Trial Outcomes & Findings for A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML) (NCT NCT04240002)

NCT ID: NCT04240002

Last Updated: 2026-01-05

Results Overview

MTD reflects the highest dose that did not cause a Dose Limiting Toxicity (DLT).DLT:any Grade ≥3 non-hematologic/extramedullary toxicity with the following exceptions that occurred during the DLT observations like alopecia, anorexia, or fatigue, grade 3 vomiting or diarrhea that resolved(with or without supportive care) to ≤ grade 2 within 48 hours, grade 3 nausea that resolved(with or without supportive care) to ≤ grade 2 within 7 days, grade 3 elevation in total bilirubin (TBL) that is asymptomatic and that returned to ≤ grade 2 elevation within 7 days, grade 3 elevation in hepatic transaminases\[alanine aminotransferase (ALT/SGPT) aspartate aminotransferase (AST/SGOT) and gammaglutamyl transferase (GGT)\] or Alkaline Phosphatase (ALP) level that returns to ≤ grade 2 elevation within 14 days, grade 3 fever with neutropenia, with/without infection, grade 3 infection/grade 4 infections expected as direct complication of cytopenia due to active underlying leukemia, grade 3 mucositis.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 9 participants
• Primary outcome timeframe: C1D1 up to day 28
• Results posted on: 2026-01-05

Participant Flow

Participants positive for FMS-like tyrosine kinase 3 (FLT3) [internal tandem duplication (ITD) and/or tyrosine kinase domain (TKD)] mutation in bone marrow or blood and positive for the FLT3 (ITD) mutation in bone marrow or blood were enrolled for phase 1 and phase 2 respectively.

The sponsor decided not to open the phase 2 extension part of the study and terminated the study after completion of the phase 1 dose escalation part for Group 1, hence, no participants were enrolled for dose escalation groups 2 and 3. The decision to terminate the study was based upon enrollment challenges due to the rarity of Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) in children and not related to any safety or efficacy issues.

Participant milestones

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) Participants aged 2 years to less than 21 years with relapsed/refractory (R/R) FLT3 ITD and/or TKD Acute Myeloid Leukemia (AML) received 2 cycles (Cycle \[C\] 1 and C2) induction therapy with gilteritinib in combination with FLAG \[fludarabine, cytarabine and granulocyte colony-stimulating factor (G-CSF)\] chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 milligrams/kilogram/day (mg/kg/day) (maximum 120 mg/day) orally once daily (QD) from days 8 to 21. FLAG regimen consisted of fludarabine: 30 milligrams per square meter (mg/m\^2) per day intravenously (IV) from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 micrograms per kilogram (μg/kg) per day subcutaneously (SC) or IV from day -1 to day 5. Each cycle = 28 days.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Baseline characteristics by cohort

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Age, Continuous	11.9 Years STANDARD_DEVIATION 2.4 • n=9667 Participants
Sex: Female, Male Female	3 Participants n=9667 Participants
Sex: Female, Male Male	6 Participants n=9667 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=9667 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=9667 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=9667 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=9667 Participants
Race (NIH/OMB) Asian	0 Participants n=9667 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=9667 Participants
Race (NIH/OMB) Black or African American	0 Participants n=9667 Participants
Race (NIH/OMB) White	9 Participants n=9667 Participants
Race (NIH/OMB) More than one race	0 Participants n=9667 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=9667 Participants
Percent Inhibition of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3)	100.00 Percentage of pFLT3 STANDARD_DEVIATION 0.00 • n=9667 Participants

PRIMARY outcome

Timeframe: C1D1 up to day 28

Population: FAS

MTD reflects the highest dose that did not cause a Dose Limiting Toxicity (DLT).DLT:any Grade ≥3 non-hematologic/extramedullary toxicity with the following exceptions that occurred during the DLT observations like alopecia, anorexia, or fatigue, grade 3 vomiting or diarrhea that resolved(with or without supportive care) to ≤ grade 2 within 48 hours, grade 3 nausea that resolved(with or without supportive care) to ≤ grade 2 within 7 days, grade 3 elevation in total bilirubin (TBL) that is asymptomatic and that returned to ≤ grade 2 elevation within 7 days, grade 3 elevation in hepatic transaminases[alanine aminotransferase (ALT/SGPT) aspartate aminotransferase (AST/SGOT) and gammaglutamyl transferase (GGT)] or Alkaline Phosphatase (ALP) level that returns to ≤ grade 2 elevation within 14 days, grade 3 fever with neutropenia, with/without infection, grade 3 infection/grade 4 infections expected as direct complication of cytopenia due to active underlying leukemia, grade 3 mucositis.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Phase 1: Maximum Tolerated Dose (MTD) of Gilteritinib

