g/m\^2 cytarabine on Days 1, 3, and 5.

What researchers measure

Primary outcomes

Phase 1 Part: Maximum Tolerated Dose (MTD) of Gilteritinib
The MTD was defined as the highest dose of gilteritinib at which the posterior mean of the DLT incidence during Cycle 1 of induction therapy was estimated to be closest to 33%.
Time frame: Day 1 up to the end of Induction period cycle 1 (up to 42 days)
Phase 1 Part: Recommended Expansion Dose (RED) of Gilteritinib
RED was the recommended dose used in Phase 2 of the study that was decided by the sponsor's responsible person by comprehensively assessing the data obtained from the study.
Time frame: Day 1 up to the end of Induction period cycle 1 (up to 42 days)
Phase 1 Part: Number of Participants With Dose Limiting Toxicities (DLTs) of Gilteritinib
DLTs were defined as: Any Grade ≥ 3 non-hematologic or extramedullary toxicity with the following exceptions: * Anorexia or fatigue * Grade 3 nausea and/or vomiting if participant did not require tube feeding or total parenteral nutrition, or diarrhea if the events did not require or prolong hospitalization that could be managed to grade ≤ 2 with standard antiemetic or antidiarrheal medications used at prescribed dose within 7 days of onset. * Grade 3 mucositis that resolved to Grade ≤ 2 within 7 days of onset, fever with neutropenia, with or without infection, infection * Grade 4 peripheral neutrophil count \< 500/cubic millimeters (mm\^3) * Platelet count \< 20,000/mm\^3 due to bone marrow hypoplasia * Grade ≥ 3 platelet count \< 50,000/mm\^3 accompanying bleeding * Grade 4 platelet count \< 25,000/mm\^3 requiring platelet transfusion DLT was assessed until cycle 1 of dose evaluation induction period and until cycle 1 of dose expansion consolidation period.
Time frame: Day 1 up to the end of Consolidation Cycle 1 (approximately up to 4 months)
Phase 1 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE could therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. TEAE included both serious and non-serious AEs. TEAE for Phase 1 was defined as an AE observed after the date of first dose until 30 days after the last dose.
Time frame: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.1 years)
Phase 2 Part: Complete Remission (CR) Rate: Induction Period
Percentage of participants with CR was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm3 and platelet count of ≥ 100,000/mm3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%.
Time frame: From the date of first dose up to the start of Consolidation (approximately up to 4 months)

