Trial Outcomes & Findings for A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase 3 (FLT3) Mutation (NCT NCT03182244)

Last Updated: 2025-10-27

Results Overview

OS was defined as the time from the date of randomization to the date of death due to any cause. Participants who were still alive or lost to follow up were censored at the time they were last known to be alive. Kaplan-Meier (KM) estimates was used for analysis.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

276 participants

Primary outcome timeframe

From the date of randomization up to the date of death (up to approximatley 74 months)

Results posted on

2025-10-27

Participant Flow

Participants with FMS-like tyrosine kinase 3 (FLT3) mutations with relapsed or refractory Acute Myeloid Leukemia (AML) after first-line therapy were enrolled for this study. 21 participants from six china sites were included in Giltertinib PK cohort.

Randomization was stratified by response to first-line therapy and preselected salvage chemotherapy. Results were reported up to primary analysis data cut-off of 25 December 2023 (approximately 74 months).

Participant milestones

Participant milestones
Measure	Gilteritinib Participants received 120 milligrams (mg) gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: Low dose cytarabine (LoDAC): 20 mg cytarabine administered twice daily by subcutaneous (SC)/intravenous (IV) injection for 10 days. Mitoxantrone, etoposide and intermediate dose cytarabine (MEC): mitoxantrone 6 milligrams per square meter(mg/m\^2) per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). Fludarabine, cytarabine and granulocyte colony-stimulating factor (FLAG): granulocyte colony-stimulating factor (G-CSF) 300 micrograms per square meter (μg/m\^2) per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter crossover extension (COE) period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Treatment Period (Upto 1917 Days) STARTED	137	139
Treatment Period (Upto 1917 Days) LoDAC	0	27
Treatment Period (Upto 1917 Days) MEC	0	42
Treatment Period (Upto 1917 Days) FLAG	0	70
Treatment Period (Upto 1917 Days) Chinese PK Cohort	21	0
Treatment Period (Upto 1917 Days) COMPLETED	0	26
Treatment Period (Upto 1917 Days) NOT COMPLETED	137	113
Long Term Follow-up (Up to 1232 Days) STARTED	100	105
Long Term Follow-up (Up to 1232 Days) COMPLETED	9	12
Long Term Follow-up (Up to 1232 Days) NOT COMPLETED	91	93
Cross Over Extension (COE) Period STARTED	0	0
Cross Over Extension (COE) Period COMPLETED	0	0
Cross Over Extension (COE) Period NOT COMPLETED	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Gilteritinib Participants received 120 milligrams (mg) gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: Low dose cytarabine (LoDAC): 20 mg cytarabine administered twice daily by subcutaneous (SC)/intravenous (IV) injection for 10 days. Mitoxantrone, etoposide and intermediate dose cytarabine (MEC): mitoxantrone 6 milligrams per square meter(mg/m\^2) per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). Fludarabine, cytarabine and granulocyte colony-stimulating factor (FLAG): granulocyte colony-stimulating factor (G-CSF) 300 micrograms per square meter (μg/m\^2) per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter crossover extension (COE) period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Treatment Period (Upto 1917 Days) Adverse Event	8	9
Treatment Period (Upto 1917 Days) Death	19	8
Treatment Period (Upto 1917 Days) Miscellaneous	12	0
Treatment Period (Upto 1917 Days) Disease Relapse	41	5
Treatment Period (Upto 1917 Days) Physician Decision	5	10
Treatment Period (Upto 1917 Days) Withdrawal by Subject	10	28
Treatment Period (Upto 1917 Days) Protocol Violation	4	1
Treatment Period (Upto 1917 Days) Progressive Disease	18	9
Treatment Period (Upto 1917 Days) Lack of Efficacy	20	43
Long Term Follow-up (Up to 1232 Days) Miscellaneous	4	3
Long Term Follow-up (Up to 1232 Days) Withdrawal by Subject	5	8
Long Term Follow-up (Up to 1232 Days) Lost to Follow-up	6	7
Long Term Follow-up (Up to 1232 Days) Death	76	75