2 mg/kg/day

PRIMARY outcome

Timeframe: C1D1 up to day 28

Population: FAS

The RP2D was a safe dose of gilteritinib that demonstrated sufficient activity.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Phase 1: Recommended Phase 2 Dose (RP2D) of Gilteritinib

2 mg/kg/day

PRIMARY outcome

Timeframe: From date of randomization to end of induction (induction= 1-2 cycles, each cycle = 28 days)

Population: FAS

CR rate was defined as the number of participants with best response of CR divided by the number of participants in the analysis population. CR was defined as a morphologically leukemia-free state post-baseline and had absolute neutrophil count (ANC) >= 1 x 10^9/L, platelet count >= 100 x 10^9/L and normal marrow differential with < 5% blasts. and were red blood cell (RBC) and platelet transfusion independent (defined as 1 week without RBC transfusion and 1 week without platelet transfusion). There should be no evidence of extramedullary leukemia and no evidence of Auer rods. The blast counts in peripheral blood must be <= 2%. Derived and investigator-assessed responses are reported.

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Phase 2: Complete Remission (CR) Rate Derived	33.3 Percentage of Participants Interval 7.5 to 70.1
Phase 2: Complete Remission (CR) Rate Investigator	33.3 Percentage of Participants Interval 7.5 to 70.1

PRIMARY outcome

Timeframe: From date of randomization to end of induction (induction= 1-2 cycles, each cycle = 28 days)

Population: FAS

CRc was defined as participants who achieved either CR, complete remission with incomplete platelet recovery (CRp) or complete remission with incomplete hematologic recovery (CRi) at the visit. CR was defined as a morphologically leukemia-free state post-baseline and had ANC >= 1 x 10^9/L, platelet count >= 100 x 10^9/L and normal marrow differential with < 5% blasts. and were RBC and platelet transfusion independent (defined as 1 week without RBC transfusion and 1 week without platelet transfusion). There should be no evidence of extramedullary leukemia and no evidence of Auer rods. The blast counts in peripheral blood must be <= 2%. CRp was defined as met all CR criteria except incomplete platelet recovery (< 100x10^9/L). CRi was defined as met all CR criteria, except for incomplete hematological recovery with residual neutropenia < 1x10^9/L with or without complete platelet recovery. Derived and investigator-assessed responses are reported.

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Phase 2: Composite CR (CRc) Rate Derived	66.7 Percentage of Participants Interval 29.9 to 92.5
Phase 2: Composite CR (CRc) Rate Investigator	55.6 Percentage of Participants Interval 21.2 to 86.3

PRIMARY outcome

Timeframe: From the date of first CR until the date of documented relapse for participants who achieved CR (Maximum duration: 28 months)

Population: FAS population included participants who achieved CR.

Duration of CR was defined as the time from the date of first CR until the date of documented relapse for participants who achieved CR. CR was defined as a morphologically leukemia-free state post-baseline and had ANC >= 1 x 10^9/L, platelet count >= 100 x 10^9/L and normal marrow differential with < 5% blasts, and were RBC and platelet transfusion independent (defined as 1 week without RBC transfusion and 1 week without platelet transfusion). There should be no evidence of extramedullary leukemia and no evidence of Auer rods. The blast counts in peripheral blood must be <= 2%. Relapse was defined as a reappearance of leukemic blasts in the peripheral blood or >= 5% blasts in the bone marrow aspirate (BMA) not attributable to any other cause or reappearance or new appearance of extramedullary leukemia. Duration of CR was estimated using the Kaplan-Meier method.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=3 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Duration of CR

NA days Interval 55.0 to Median and upper limit of the confidence interval was not estimable, due to insufficient number participants with events.