Secondary outcomes

Phase 1 Part: Maximum Concentration (Cmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
Cmax was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.
Time frame: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Phase 1 Part: Time to Attain Cmax (Tmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
Tmax was derived from the PK samples collected.
Time frame: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Phase 1 Part: Area Under Plasma Concentration-time Curve From Time 0 to 24 (AUC24) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
AUC24 was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.
Time frame: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Phase 1 Part: Area Under The Concentration-Time Curve From The Time Zero to The Last Measurable Concentration (AUClast) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
AUClast was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.
Time frame: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Phase 1 Part: Plasma Trough Concentration (Ctrough) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
Ctrough is the plasma concentration prior to drug administration. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.
Time frame: Induction period: Pre-dose on Cycle 1 Day 8, 11, and 17 Consolidation period: Pre-dose on Cycle 1 Day 6 and 15
Phase 1 Part: Plasma Trough Concentration of Cytarabine Concomitant With Gilteritinib With Induction and Consolidation Period
Ctrough is the plasma concentration prior to drug administration. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.
Time frame: Induction period: Predose on Cycle 1 Day 1, 3, and 8 Consolidation period: Predose on Cycle 1 Day 2 and 6
Phase 2 Part: Plasma Trough Concentration of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
Ctrough is the plasma concentration prior to drug administration.
Time frame: Induction period : Predose on Cycle 1 Day 15 and 21 Consolidation period: Predose on Cycle 1 Day 8 and 15
Phase 2 Part: Overall Survival (OS)
Overall survival (OS) was defined as the time from the date of first dose of day 1 to the date of death due to any cause. Participants still alive or lost to follow up was censored at the time they were last known to be alive. Kaplan-Meier (KM) estimate was used for analysis.
Time frame: From the date of first dose up to the date of death (maximum duration: approximately 4.4 years)
Phase 2 Part: Event Free Survival (EFS)
Event-free survival (EFS): time from date of first dose of study regimen until date of documented relapse, treatment failure or death from any cause, whichever occurred first. KM estimate was used for analysis. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in bone marrow aspirate (BMA)/reappearance of significant numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%. Treatment failure: participants who failed to achieve composite complete remission (CRc) or who discontinued treatment due to "lack of efficacy" without previous response. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: defined in outcome measure #5.
Time frame: From the date of first dose up to the date of documented relapse, treatment failure or death from any cause (maximum duration: approximately 4.4 years)
Phase 2 Part: Relapse Free Survival (RFS)
Relapse-free survival (RFS): time from date of achievement of first CRc until relapse or death from any cause. KM estimate was used for analysis. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in bone marrow aspirate (BMA)/reappearance of significant numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%.
Time frame: From the date of achievement of first CRc up to the date of documented relapse or death from any cause (maximum duration: approximately 3.9 years)
Phase 2 Part: CR Rate After Consolidation and Maintenance Period
Percentage of participants with CR was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the consolidation period refers to the best response from the start of treatment in the induction period until the end of the consolidation period and for the maintenance period refers to the best response from the start of treatment in the induction period until the end of the maintenance period.
Time frame: CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Phase 2 Part: CR Rate Without Minimal Residual Disease (MRD) After Each Treatment Therapy Period
CR% without MRD reported. CR rate without MRD after each treatment therapy was defined similarly as CR rate after each treatment therapy. Responders achieve that the best response is CR and MRD status was negative. CR was defined as a morphologically leukemia-free state, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.
Time frame: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Phase 2 Part: CR With Partial Hematological Recovery (CRh) Rate After Each Treatment Therapy Period
Percentage of participants with CRh was reported. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.
Time frame: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Phase 2 Part: Composite CR (CRc) Rate After Each Treatment Therapy Period
Percentage of participants with CRc reported. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recovery (CRi). CRp: met all CR criteria, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.
Time frame: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Phase 2 Part: CR/CRh Rate After Each Treatment Therapy Period
Percentage of participants with CR/CRh was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.
Time frame: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Phase 2 Part: Duration of CR
Duration of CR was defined as the time from the date of achieving first CR until the date of first documented relapse for participants who achieved CR. KM estimate was used for analysis. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.
Time frame: From the date of achieving CR up to the date of documented relapse (maximum duration: approximately 2.6 years)
Phase 2 Part: Duration of CR/CRh
Duration of CR/CRh is defined as the time from the date of achieving first CR/CRh until the date of first documented relapse for participants who achieved CR/CRh. KM estimate was used for analysis. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.
Time frame: From the date of achieving CR/CRh up to the date of documented relapse (maximum duration: approximately 2.6 years)
Phase 2 Part: Duration of CRh
Duration of CRh was defined as the time from date of achieving first CRh until date of first documented relapse for participants who achieved CRh. KM estimate was used for analysis. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.
Time frame: From the date of achieving CRh up to the date of documented relapse (maximum duration: approximately 2.6 years)
Phase 2 Part: Duration of CRc
Duration of CRc is defined as the time from the date of achieving first CRc until the date of first documented relapse for participants who achieved CRc. KM estimate was used for analysis. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.
Time frame: From the date of achieving CRc up to the date of documented relapse (maximum duration: approximately 2.7 years)
Phase 2 Part: Duration of Response (DoR)
DoR: from first day of achieving CRc (CR+ CRp,+CRi)/partial remission (PR) to first day of relapse. KM estimate was used for analysis. CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 \& platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Peripheral blood blast counts was ≤ 2%. CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). PR: condition with regeneration of normal hematopoietic cells in bone marrow, no detectable blasts, ≥ 50% decrease of blasts in BMA \& total bone marrow blasts of 5-25%. No evidence of extramedullary leukemia. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%
Time frame: From the date of achieving CR, CRp, CRi/PR up to the date of documented relapse (maximum duration: approximately 2.7 years)
Phase 2 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE could therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. TEAE for Phase 2 was defined as an AE observed after the date of first dose until 30 days after the last dose. TEAE included both serious and non-serious AEs.
Time frame: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.4 years)

Results

Posted Oct 16, 2024

Participant flow

Participants with newly diagnosed acute myeloid leukemia (AML) and participants newly diagnosed FMS-like tyrosine kinase-3, FMS-related tyrosine kinase 3 (FLT3)-mutated AML; were enrolled in Phase 1 and Phase 2, respectively.