Baseline Characteristics

A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase 3 (FLT3) Mutation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.	Total n=276 Participants Total of all reporting groups
Age, Continuous	46.9 Years STANDARD_DEVIATION 16.2 • n=5 Participants	46.2 Years STANDARD_DEVIATION 15.1 • n=7 Participants	46.6 Years STANDARD_DEVIATION 15.7 • n=5 Participants
Sex: Female, Male Female	76 Participants n=5 Participants	70 Participants n=7 Participants	146 Participants n=5 Participants
Sex: Female, Male Male	61 Participants n=5 Participants	69 Participants n=7 Participants	130 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	136 Participants n=5 Participants	139 Participants n=7 Participants	275 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	123 Participants n=5 Participants	120 Participants n=7 Participants	243 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants	19 Participants n=7 Participants	33 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment China	90 participants n=5 Participants	92 participants n=7 Participants	182 participants n=5 Participants
Region of Enrollment Russia	14 participants n=5 Participants	19 participants n=7 Participants	33 participants n=5 Participants
Region of Enrollment Malaysia	15 participants n=5 Participants	14 participants n=7 Participants	29 participants n=5 Participants
Region of Enrollment Thailand	14 participants n=5 Participants	10 participants n=7 Participants	24 participants n=5 Participants
Region of Enrollment Singapore	4 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants
Response to First-Line Therapy Relapse within 6 months after allogeneic HSCT	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Response to First-Line Therapy Relapse after 6 months after allogeneic HSCT	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Response to First-Line Therapy Primary refractory without HSCT	80 Participants n=5 Participants	81 Participants n=7 Participants	161 Participants n=5 Participants
Response to First-Line Therapy Relapse within 6 months after CRc and no HSCT	31 Participants n=5 Participants	29 Participants n=7 Participants	60 Participants n=5 Participants
Response to First-Line Therapy Relapse after 6 months after CRc and no HSCT	23 Participants n=5 Participants	23 Participants n=7 Participants	46 Participants n=5 Participants
Preselected Salvage Chemotherapy High intensity chemotherapy	110 Participants n=5 Participants	112 Participants n=7 Participants	222 Participants n=5 Participants
Preselected Salvage Chemotherapy Low intensity chemotherapy	27 Participants n=5 Participants	27 Participants n=7 Participants	54 Participants n=5 Participants

PRIMARY outcome

Timeframe: From the date of randomization up to the date of death (up to approximatley 74 months)

Population: ITT

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Overall Survival (OS)	10.3 months Interval 8.8 to 12.7	5.4 months Interval 4.1 to 8.1

SECONDARY outcome

Timeframe: From the date of randomization up to the date of documented relapse, treatment failure or death from any cause, off-treatment relapse and start of new AML therapy (up to approximately 74 months)

Population: ITT

EFS: time from the date of randomization until the date of documented relapse, treatment failure, death, reported off treatment relapse or new AML therapy start whichever occued first, including the long-term follow-up data. KM estimate was used for analysis. Relapse was defined as documentation of any of following events: * Bone marrow (BM) blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts Treatment failure: Treatment failure was defined as participant who ends treatment without having a previous response of CR, CR with incomplete platelet recovery (CRp) and CR with incomplete hematological recover (CRi).

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Event-Free Survival (EFS)	2.1 months Interval 0.1 to 3.2	0.6 months Interval 0.2 to 1.2

SECONDARY outcome

Timeframe: From the date of randomization up to approximately 74 months

Population: ITT

Percentage of participants with CR were reported. CR: morphologically leukemia-free state, with absolute neutrophil count (ANC) \> 1x10\^9 per liter (1x10\^9/L), platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were red blood cell (RBC) and platelet transfusion independent and no evidence of extramedullary leukemia or Auer rods was necessary.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Complete Remission (CR) Rate	20.4 Percentage of participants Interval 14.0 to 28.2	11.5 Percentage of participants Interval 6.7 to 18.0

SECONDARY outcome

Timeframe: From date of achieving CR until date of confirmed relapse (maximum duration was 53.4 months )

Population: ITT. Participants with CR were analyzed.

Duration of CR: time from the date of achieving first CR until date of first documented relapse for participants who achieved CR. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. Relapse was defined as documentation of any of following events: * BM blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts

Outcome measures

Outcome measures
Measure	Gilteritinib n=28 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=16 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Duration of CR	24.0 months Interval 4.6 to Upper limit of confidence interval was not estimable due to an insufficient number of events to determine 50% survival.	NA months Interval 1.0 to Median and upper limit of confidence interval was not estimable due to an insufficient number of events to determine 50% survival.

SECONDARY outcome

Timeframe: From date of achieving CRc until date of confirmed relapse (maximum duration was 60 months)

Population: ITT. Participants with best overall response of CRc were analyzed.