PRIMARY outcome

Timeframe: C1D1 up to day 28

Population: Safety Analysis Set (SAF) included all enrolled participants who took at least 1 dose of treatment regimen. Participants who were DLT evaluable were analyzed.

DLT: any Grade >=3 non-hematologic/extramedullary toxicity with the following exceptions that occurred during the DLT observation: Alopecia, anorexia, or fatigue. Grade 3 vomiting or diarrhea that resolved(with or without supportive care) to <= grade 2 within 48 hours. Grade 3 nausea that resolved(with or without supportive care) to <= grade 2 within 7 days. Grade 3 elevation in TBL that was asymptomatic and that returned to <= grade 2 elevation within 7 days. Grade 3 elevation in hepatic transaminases (ALT/SGPT, AST/SGOT) and GGT) or ALP level that returns to <= grade 2 elevation within 14 days. Grade 3 fever with neutropenia, with or without infection. Grade 3 infection or grade 4 infections expected as direct complication of cytopenia due to active underlying leukemia. Grade 3 mucositis.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=8 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Phase 1: Number of Participants With Dose Limiting Toxicity (DLT) of Gilteritinib

0 Participants

SECONDARY outcome

Timeframe: Baseline, predose on C1D15, C1D21, C2D8, C2D15, C2D21 and 4 to 6 hours post-dose on C1D21

Population: Pharmacodynamic Analysis Set (PDAS) included subset of the SAF for which sufficient pharmacodynamic measurements were collected. PDAS with available data was analyzed.

Plasma inhibitory assay (PIA) of phosphorylated FLT3 was conducted by comparing baseline and post-treatment samples. Inhibition was summarized at each time point. PIA assay assesses the target inhibition in plasma. Plasma was incubated with a cell line expressing the drug target, and inhibition was reported as percent change from baseline (normalized to 100%).

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 1 Day 15 Pre-Dose	15.00 Percent Change of pFLT3 Standard Deviation 14.41
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 1 Day 21 Pre-Dose	11.96 Percent Change of pFLT3 Standard Deviation 11.85
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 1 Day 21 4-6 Hours Post-Dose	7.32 Percent Change of pFLT3 Standard Deviation 9.14
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 2 Day 8 Pre-Dose	63.97 Percent Change of pFLT3 Standard Deviation 25.40
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 2 Day 15 Pre-Dose	9.90 Percent Change of pFLT3 Standard Deviation 2.95
Percent Change of Phosphorylated FMS-like Tyrosine Kinase 3 (FLT3) Cycle 2 Day 21 Pre-Dose	6.03 Percent Change of pFLT3 Standard Deviation 8.72

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 to 6 hours post-dose on C1D21

Population: The pharmacokinetic analysis set (PKAS) included the administered population for which sufficient plasma concentration data was available to facilitate derivation of at least 1 pharmacokinetic parameter and for whom the time of dosing on the day of sampling was known. PKAS with available data was analyzed.

Plasma samples were used for pharmacokinetic assessments.

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Gilteritinib Plasma Concentration Cycle 1 Day 8 Pre-Dose	0 nanogram (ng)/mL Standard Deviation 0
Gilteritinib Plasma Concentration Cycle 1 Day 15 Pre-Dose	251.93 nanogram (ng)/mL Standard Deviation 202.55
Gilteritinib Plasma Concentration Cycle 1 Day 21 Pre-Dose	225.25 nanogram (ng)/mL Standard Deviation 135.18
Gilteritinib Plasma Concentration Cycle 1 Day 21 4-6 Hours Post-Dose	439.00 nanogram (ng)/mL Standard Deviation 209.62
Gilteritinib Plasma Concentration Cycle 2 Day 15 Pre-Dose	145.07 nanogram (ng)/mL Standard Deviation 67.09

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 and 6 hours post-dose on C1D21

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 and 6 hours post-dose on C1D21

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 and 6 hours post-dose on C1D21

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 and 6 hours post-dose on C1D21

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Predose on C1D8, C1D15, C1D21, C2D15, and 4 and 6 hours post-dose on C1D21