Phase 1(DEv):IP (2 Cycles;42 Days/Cycle)

Participant flow — Phase 1(DEv):IP (2 Cycles;42 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	3	0	0
Completed	2	0	0
Not completed	1	0	0
Withdrew: Miscellaneous	1	0	0

Phase 1(DEv):CP (3 Cycles;28 Days/Cycle)

Participant flow — Phase 1(DEv):CP (3 Cycles;28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	2	0	0
Completed	1	0	0
Not completed	1	0	0
Withdrew: Miscellaneous	1	0	0

Phase 1(DEv):MP(26 Cycles;28 Days/Cycle)

Participant flow — Phase 1(DEv):MP(26 Cycles;28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	1	0	0
Completed	0	0	0
Not completed	1	0	0
Withdrew: Miscellaneous	1	0	0

Phase 1(DEx):IP (2 Cycles;42 Days/Cycle)

Participant flow — Phase 1(DEx):IP (2 Cycles;42 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	10	0
Completed	0	3	0
Not completed	0	7	0
Withdrew: Adverse event	0	1	0
Withdrew: Miscellaneous	0	4	0
Withdrew: There is no cr, crp, or cri after 2 cycles of therapy	0	1	0
Withdrew: Disease relapse	0	1	0

Phase 1(DEx):CP (3 Cycles;28 Days/Cycle)

Participant flow — Phase 1(DEx):CP (3 Cycles;28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	3	0
Completed	0	3	0
Not completed	0	0	0

Phase 1(DEx):MP(26 Cycles;28 Days/Cycle)

Participant flow — Phase 1(DEx):MP(26 Cycles;28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	3	0
Completed	0	0	0
Not completed	0	3	0
Withdrew: Micellaneous	0	3	0

Phase 2:IP(2 Cycles;up to 56 Days/Cycle)

Participant flow — Phase 2:IP(2 Cycles;up to 56 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	0	84
Completed	0	0	62
Not completed	0	0	22
Withdrew: Miscellaneous	0	0	2
Withdrew: Disease relapse	0	0	2
Withdrew: Physician decision	0	0	6
Withdrew: Withdrawal by subject	0	0	4
Withdrew: Lack of efficacy	0	0	3
Withdrew: Death	0	0	3
Withdrew: Adverse event	0	0	2

Phase 2:CP(4 Cycles;up to 28 Days/Cycle)

Participant flow — Phase 2:CP(4 Cycles;up to 28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	0	57
Completed	0	0	42
Not completed	0	0	15
Withdrew: Miscellaneous	0	0	7
Withdrew: Disease relapse	0	0	4
Withdrew: Physician decision	0	0	4

Phase 2:MP(26 Cycles; 28 Days/Cycle)

Participant flow — Phase 2:MP(26 Cycles; 28 Days/Cycle)
Milestone	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Started	0	0	46
Completed	0	0	23
Not completed	0	0	23
Withdrew: Disease relapse	0	0	11
Withdrew: Miscellaneous	0	0	2
Withdrew: Adverse event	0	0	3
Withdrew: Death	0	0	1
Withdrew: Physician decision	0	0	6

Outcome measures

PrimaryPhase 1 Part: Maximum Tolerated Dose (MTD) of Gilteritinib

The MTD was defined as the highest dose of gilteritinib at which the posterior mean of the DLT incidence during Cycle 1 of induction therapy was estimated to be closest to 33%.

Time frame:: Day 1 up to the end of Induction period cycle 1 (up to 42 days)
Reported as:: Number · miligram (mg)

Phase 1 Part: Maximum Tolerated Dose (MTD) of Gilteritinib
miligram (mg)	Phase 1: Dose Evaluation (DEv)
Phase 1 Part: Maximum Tolerated Dose (MTD) of Gilteritinib	NA

PrimaryPhase 1 Part: Recommended Expansion Dose (RED) of Gilteritinib

RED was the recommended dose used in Phase 2 of the study that was decided by the sponsor's responsible person by comprehensively assessing the data obtained from the study.

Time frame:: Day 1 up to the end of Induction period cycle 1 (up to 42 days)
Reported as:: Number · mg

Phase 1 Part: Recommended Expansion Dose (RED) of Gilteritinib
mg	Phase 1: Dose Evaluation (DEv)
Phase 1 Part: Recommended Expansion Dose (RED) of Gilteritinib	120

PrimaryPhase 1 Part: Number of Participants With Dose Limiting Toxicities (DLTs) of Gilteritinib

DLTs were defined as: Any Grade ≥ 3 non-hematologic or extramedullary toxicity with the following exceptions: * Anorexia or fatigue * Grade 3 nausea and/or vomiting if participant did not require tube feeding or total parenteral nutrition, or diarrhea if the events did not require or prolong hospitalization that could be managed to grade ≤ 2 with standard antiemetic or antidiarrheal medications used at prescribed dose within 7 days of onset. * Grade 3 mucositis that resolved to Grade ≤ 2 within 7 days of onset, fever with neutropenia, with or without infection, infection * Grade 4 peripheral neutrophil count \< 500/cubic millimeters (mm\^3) * Platelet count \< 20,000/mm\^3 due to bone marrow hypoplasia * Grade ≥ 3 platelet count \< 50,000/mm\^3 accompanying bleeding * Grade 4 platelet count \< 25,000/mm\^3 requiring platelet transfusion DLT was assessed until cycle 1 of dose evaluation induction period and until cycle 1 of dose expansion consolidation period.