Duration of CRc: time from date of achieving first CRc until date of first documented relapse for participants who achieved CRc. KM estimate was used for analysis. CRc: rate of all complete \& incomplete remissions \[CR + CRp + CRi\]. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.

Outcome measures

Outcome measures
Measure	Gilteritinib n=73 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=31 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Duration of Composite Complete Remission (CRc)	4.1 months Interval 2.8 to 5.6	NA months Median, upper and lower limit of confidence interval were not estimable due to an insufficient number of events to determine 50% survival.

SECONDARY outcome

Timeframe: From date of achieving CR/CRh until date of confirmed relapse (maximum duration was 58.1 months)

Population: ITT. Participants with CR/CRh were analyzed.

Duration of CR/CRh: time from date of achieving first CR/CRh until date of first documented relapse for participants who achieved CR/CRh. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.

Outcome measures

Outcome measures
Measure	Gilteritinib n=44 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=20 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Duration of CR/Complete Remission With Partial Hematologic Recovery (CRh)	5.6 months Interval 2.8 to 24.0	NA months Upper and lower limits of confidence interval was not estimable due to an insufficient number of events to determine 50% survival.

SECONDARY outcome

Timeframe: From date of achieving CRc/PR until date of confirmed relapse (maximum duration was 52.1 months)

Population: ITT. Participants with best overall response of CRc/PR were analyzed.

DOR: time from date of first CRc (CR+CRp+CRi)/PR until date of first documented relapse for participants who achieved CRc or PR. KM estimate used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts and increase in the percentage of blasts in the BM aspirate to \> 25%.

Outcome measures

Outcome measures
Measure	Gilteritinib n=93 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=38 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Duration Of Response (DOR)	3.7 months Interval 2.5 to 4.7	NA months Interval 1.2 to Median and lower limit of confidence interval was not estimable due to an insufficient number of events to determine 50% survival.

SECONDARY outcome

Timeframe: From the date of randomization up to approximately 74 months

Population: ITT

Percentage of participants with CR/CRh was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
CR/CRh Rate	32.1 Percentage of participants Interval 24.4 to 40.6	14.4 Percentage of participants Interval 9.0 to 21.3

SECONDARY outcome

Timeframe: From the date of randomization up to approximately 74 months

Population: ITT

Defined as percentage of participants with CR, CRp, CRi, PR, no response (NR) \& not estimable (NE). CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L \& normal marrow differential with \< 5% blasts. They were RBC \& platelet transfusion independent \& no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\<100x10\^9/L). CRi: met all CR criteria, except incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with/without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. \<=5% BM blasts if Auer rods are present, no evidence of extramedullary leukemia. Not Estimable (NE): No BM assessed/no myeloblast value, no blast value from peripheral blood or ≤2%, \& no extramedullary leukemia. No Response (NR): Response not categorized as CR, CRp, CRi, PR or NE.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Best Response Rate PR	14.6 Percentage of participants	5.0 Percentage of participants
Best Response Rate CRp	13.1 Percentage of participants	0.7 Percentage of participants
Best Response Rate CR	20.4 Percentage of participants	11.5 Percentage of participants
Best Response Rate CRi	19.7 Percentage of participants	10.1 Percentage of participants
Best Response Rate NR	27.7 Percentage of participants	34.5 Percentage of participants
Best Response Rate NE	4.4 Percentage of participants	38.1 Percentage of participants

SECONDARY outcome

Timeframe: From first day of achieving first CRc to the first day of confirmed relapse/death (maximum duration was 60.0 months)

Population: ITT. Participants with best overall response of CRc were analyzed.

LFS: time from the date of first CRc (CR+CRp+CRi) until the date of documented relapse or death for participants who achieved CRc. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.

Outcome measures

Outcome measures
Measure	Gilteritinib n=73 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=31 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Leukemia-Fee Survival (LFS)	3.9 months Interval 2.8 to 5.4	6.9 months Interval 2.9 to 10.4

SECONDARY outcome

Timeframe: From the date of randomization up to approximately 74 months

Population: ITT

Percentage of participants with CRc (CR+CRp+CRi) was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Composite Complete Remission (CRc)	53.3 Percentage of participants Interval 44.6 to 61.9	22.3 Percentage of participants Interval 15.7 to 30.1

SECONDARY outcome

Timeframe: From randomization until date of first CRc (up to approximately 74 months)

Population: ITT population. Participants who achieved CRc were analyzed.