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From first dose up to 28 days after last dose (maximum duration approximately 55 months)

Population: SAF

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 until 28 days from the last study treatment.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

9 Participants

SECONDARY outcome

Timeframe: From first dose to documented relapse or death, whichever occurred first (maximum duration: 55 month)

Population: FAS

EFS was defined as the time from first study drug dose to documented relapse or death, whichever occurred first. If none of these events occurred, EFS was censored at the last relapse-free assessment. Relapse was defined as a reappearance of leukemic blasts in the peripheral blood or >= 5% blasts in the BMA not attributable to any other cause or reappearance or new appearance of extramedullary leukemia. EFS was estimated using the Kaplan-Meier method.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Event Free Survival (EFS)

123.00 days Interval 1.0 to Upper limit of the confidence interval was not estimable, due to insufficient number participants with events.

SECONDARY outcome

Timeframe: From the date of enrollment until the date of death from any cause (maximum duration: 55 months)

Population: FAS

OS was defined as the time from the date of enrollment until the date of death from any cause (death date - enrollment date + 1). For a participant who was not known to have died by the end of study follow-up, OS was censored at the date of last contact (date of last contact - enrollment date + 1). OS was estimated using the Kaplan-Meier method.

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Overall Survival (OS)

NA months Interval 4.8 to Median and upper limit of the confidence interval was not estimable, due to insufficient number participants with events.

SECONDARY outcome

Timeframe: From baseline up to approximately 55 months

Population: The MRD Analysis Set (MAS) included a subset of the FAS for which participants enrolled, received at least 1 dose of the treatment regimen, and had at least 1 post-baseline sample with MRD data.

MRD negative was defined as summed FLT3-ITD signal ratio of any post-baseline sample ≤10^(-4).

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=5 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Number of Participants With Negative Minimal Residual Disease (MRD) Status

4 Participants

SECONDARY outcome

Timeframe: From baseline up to approximately 55 months

Population: MAS with available data was analyzed.

MRD negative was defined as summed FLT3-ITD signal ratio of any post-baseline sample <=10^(-4). CR rate was defined as the number of participants with best response of CR divided by the number of participants in the analysis population. CR was defined as a morphologically leukemia-free state post-baseline and had ANC >= 1 x 10^9/L, platelet count >= 100 x 10^9/L and normal marrow differential with < 5% blasts. and were RBC and platelet transfusion independent (defined as 1 week without RBC transfusion and 1 week without platelet transfusion). There should be no evidence of extramedullary leukemia and no evidence of Auer rods. The blast counts in peripheral blood must be <= 2%. Derived and investigator-assessed responses are reported.

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=4 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Percentage of Participants With MRD Negative Status in Relation to CR Rate Derived	75.0 percentage of participants Interval 19.4 to 99.4
Percentage of Participants With MRD Negative Status in Relation to CR Rate Investigator	75.0 percentage of participants Interval 19.4 to 99.4

SECONDARY outcome

Timeframe: From baseline up to approximately 55 months

Population: MAS with available data was analyzed.

MRD negative: summed FLT3-ITD signal ratio of any post-baseline sample <=10^(-4). CRc: participants who achieved either CR, complete remission with CRp or CRi at the visit. CR: morphologically leukemia-free state post-baseline and had ANC >= 1 x 10^9/L, platelet count >= 100 x 10^9/L and normal marrow differential with < 5% blasts. and were RBC and platelet transfusion independent. There should be no evidence of extramedullary leukemia and no evidence of Auer rods. The blast counts in peripheral blood must be <= 2%. CRp: 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. CRc rate: as number of participants with best response of CR divided by the number of participants in the analysis population. Derived and investigator-assessed responses are reported.

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=4 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Percentage of Participants With MRD Negative Status in Relation to CRc Rate Derived	100.0 percentage of participants Interval 39.8 to 100.0
Percentage of Participants With MRD Negative Status in Relation to CRc Rate Investigator	100.0 percentage of participants Interval 39.8 to 100.0

SECONDARY outcome

Timeframe: From baseline up to approximately 55 months

Population: MAS with available data was analyzed.