Time frame:: Day 1 up to the end of Consolidation Cycle 1 (approximately up to 4 months)
Reported as:: Number · Participants

Phase 1 Part: Number of Participants With Dose Limiting Toxicities (DLTs) of Gilteritinib
Participants	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Phase 1 Part: Number of Participants With Dose Limiting Toxicities (DLTs) of Gilteritinib	0	1

PrimaryPhase 1 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

An AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE could therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. TEAE included both serious and non-serious AEs. TEAE for Phase 1 was defined as an AE observed after the date of first dose until 30 days after the last dose.

Time frame:: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.1 years)
Reported as:: Number · Participants

Phase 1 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Participants	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Phase 1 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)	3	10

PrimaryPhase 2 Part: Complete Remission (CR) Rate: Induction Period

Percentage of participants with CR was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm3 and platelet count of ≥ 100,000/mm3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%.

Time frame:: From the date of first dose up to the start of Consolidation (approximately up to 4 months)
Reported as:: Number · Percentage of participants

Phase 2 Part: Complete Remission (CR) Rate: Induction Period
Percentage of participants	Phase 2: FLT3-mutated AML
Phase 2 Part: Complete Remission (CR) Rate: Induction Period	50 (40.4 to 59.6)

SecondaryPhase 1 Part: Maximum Concentration (Cmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

Cmax was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.

Time frame:: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Reported as:: Mean · nanograms per milliliter (ng/mL)

Phase 1 Part: Maximum Concentration (Cmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
nanograms per milliliter (ng/mL)	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction Period	246 ± 154	132 ± 27.5
Consolidation period	133 ± NA	73.5 ± 17.5

SecondaryPhase 1 Part: Time to Attain Cmax (Tmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

Tmax was derived from the PK samples collected.

Time frame:: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Reported as:: Median · hours

Phase 1 Part: Time to Attain Cmax (Tmax) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
hours	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction period	3.93 (3.82 to 6.13)	5.77 (3.83 to 9.77)
Consolidation period	2.94 (1.85 to 4.03)	4.32 (3.90 to 9.70)

SecondaryPhase 1 Part: Area Under Plasma Concentration-time Curve From Time 0 to 24 (AUC24) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

AUC24 was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.

Time frame:: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Reported as:: Mean · ng*hr/mL

Phase 1 Part: Area Under Plasma Concentration-time Curve From Time 0 to 24 (AUC24) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
ng*hr/mL	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction period	3880 ± 2530	2210 ± 567
Consolidation period	2110 ± NA	1160 ± 204

SecondaryPhase 1 Part: Area Under The Concentration-Time Curve From The Time Zero to The Last Measurable Concentration (AUClast) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

AUClast was derived from the PK samples collected. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.

Time frame:: Induction period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 4 Consolidation period: Pre-dose, 1, 2, 4, 6, 10, and 24 hours post-dose on Cycle 1 Day 1
Reported as:: Mean · ng*hr/mL

Phase 1 Part: Area Under The Concentration-Time Curve From The Time Zero to The Last Measurable Concentration (AUClast) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
ng*hr/mL	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction period	3830 ± 2520	2190 ± 552
Consolidation period	2090 ± NA	1150 ± 202

SecondaryPhase 1 Part: Plasma Trough Concentration (Ctrough) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

Ctrough is the plasma concentration prior to drug administration. It was planned that standard deviation(SD) would only be calculated if there are more than 3 participants analyzed otherwise, SD was not calculated.

Time frame:: Induction period: Pre-dose on Cycle 1 Day 8, 11, and 17 Consolidation period: Pre-dose on Cycle 1 Day 6 and 15
Reported as:: Mean · ng/mL

Phase 1 Part: Plasma Trough Concentration (Ctrough) of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
ng/mL	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction period: Cycle 1 day 8	245 ± 144	245 ± 77.7
Induction period: Cycle 1 day 11	283 ± 183	417 ± 203
Induction period: Cycle 1 day 17	393 ± 252	455 ± 143
Consolidation period: Cycle 1 day 6	194 ± NA	159 ± 12.1
Consolidation period: Cycle 1 day 15	285 ± NA	171 ± 154

SecondaryPhase 1 Part: Plasma Trough Concentration of Cytarabine Concomitant With Gilteritinib With Induction and Consolidation Period