Time to CRc (TTCRc) was defined as the time from the date of randomization until the date of first CRc. CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.

Outcome measures

Outcome measures
Measure	Gilteritinib n=73 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=31 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Time to CRc	1.8 months Interval 1.0 to 8.0	1.0 months Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: From randomization until date of first CR (up to approximately 74 months)

Population: ITT population. Participants who achieved CR were analyzed.

Time to CR (TTCR) was defined as the time from the date of randomization until the date of first CR. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia.

Outcome measures

Outcome measures
Measure	Gilteritinib n=28 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=16 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Time to CR	3.7 months Interval 1.0 to 19.0	1.1 months Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: From randomization until date of first CRc or PR (up to approximately 74 months)

Population: ITT population. Participants who achieved CRc or PR were analyzed.

Time to Response (TTR) was defined as the time from the date of randomization until the date of either first response (CRc or PR). CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%.

Outcome measures

Outcome measures
Measure	Gilteritinib n=93 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=38 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Time to Response	1.0 months Interval 1.0 to 7.0	1.0 months Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: From randomization until date of first CR/CRh (up to approximately 74 months)

Population: ITT population. Participants who achieved CR/CRh were analyzed.

Time to CR/CRh (TTCRCRh) was defined as the time from the date of randomization until the date of first CR/CRh. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%.

Outcome measures

Outcome measures
Measure	Gilteritinib n=44 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=20 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Time to CR/CRh	2.8 months Interval 1.0 to 11.0	1.1 months Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: Baseline up to approximately 74 months

Population: ITT population with available data at specified timepoint. This endpoint was planned to be analyzed only for Giltertinib arm.

Transfusion conversion rate: Percentage of transfusion dependent participants at baseline period but became transfusion independent at post-baseline period divided by total participants who were transfusion dependent at baseline period. Transfusion maintenance rate: Percentage of transfusion independent participants at baseline period and still maintained transfusion independent at post-baseline period divided by total participants who were transfusion independent at baseline period. Baseline transfusion status: Participants were classified as transfusion independent if there were no RBC or platelet transfusions within 28 days prior to first dose to 28 days after first dose; otherwise classified as transfusion dependent. Post baseline transfusion status: Participants on treatment ≥ 84 days were classified as transfusion independent, if consecutive 56 days without any RBC or platelet transfusion; otherwise classified as transfusion dependent

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Percentage of Participants With Transfusion Conversion and Transfusion Maintenance Transfusion Conversion Rate	42.7 Percentage of participants	—
Percentage of Participants With Transfusion Conversion and Transfusion Maintenance Transfusion Maintenance Rate	70.8 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline up to approximately 74 months

Population: ITT

Transplantation rate is defined as the percentage of participants undergoing Hematopoietic stem cell transplant (HSCT) during the study period.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=139 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Percentage of Participants With Transplantation Rate	22.6 Percentage of participants Interval 15.9 to 30.6	7.9 Percentage of participants Interval 4.0 to 13.7

SECONDARY outcome

Timeframe: Baseline, End of treatment (63 months)

Population: ITT population with data available at specified timepoint.

The BFI is a screening tool designed to assess the severity and impact of fatigue on daily functioning of participants with cancer during the 24 hours. There are 9 items on the scale. The first three questions ask participants to rate their fatigues on a scale from 0 (no fatigue) - 10 (as bad as you can imagine), with higher scores indicating worse outcome. The remaining six questions ask participants to rate how much fatigue has interfered with their daily activities on a scale from 0 (Does not interfere) to 10 (Completely interferes). A global fatigue score can be obtained by averaging all the items on the BFI, ranging between 0 to 10; a higher BFI fatigue score indicates worse outcome. The global BFI scores were calculated only if at least 5 of the 9 items are answered.

Outcome measures

Outcome measures
Measure	Gilteritinib n=84 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=89 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Change From Baseline in Brief Fatigue Inventory (BFI)	1.42 Scores on scale Standard Deviation 2.99	0.75 Scores on scale Standard Deviation 2.92

SECONDARY outcome

Timeframe: From the date of first dose up to approximately 74 months

Population: Safety Analysis Set (SAF): The SAF consisted of all participants who received at least 1 dose of study treatment (gilteritinib or salvage chemotherapy).

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 was defined as an adverse event observed after starting administration of the study drug.