MRD negative: summed FLT3-ITD signal ratio of any post-baseline sample ≤10^(-4). OS was defined as the time from the date of enrollment until the date of death from any cause (death date - enrollment date + 1). For a participant who was not known to have died by the end of study follow-up, OS was censored at the date of last contact (date of last contact - enrollment date + 1).

Outcome measures

Measure

Gilteritinib 2 mg/kg/Day (Escalation Phase) n=4 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.

Number of Participants With MRD Negative Status in Relation to OS

1 participants

SECONDARY outcome

Timeframe: C1D1, C1D8

Population: FAS with available data was analyzed.

Participants evaluated the taste of the study drug/tablets and indicated whether they would be willing (feeling) to take the study drug/tablets again by selecting one of the following categories: 'Like a lot,' 'Like a little,' 'Neither like nor dislike,' 'Dislike a little,' or 'Dislike a lot.'

Outcome measures

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=7 Participants Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Tastes: Like a lot	1 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Tastes: Like a little	2 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Tastes: Neither like or dislike	3 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Tastes: Dislike a little	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Tastes: Dislike a lot	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Feeling: Like a lot	3 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Feeling: Like a little	1 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Feeling: Neither like or dislike	2 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Feeling: Dislike a little	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D8 Feeling: Dislike a lot	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Tastes: Like a lot	1 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Tastes: Like a little	1 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Tastes: Neither like or dislike	5 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Tastes: Dislike a little	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Tastes: Dislike a lot	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Feeling: Like a lot	3 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Feeling: Like a little	1 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Feeling: Neither like or dislike	3 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Feeling: Dislike a little	0 Participants
Number of Participants With Gilteritinib Acceptability and Palatability for Tablet C1D21 Feeling: Dislike a lot	0 Participants

Adverse Events

Gilteritinib 2 mg/kg/Day (Escalation Phase)

Serious events: 7 serious events

Other events: 9 other events

Deaths: 4 deaths

Serious adverse events

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 participants at risk Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
Blood and lymphatic system disorders Febrile neutropenia	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Pyrexia	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Immune system disorders Graft versus host disease in eye	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Immune system disorders Graft versus host disease in liver	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Immune system disorders Graft versus host disease oral	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Bacterial sepsis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Cellulitis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Device related bacteraemia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Device related infection	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Device related sepsis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Respiratory syncytial virus infection	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Sepsis	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Tooth abscess	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Injury, poisoning and procedural complications Allergic transfusion reaction	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Alanine aminotransferase increased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Aspartate aminotransferase increased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood bilirubin increased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations White blood cell count increased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hyperkalaemia	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypokalaemia	11.1% 1/9 • Number of events 4 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Hypoxia	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.