Time frame:: Induction period: Predose on Cycle 1 Day 1, 3, and 8 Consolidation period: Predose on Cycle 1 Day 2 and 6
Reported as:: Mean · ng/mL

Phase 1 Part: Plasma Trough Concentration of Cytarabine Concomitant With Gilteritinib With Induction and Consolidation Period
ng/mL	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)
Induction period: Cycle 1 Day 1	0 ± NA	0 ± NA
Induction period: Cycle 1 Day 3	34.9 ± 14.5	210 ± 417
Induction period: Cycle 1 Day 8	58.8 ± 8.67	110 ± 107
Consolidation period: Cycle 1 Day 2	4.87 ± NA	9.39 ± 2.19
Consolidation period: Cycle 1 Day 6	7.09 ± NA	11.8 ± 1.91

SecondaryPhase 2 Part: Plasma Trough Concentration of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy

Ctrough is the plasma concentration prior to drug administration.

Time frame:: Induction period : Predose on Cycle 1 Day 15 and 21 Consolidation period: Predose on Cycle 1 Day 8 and 15
Reported as:: Mean · ng/mL

Phase 2 Part: Plasma Trough Concentration of Gilteritinib Concomitant With Induction and Consolidation Chemotherapy
ng/mL	Phase 2: FLT3-mutated AML
Induction Period: Cycle 1 Day 15	522 ± 331
Induction Period: Cycle 1 Day 21	647 ± 518
Consolidation Period: Cycle 1 Day 8	244 ± 152
Consolidation Period: Cycle 1 Day 15	375 ± 249

SecondaryPhase 2 Part: Overall Survival (OS)

Overall survival (OS) was defined as the time from the date of first dose of day 1 to the date of death due to any cause. Participants still alive or lost to follow up was censored at the time they were last known to be alive. Kaplan-Meier (KM) estimate was used for analysis.

Time frame:: From the date of first dose up to the date of death (maximum duration: approximately 4.4 years)
Reported as:: Median · Months

Phase 2 Part: Overall Survival (OS)
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Overall Survival (OS)	48.2 (45.1 to NA)

SecondaryPhase 2 Part: Event Free Survival (EFS)

Event-free survival (EFS): time from date of first dose of study regimen until date of documented relapse, treatment failure or death from any cause, whichever occurred first. KM estimate was used for analysis. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in bone marrow aspirate (BMA)/reappearance of significant numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%. Treatment failure: participants who failed to achieve composite complete remission (CRc) or who discontinued treatment due to "lack of efficacy" without previous response. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: defined in outcome measure #5.

Time frame:: From the date of first dose up to the date of documented relapse, treatment failure or death from any cause (maximum duration: approximately 4.4 years)
Reported as:: Median · Months

Phase 2 Part: Event Free Survival (EFS)
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Event Free Survival (EFS)	25.0 (11.3 to 48.2)

SecondaryPhase 2 Part: Relapse Free Survival (RFS)

Relapse-free survival (RFS): time from date of achievement of first CRc until relapse or death from any cause. KM estimate was used for analysis. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in bone marrow aspirate (BMA)/reappearance of significant numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%.

Time frame:: From the date of achievement of first CRc up to the date of documented relapse or death from any cause (maximum duration: approximately 3.9 years)
Reported as:: Median · Months

Phase 2 Part: Relapse Free Survival (RFS)
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Relapse Free Survival (RFS)	27.8 (17.4 to 39.8)

SecondaryPhase 2 Part: CR Rate After Consolidation and Maintenance Period

Percentage of participants with CR was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the consolidation period refers to the best response from the start of treatment in the induction period until the end of the consolidation period and for the maintenance period refers to the best response from the start of treatment in the induction period until the end of the maintenance period.

Time frame:: CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Reported as:: Number · Percentage of participants

Phase 2 Part: CR Rate After Consolidation and Maintenance Period
Percentage of participants	Phase 2: FLT3-mutated AML
After consolidation period	63.4 (52.0 to 73.8)
After maintenance period	63.4 (52.0 to 73.8)

SecondaryPhase 2 Part: CR Rate Without Minimal Residual Disease (MRD) After Each Treatment Therapy Period

CR% without MRD reported. CR rate without MRD after each treatment therapy was defined similarly as CR rate after each treatment therapy. Responders achieve that the best response is CR and MRD status was negative. CR was defined as a morphologically leukemia-free state, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.