Outcome measures

Outcome measures
Measure	Gilteritinib n=134 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=119 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	134 Participants	119 Participants

SECONDARY outcome

Timeframe: Baseline, end of treatment visit (63 months)

Population: SAF population with available data at specified timepoint.

The ECOG Scale was used to assess performance status. Number of participants with each grade was reported. Grade Description: 0: Fully active, able to carry on all pre-disease performance without restriction. 1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work. 2. Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours. 3. Capable of only limited self-care, confined to bed or chair more than 50% of waking hours. 4. Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair. 5. Dead.

Outcome measures

Outcome measures
Measure	Gilteritinib n=134 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy n=119 Participants Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 0 at baseline	35 Participants	37 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 1 at baseline	65 Participants	64 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 2 at baseline	34 Participants	18 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 3 at baseline	0 Participants	0 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 4 at baseline	0 Participants	0 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 5 at baseline	0 Participants	0 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 0 at End of Treatment	17 Participants	26 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 1 at End of Treatment	36 Participants	39 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 2 at End of Treatment	19 Participants	21 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 3 at End of Treatment	10 Participants	6 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 4 at End of Treatment	1 Participants	2 Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores Grade 5 at End of Treatment	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Cycle 1 Day 1(C1D1): predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose, Cycle 1 Day 15(C1D15): predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose

Population: Pharmacokinetics Analysis Set (PKAS): The PKAS consisted of all participants who received at least 1 administration of study intervention for which at least 1 concentration data with time of dosing and sampling were available. This endpoint was planned to be analyzed only for participants in Giltertinib PK cohort which included 21 participants from six china sites.

AUC24 was derived from the PK samples collected.

Outcome measures

Outcome measures
Measure	Gilteritinib n=21 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Pharmacokinetics (PK) of Gilteritinib in Chinese PK Cohort: Area Under the Concentration Curve at 24 Hours (AUC24) C1D1	3230 nanogramhour per milliliters(ngh/mL) Standard Deviation 1970	—
Pharmacokinetics (PK) of Gilteritinib in Chinese PK Cohort: Area Under the Concentration Curve at 24 Hours (AUC24) C1D15	10000 nanogramhour per milliliters(ngh/mL) Standard Deviation 6670	—

SECONDARY outcome

Timeframe: C1D1: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose, C1D15: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose

Population: PKAS. This endpoint was planned to be analysed only for participants in Giltertinib PK cohort which included 21 participants from six china sites.

Cmax was derived from the PK samples collected.

Outcome measures

Outcome measures
Measure	Gilteritinib n=21 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
PK of Gilteritinib in Chinese PK Cohort: Maximum Concentration (Cmax) C1D1	206 ng/mL Standard Deviation 112	—
PK of Gilteritinib in Chinese PK Cohort: Maximum Concentration (Cmax) C1D15	536 ng/mL Standard Deviation 323	—

SECONDARY outcome

Timeframe: Predose on C1D15

Population: PKAS with available data at timepoint were analyzed. This endpoint was planned to be analyzed only for participants at the China site in the Giltertinib PK cohort.

Ctrough was derived from the PK samples collected.

Outcome measures

Outcome measures
Measure	Gilteritinib n=15 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
PK of Gilteritinib: Observed Trough Concentration (Ctrough)	323 ng/mL Standard Deviation 222	—

SECONDARY outcome

Timeframe: C1D1: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hours (+/- 20 minutes) post dose, C1D15: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hours (+/- 20 minutes) post dose

Population: PKAS. This endpoint was planned to be analysed only for participants in Giltertinib PK cohort which included 21 participants from six china sites.

tmax was derived from the PK samples collected.

Outcome measures

Outcome measures
Measure	Gilteritinib n=21 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
PK of Gilteritinib in Chinese PK Cohort: Time to Maximum Concentration (Tmax) Cycle 1 Day 1	4.00 hours Interval 2.0 to 24.1	—
PK of Gilteritinib in Chinese PK Cohort: Time to Maximum Concentration (Tmax) Cycle 1 Day 15	3.85 hours Interval 1.0 to 24.3	—

SECONDARY outcome

Timeframe: Predose C1D8, C1D15, Day 1 of each cycle from C2 to C65, C67D1,C68D1,C69D1,C70D1

Population: PKAS with available data at timepoint were analyzed. This endpoint was planned to be analyzed only for participants at the China site in the Giltertinib PK cohort.