Other adverse events

Measure	Gilteritinib 2 mg/kg/Day (Escalation Phase) n=9 participants at risk Participants aged 2 years to less than 21 years with R/R FLT3 ITD and/or TKD AML received 2 cycles (C1 and C2) induction therapy with gilteritinib in combination with FLAG (fludarabine, cytarabine and G-CSF) chemotherapy. Gilteritinib tablet was administered at a starting dose of 2 mg/kg/day (maximum 120 mg/day) orally QD from days 8 to 21. FLAG regimen consisted of fludarabine: 30 mg/m\^2 per day IV from day 1 to day 5; cytarabine: 2000 mg/m\^2 per day IV from day 1 to day 5; G-CSF (filgrastim): 5 μg/kg per day SC or IV from day -1 to day 5. Each cycle = 28 days.
General disorders Chills	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Facial pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Non-cardiac chest pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Oedema peripheral	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Pyrexia	66.7% 6/9 • Number of events 12 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Swelling face	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Hepatobiliary disorders Hypertransaminasaemia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Immune system disorders Drug hypersensitivity	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Atypical pneumonia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations COVID-19	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Conjunctivitis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Device related infection	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Folliculitis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Fungal foot infection	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Herpes simplex	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Herpes zoster	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Nail infection	11.1% 1/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Nasal herpes	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Oral candidiasis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Eye disorders Eyelid oedema	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Abdominal pain	33.3% 3/9 • Number of events 5 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Abdominal pain upper	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Anal fissure	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Constipation	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Diarrhoea	44.4% 4/9 • Number of events 7 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Nausea	55.6% 5/9 • Number of events 8 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Odynophagia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Oral pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Gastrointestinal disorders Vomiting	44.4% 4/9 • Number of events 14 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
General disorders Catheter site pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Blood and lymphatic system disorders Anaemia	55.6% 5/9 • Number of events 41 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Blood and lymphatic system disorders Febrile neutropenia	33.3% 3/9 • Number of events 9 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Blood and lymphatic system disorders Leukocytosis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Blood and lymphatic system disorders Neutropenia	11.1% 1/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Blood and lymphatic system disorders Thrombocytopenia	22.2% 2/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Cardiac disorders Cardiac failure	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Eye disorders Eye pruritus	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Pneumonia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Rhinitis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Sinusitis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Infections and infestations Upper respiratory tract infection	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Injury, poisoning and procedural complications Allergic transfusion reaction	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Injury, poisoning and procedural complications Avulsion fracture	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Injury, poisoning and procedural complications Foot fracture	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Activated partial thromboplastin time prolonged	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Alanine aminotransferase increased	66.7% 6/9 • Number of events 39 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Aspartate aminotransferase increased	66.7% 6/9 • Number of events 20 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood bicarbonate decreased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood bilirubin increased	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood creatine phosphokinase increased	33.3% 3/9 • Number of events 6 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood creatinine increased	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood culture positive	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood lactate dehydrogenase increased	33.3% 3/9 • Number of events 7 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Blood potassium decreased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Gamma-glutamyltransferase increased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Lymphocyte count decreased	22.2% 2/9 • Number of events 36 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Mean arterial pressure decreased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Neutrophil count decreased	11.1% 1/9 • Number of events 4 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Oxygen saturation decreased	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations Platelet count decreased	33.3% 3/9 • Number of events 55 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Investigations White blood cell count decreased	33.3% 3/9 • Number of events 11 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Decreased appetite	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hyperglycaemia	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hyperkalaemia	22.2% 2/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypermagnesaemia	22.2% 2/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hyperphosphataemia	22.2% 2/9 • Number of events 7 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypertriglyceridaemia	11.1% 1/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypervolaemia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypoalbuminaemia	22.2% 2/9 • Number of events 29 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypocalcaemia	11.1% 1/9 • Number of events 15 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypokalaemia	22.2% 2/9 • Number of events 11 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypomagnesaemia	33.3% 3/9 • Number of events 15 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hyponatraemia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Hypophosphataemia	33.3% 3/9 • Number of events 6 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Metabolism and nutrition disorders Iron overload	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Musculoskeletal and connective tissue disorders Back pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Musculoskeletal and connective tissue disorders Pain in extremity	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Nervous system disorders Dizziness	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Nervous system disorders Headache	33.3% 3/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Nervous system disorders Hypotonia	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Nervous system disorders Neuralgia	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Nervous system disorders Presyncope	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Psychiatric disorders Depression	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Psychiatric disorders Irritability	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Cough	44.4% 4/9 • Number of events 10 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Dyspnoea	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Epistaxis	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Hypoxia	22.2% 2/9 • Number of events 4 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Respiratory, thoracic and mediastinal disorders Tachypnoea	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Drug eruption	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Erythema	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Pain of skin	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Pruritus	22.2% 2/9 • Number of events 3 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Rash	44.4% 4/9 • Number of events 7 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Rash maculo-papular	11.1% 1/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Skin erosion	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Skin and subcutaneous tissue disorders Urticaria	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Vascular disorders Axillary vein thrombosis	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Vascular disorders Hypertension	22.2% 2/9 • Number of events 2 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.
Vascular disorders Hypotension	11.1% 1/9 • Number of events 1 • All Cause: From randomization up to end of study (maximum duration approximately 55 months) AE: From first dose up to 28 days after last dose (maximum duration approximately 55 months) SAF included all participants who took at least 1 dose of the treatment regimen.

Additional Information

Clinical Transparency

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Results disclosure agreements

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