Time frame:: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Reported as:: Number · Percentage of participants

Phase 2 Part: CR Rate Without Minimal Residual Disease (MRD) After Each Treatment Therapy Period
Percentage of participants	Phase 2: FLT3-mutated AML
After Induction Period	18.3 (10.6 to 28.4)
After Consolidation Period	35.4 (25.1 to 46.7)
After Maintenance Period	35.4 (25.1 to 46.7)

SecondaryPhase 2 Part: CR With Partial Hematological Recovery (CRh) Rate After Each Treatment Therapy Period

Percentage of participants with CRh was reported. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.

Time frame:: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Reported as:: Number · Percentage of participants

Phase 2 Part: CR With Partial Hematological Recovery (CRh) Rate After Each Treatment Therapy Period
Percentage of participants	Phase 2: FLT3-mutated AML
After Induction Period	17.1 (9.7 to 27.0)
After Consolidation Period	9.8 (4.3 to 18.3)
After Maintenance Period	9.8 (4.3 to 18.3)

SecondaryPhase 2 Part: Composite CR (CRc) Rate After Each Treatment Therapy Period

Percentage of participants with CRc reported. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recovery (CRi). CRp: met all CR criteria, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.

Time frame:: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Reported as:: Number · Percentage of participants

Phase 2 Part: Composite CR (CRc) Rate After Each Treatment Therapy Period
Percentage of participants	Phase 2: FLT3-mutated AML
After Induction Period	86.6 (77.3 to 93.1)
After Consolidation Period	87.8 (78.7 to 94.0)
After Maintenance Period	87.8 (78.7 to 94.0)

SecondaryPhase 2 Part: CR/CRh Rate After Each Treatment Therapy Period

Percentage of participants with CR/CRh was reported. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Derived response assessment after the IP refers to the best response from the start of treatment in the IP until the end of the IP, after CP refers to the best response from the start of treatment in the IP until the end of the CP and for the MP refers to the best response from the start of treatment in the IP until the end of the MP.

Time frame:: IP: From date of first dose up to end of period (approximately 1.4 years), CP: From date of first dose up to end of period (approximately 1.8 years), MP: From date of first dose up to the end of period (approximately 3.8 years)
Reported as:: Number · Percentage of participants

Phase 2 Part: CR/CRh Rate After Each Treatment Therapy Period
Percentage of participants	Phase 2: FLT3-mutated AML
After Induction Period	67.1 (55.8 to 77.1)
After Consolidation Period	73.2 (62.2 to 82.4)
After Maintenance Period	73.2 (62.2 to 82.4)

SecondaryPhase 2 Part: Duration of CR

Duration of CR was defined as the time from the date of achieving first CR until the date of first documented relapse for participants who achieved CR. KM estimate was used for analysis. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.

Time frame:: From the date of achieving CR up to the date of documented relapse (maximum duration: approximately 2.6 years)
Reported as:: Median · Months

Phase 2 Part: Duration of CR
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Duration of CR	NA (NA to NA)

SecondaryPhase 2 Part: Duration of CR/CRh

Duration of CR/CRh is defined as the time from the date of achieving first CR/CRh until the date of first documented relapse for participants who achieved CR/CRh. KM estimate was used for analysis. CR was defined as a morphologically leukemia-free state at the post-baseline visit, having a neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods and no evidence of extramedullary leukemia. The blast counts in peripheral blood was ≤ 2%. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.

Time frame:: From the date of achieving CR/CRh up to the date of documented relapse (maximum duration: approximately 2.6 years)
Reported as:: Median · Months

Phase 2 Part: Duration of CR/CRh
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Duration of CR/CRh	NA (NA to NA)

SecondaryPhase 2 Part: Duration of CRh

Duration of CRh was defined as the time from date of achieving first CRh until date of first documented relapse for participants who achieved CRh. KM estimate was used for analysis. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery neutrophil count≥ 500/mm\^3 and platelet count ≥ 50,000/mm\^3, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.

Time frame:: From the date of achieving CRh up to the date of documented relapse (maximum duration: approximately 2.6 years)
Reported as:: Median · Months

Phase 2 Part: Duration of CRh
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Duration of CRh	NA (NA to NA)

SecondaryPhase 2 Part: Duration of CRc

Duration of CRc is defined as the time from the date of achieving first CRc until the date of first documented relapse for participants who achieved CRc. KM estimate was used for analysis. CRc: rate of all complete \& incomplete remissions i.e. CR + CR with incomplete platelet recovery (CRp) + CR with incomplete hematological recover (CRi). CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 and platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Blast counts in peripheral blood was ≤ 2%. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%.