Concentrations below the lower limit of quantification (0.5 ng/mL) were set to zero.

Outcome measures

Outcome measures
Measure	Gilteritinib n=137 Participants Participants received 120 mg gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.	Salvage Chemotherapy Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines. Salvage chemotherapy included: LoDAC: 20 mg cytarabine administered twice daily by SC/IV injection for 10 days. MEC: mitoxantrone 6 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), etoposide 100 mg/m\^2 per day administered by IV for 5 days (days 1 through 5), cytarabine 1000 mg/m\^2 per day administered by IV for 5 days (days 1 through 5). FLAG: G-CSF 300 μg/m\^2 per day administered by SC/IV for 5 days (days 1 through 5), fludarabine 30 mg/m\^2 per day administered by IV for 5 days (days 2 through 6), cytarabine 2000 mg/m\^2 per day administered by IV for 5 days (days 2 through 6). Participants who met treatment discontinuation criteria, entered long-term follow-up and remained in the period for up to 3 years from the participant's end of treatment visit/until discontinuation from study. Based on the outcome of interim analysis, at investigators discretion, participants in the treatment period had the option to enter COE period to receive 120 mg gilteritinib, orally, once a day in continuous 28-day cycles until treatment discontinuation criteria was met.
Ctrough Concentration of Gilteritinib C61D1	292 ng/mL	—
Ctrough Concentration of Gilteritinib C62D1	287 ng/mL	—
Ctrough Concentration of Gilteritinib C69D1	345 ng/mL	—
Ctrough Concentration of Gilteritinib C70D1	313 ng/mL	—
Ctrough Concentration of Gilteritinib C1D8	310 ng/mL Standard Deviation 177	—
Ctrough Concentration of Gilteritinib CID15	367 ng/mL Standard Deviation 243	—
Ctrough Concentration of Gilteritinib C2D1	425 ng/mL Standard Deviation 305	—
Ctrough Concentration of Gilteritinib C3D1	457 ng/mL Standard Deviation 358	—
Ctrough Concentration of Gilteritinib C4D1	420 ng/mL Standard Deviation 428	—
Ctrough Concentration of Gilteritinib C5D1	407 ng/mL Standard Deviation 487	—
Ctrough Concentration of Gilteritinib C6D1	284 ng/mL Standard Deviation 275	—
Ctrough Concentration of Gilteritinib C7D1	370 ng/mL Standard Deviation 477	—
Ctrough Concentration of Gilteritinib C8D1	328 ng/mL Standard Deviation 323	—
Ctrough Concentration of Gilteritinib C9D1	351 ng/mL Standard Deviation 381	—
Ctrough Concentration of Gilteritinib C10D1	322 ng/mL Standard Deviation 383	—
Ctrough Concentration of Gilteritinib C11D1	336 ng/mL Standard Deviation 426	—
Ctrough Concentration of Gilteritinib C12D1	460 ng/mL Standard Deviation 703	—
Ctrough Concentration of Gilteritinib C13D1	368 ng/mL Standard Deviation 409	—
Ctrough Concentration of Gilteritinib C14D1	386 ng/mL Standard Deviation 449	—
Ctrough Concentration of Gilteritinib C15D1	396 ng/mL Standard Deviation 541	—
Ctrough Concentration of Gilteritinib C16D1	200 ng/mL Standard Deviation 116	—
Ctrough Concentration of Gilteritinib C17D1	228 ng/mL Standard Deviation 138	—
Ctrough Concentration of Gilteritinib C18D1	201 ng/mL Standard Deviation 143	—
Ctrough Concentration of Gilteritinib C19D1	231 ng/mL Standard Deviation 270	—
Ctrough Concentration of Gilteritinib C20D1	241 ng/mL Standard Deviation 190	—
Ctrough Concentration of Gilteritinib C21D1	221 ng/mL Standard Deviation 112	—
Ctrough Concentration of Gilteritinib C22D1	238 ng/mL Standard Deviation 149	—
Ctrough Concentration of Gilteritinib C23D1	152 ng/mL Standard Deviation 137	—
Ctrough Concentration of Gilteritinib C24D1	188 ng/mL Standard Deviation 141	—
Ctrough Concentration of Gilteritinib C25D1	192 ng/mL Standard Deviation 137	—