Time frame:: From the date of achieving CRc up to the date of documented relapse (maximum duration: approximately 2.7 years)
Reported as:: Median · Months

Phase 2 Part: Duration of CRc
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Duration of CRc	NA (NA to NA)

SecondaryPhase 2 Part: Duration of Response (DoR)

DoR: from first day of achieving CRc (CR+ CRp,+CRi)/partial remission (PR) to first day of relapse. KM estimate was used for analysis. CR: morphologically leukemia-free state at post-baseline visit, having neutrophil count of ≥ 1,000/mm\^3 \& platelet count of ≥ 100,000/mm\^3, bone marrow blasts \< 5%. No evidence of Auer rods \& extramedullary leukemia. Peripheral blood blast counts was ≤ 2%. CRp: met all CR criteria at post-baseline visit, except unrecovered platelet count (\< 100,000/mm\^3). CRi: met all CR criteria at post-baseline visit, except unrecovered neutrophil count (\< 1,000/mm\^3). PR: condition with regeneration of normal hematopoietic cells in bone marrow, no detectable blasts, ≥ 50% decrease of blasts in BMA \& total bone marrow blasts of 5-25%. No evidence of extramedullary leukemia. Relapse: reappearance of leukemic blasts in peripheral blood (\>2%)/≥ 5% blasts in BMA/reappearance of numbers of peripheral blasts and an increase in percentage of blasts in BMA to \> 25%

Time frame:: From the date of achieving CR, CRp, CRi/PR up to the date of documented relapse (maximum duration: approximately 2.7 years)
Reported as:: Median · Months

Phase 2 Part: Duration of Response (DoR)
Months	Phase 2: FLT3-mutated AML
Phase 2 Part: Duration of Response (DoR)	NA (NA to NA)

SecondaryPhase 2 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

An AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE could therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. TEAE for Phase 2 was defined as an AE observed after the date of first dose until 30 days after the last dose. TEAE included both serious and non-serious AEs.

Time frame:: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.4 years)
Reported as:: Number · Participants

Phase 2 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Participants	Phase 2: FLT3-mutated AML
Phase 2 Part: Number of Participants With Treatment Emergent Adverse Events (TEAEs)	84

Adverse events

Collected over Adverse events: Phase 1: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.1 years) Phase 2: From the date of first dose up to 30 days after the last dose ( maximum duration up to approximately 4.4 years) All-cause mortality: From randomization up to approximately 9.4 years.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Phase 1: Dose Evaluation (DEv)	0/3 (0%)	2/3 (66.7%)	3/3 (100%)
Phase 1: Dose Expansion (DEx)	0/10 (0%)	1/10 (10%)	10/10 (100%)
Phase 2: FLT3-mutated AML	25/84 (29.8%)	45/84 (53.6%)	84/84 (100%)

Most frequent serious events

Showing 10 of 56

Most frequent serious events
Event	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
Hepatic function abnormalHepatobiliary disorders	1/3	0/10	6/84
Liver function test abnormalInvestigations	1/3	1/10	0/84
PneumoniaInfections and infestations	0/3	0/10	9/84
SepsisInfections and infestations	0/3	0/10	9/84
Febrile neutropeniaBlood and lymphatic system disorders	0/3	0/10	5/84
PyrexiaGeneral disorders	0/3	0/10	4/84
Cardiac failureCardiac disorders	0/3	0/10	3/84
Septic shockInfections and infestations	0/3	0/10	3/84
Drug-induced liver injuryHepatobiliary disorders	0/3	0/10	2/84
Alanine aminotransferase increasedInvestigations	0/3	0/10	2/84

Most frequent other events

Showing 10 of 110

Most frequent other events
Event	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML
AnaemiaBlood and lymphatic system disorders	3/3	6/10	22/84
Febrile neutropeniaBlood and lymphatic system disorders	3/3	9/10	53/84
ThrombocytopeniaBlood and lymphatic system disorders	3/3	2/10	13/84
AlopeciaSkin and subcutaneous tissue disorders	2/3	9/10	15/84
White blood cell count decreasedInvestigations	1/3	7/10	15/84
LeukopeniaBlood and lymphatic system disorders	2/3	2/10	7/84
NeutropeniaBlood and lymphatic system disorders	2/3	3/10	10/84
NauseaGastrointestinal disorders	2/3	1/10	27/84
OedemaGeneral disorders	2/3	0/10	5/84
PneumoniaInfections and infestations	2/3	5/10	14/84

Baseline characteristics

Safety Analysis Set (SAF): All participants who received at least 1 dose of study drug were included.