Ctrough Concentration of Gilteritinib C26D1	264 ng/mL Standard Deviation 138	—
Ctrough Concentration of Gilteritinib C27D1	199 ng/mL Standard Deviation 145	—
Ctrough Concentration of Gilteritinib C28D1	243 ng/mL Standard Deviation 141	—
Ctrough Concentration of Gilteritinib C29D1	184 ng/mL Standard Deviation 138	—
Ctrough Concentration of Gilteritinib C30D1	340 ng/mL Standard Deviation 472	—
Ctrough Concentration of Gilteritinib C31D1	426 ng/mL Standard Deviation 606	—
Ctrough Concentration of Gilteritinib C32D1	301 ng/mL Standard Deviation 395	—
Ctrough Concentration of Gilteritinib C33D1	431 ng/mL Standard Deviation 646	—
Ctrough Concentration of Gilteritinib C34D1	405 ng/mL Standard Deviation 590	—
Ctrough Concentration of Gilteritinib C35D1	298 ng/mL Standard Deviation 420	—
Ctrough Concentration of Gilteritinib C36D1	264 ng/mL Standard Deviation 343	—
Ctrough Concentration of Gilteritinib C37D1	437 ng/mL Standard Deviation 823	—
Ctrough Concentration of Gilteritinib C38D1	78.0 ng/mL Standard Deviation 76.3	—
Ctrough Concentration of Gilteritinib C39D1	107 ng/mL Standard Deviation 91.3	—
Ctrough Concentration of Gilteritinib C40D1	114 ng/mL Standard Deviation 59.2	—
Ctrough Concentration of Gilteritinib C41D1	152 ng/mL Standard Deviation 123	—
Ctrough Concentration of Gilteritinib C42D1	128 ng/mL Standard Deviation 150	—
Ctrough Concentration of Gilteritinib C43D1	96.9 ng/mL Standard Deviation 114	—
Ctrough Concentration of Gilteritinib C44D1	148 ng/mL Standard Deviation 145	—
Ctrough Concentration of Gilteritinib C45D1	166 ng/mL Standard Deviation 97.9	—
Ctrough Concentration of Gilteritinib C46D1	105 ng/mL Standard Deviation 127	—
Ctrough Concentration of Gilteritinib C47D1	116 ng/mL Standard Deviation 104	—
Ctrough Concentration of Gilteritinib C48D1	131 ng/mL Standard Deviation 107	—
Ctrough Concentration of Gilteritinib C49D1	74.4 ng/mL Standard Deviation 82.0	—
Ctrough Concentration of Gilteritinib C50D1	134 ng/mL Standard Deviation 119	—
Ctrough Concentration of Gilteritinib C51D1	130 ng/mL Standard Deviation 136	—
Ctrough Concentration of Gilteritinib C52D1	93.4 ng/mL Standard Deviation 114	—
Ctrough Concentration of Gilteritinib C53D1	148 ng/mL Standard Deviation 188	—
Ctrough Concentration of Gilteritinib C54D1	166 ng/mL Standard Deviation 246	—
Ctrough Concentration of Gilteritinib C55D1	129 ng/mL Standard Deviation 169	—
Ctrough Concentration of Gilteritinib C56D1	157 ng/mL Standard Deviation 227	—
Ctrough Concentration of Gilteritinib C57D1	380 ng/mL	—
Ctrough Concentration of Gilteritinib C58D1	338 ng/mL	—
Ctrough Concentration of Gilteritinib C59D1	451 ng/mL	—
Ctrough Concentration of Gilteritinib C60D1	362 ng/mL	—
Ctrough Concentration of Gilteritinib C63D1	411 ng/mL	—
Ctrough Concentration of Gilteritinib C65D1	362 ng/mL	—
Ctrough Concentration of Gilteritinib C67D1	326 ng/mL	—
Ctrough Concentration of Gilteritinib C68D1	584 ng/mL	—

Adverse Events

Gilteritinib

Serious events: 102 serious events

Other events: 133 other events

Deaths: 97 deaths

Salvage Chemotherapy

Serious events: 71 serious events

Other events: 113 other events

Deaths: 85 deaths

Serious adverse events

Other adverse events

Additional Information

Clinical Transparency

Astellas Pharma Inc

Phone: +81 3-3244-6500 Japanese only

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Institute and/or Principal Investigator may publish trial data generated at their specific study site after Sponsor publication of the multi-center data. Sponsor must receive a site's manuscript prior to publication for review and comment as specified in the Investigator Agreement.
Publication restrictions are in place

Restriction type: OTHER