Age, Continuous
Age, Continuous(Years)	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML	Total
Mean	48.3 ± 14	49.9 ± 15	51 ± 14.4	50.8 ± 14.3

Sex: Female, Male
Sex: Female, Male(Participants)	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML	Total
Female	2	3	43	48
Male	1	7	41	49

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML	Total
Hispanic or Latino	0	0	0	0
Not Hispanic or Latino	3	10	84	97
Unknown or Not Reported	0	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Phase 1: Dose Evaluation (DEv)	Phase 1: Dose Expansion (DEx)	Phase 2: FLT3-mutated AML	Total
American Indian or Alaska Native	0	0	0	0
Asian	3	10	84	97
Native Hawaiian or Other Pacific Islander	0	0	0	0
Black or African American	0	0	0	0
White	0	0	0	0
More than one race	0	0	0	0
Unknown or Not Reported	0	0	0	0

Study locations

55 sites

Site JP81037
Anjo, Aichi-ken, Japan
Site JP00003
Nagoya, Aichi-ken, Japan
Site JP81003
Nagoya, Aichi-ken, Japan
Site JP81027
Nagoya, Aichi-ken, Japan
Site JP81038
Toyohashi, Aichi-ken, Japan
Site JP81010
Narita, Chiba, Japan
Site JP81039
Matsuyama, Ehime, Japan
Site JP81007
Yoshida-gun, Fukui, Japan
Site JP00002
Maebashi, Gunma, Japan
Site JP81001
Maebashi, Gunma, Japan
Site JP81026
Fukuyama, Hiroshima, Japan
Site JP81018
Ōtake, Hiroshima, Japan
Site JP81014
Sapporo, Hokkaido, Japan
Site JP81015
Sapporo, Hokkaido, Japan
Site JP81043
Himeji, Hyōgo, Japan
Site JP00007
Kobe, Hyōgo, Japan
Site JP81006
Kobe, Hyōgo, Japan
Site JP81036
Mito, Ibaraki, Japan
Site JP81023
Tsukuba, Ibaraki, Japan
Site JP81020
Kanazawa, Ishikawa-ken, Japan
Site JP81013
Isehara, Kanagawa, Japan
Site JP00006
Yokohama, Kanagawa, Japan
Site JP81005
Yokohama, Kanagawa, Japan
Site JP81024
Yokohama, Kanagawa, Japan
Site JP81035
Sendai, Miyagi, Japan
SIte JP81011
Ōmura, Nagasaki, Japan
Site JP81041
Tenri, Nara, Japan
Site JP81022
Shimono, Tochigi, Japan
Site JP81040
Bunkyo-ku, Tokyo, Japan
Site JP00005
Shinagawa-ku, Tokyo, Japan
Site JP81032
Shinagawa-ku, Tokyo, Japan
Site JP81029
Akita, Japan
Site JP81008
Chiba, Japan
Site JP00001
Fukuoka, Japan
Site JP81004
Fukuoka, Japan
Site JP81025
Fukuoka, Japan
Site JP81031
Fukushima, Japan
Site JP81030
Gifu, Japan
Site JP81033
Kochi, Japan
Site JP81028
Kumamoto, Japan
Site JP81016
Kyoto, Japan
Site JP81012
Nagasaki, Japan
Site JP81009
Okayama, Japan
Site JP81019
Osaka, Japan
Site JP81021
Osaka, Japan
Site KR82005
Busan, South Korea
Site KR82002
Incheon, South Korea
Site KR82001
Seoul, South Korea
Site KR82003
Seoul, South Korea
Site KR82004
Seoul, South Korea
Site KR82006
Seoul, South Korea
Site TW88604
Kaohsiung City, Taiwan
Site TW88602
Taichung, Taiwan
Site TW88601
Tainan, Taiwan
Site TW88603
Taoyuan, Taiwan

References and documents

Publications

Sawa M, Miyamoto T, Kim HJ, Hiramatsu Y, Cheong JW, Ikezoe T, Naoe T, Akashi K, Morita S, Kosako M, Shimura M, Terada W, Kadokura T, Hill J, Miyawaki S, Gill SC, Heinloth A, Hasabou N. A phase I/II study of gilteritinib in combination with chemotherapy in newly diagnosed patients with AML in Asia: final analysis. Ther Adv Hematol. 2026 Mar 11;17:20406207261419953. doi: 10.1177/20406207261419953. eCollection 2026. PubMed 41835842 ↗

Study documents

Study protocol · Aug 29, 2021
Statistical analysis plan · Nov 16, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Supporting information: Study protocol, Sap, Csr

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Sep 2, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02310321
Lead sponsor: Astellas Pharma Inc
Responsible party: Sponsor
First posted: Dec 8, 2014
Start date: Feb 26, 2015
Primary completion: Aug 25, 2021
Completion: Jul 9, 2024
Results posted: Oct 16, 2024
Last update: Sep 2, 2025

Study contacts

Medical Director

study director · Astellas Pharma Inc

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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