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CompletedNCT02927262Updated Nov 26, 2024Results posted

A Study of ASP2215 (Gilteritinib), Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FMS-like Tyrosine Kinase 3 (FLT3/ITD) Acute Myeloid Leukemia (AML) in First Complete Remission

A Phase 2 interventional study of Gilteritinib and Placebo in Acute Myeloid Leukemia (AML) and Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation, sponsored by Astellas Pharma Global Development, Inc.. Completed at 73 sites in 21 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-11-26.

Sponsored by Astellas Pharma Global Development, Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
98
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

The purpose of this study was to compare relapse-free survival (RFS) between participants with FMS-like tyrosine kinase 3 (FLT3) / internal tandem duplication (ITD) acute myeloid leukemia (AML) in first complete remission (CR1) and who were randomized to receive gilteritinib or placebo beginning after completion of induction/consolidation chemotherapy for a two-year period.

Read the detailed description

Participants in CR1 were approached for this study after induction/consolidation therapy was complete and a decision not to proceed with transplantation was made or a suitable donor could not be identified. Participants were randomized in a 2:1 ratio to receive gilteritinib or placebo. Participants entered the screening period up to 14 days prior to the start of treatment. Participants were administered treatment over continuous 28-day cycles. Gilteritinib or placebo was given daily for up to 2 years. After treatment discontinuation, participants had a 30-day follow-up visit for safety, after which the participants entered the long-term follow up period for collection of subsequent AML treatment, remission status, and survival (cause of death and date of death). Final database lock will occur when last subject last follow-up visit is reached, per protocol. Study drug was not provided during the follow-up period.

02

Conditions studied

  • Acute Myeloid Leukemia (AML)
  • Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation

Keywords

  • Acute myeloid leukemia
  • First Complete Remission
  • gilteritinib
  • FLT3/ITD
  • AML
  • ASP2215
03

In context

Leukemia

5,442 studies on the registry are indexed under Leukemia; 637 are open to participants now.

This study's enrollment of 98 is above the median of 38 across 4,248 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

Astellas Pharma Global Development, Inc. is the lead sponsor of 204 studies on the registry; 25 are open to participants now.

Of its 80 completed or terminated interventional studies of FDA-regulated products, 38 (48%) have results posted.

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

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Subject is considered an adult according to local regulation at the time of obtaining consent form (ICF).
  • Subject consents to allow access to subject's diagnostic bone marrow aspirate or peripheral blood sample and/or the DNA derived from that sample, if available, that may be used to validate a companion diagnostic test for gilteritinib.
  • Subject has confirmed morphologically documented AML, excluding acute promyelocytic leukemia (APL), in CR1 (including CRp and CRi). For the purposes of enrollment, CR will be defined as \< 5% blasts in the bone marrow with no morphologic characteristics of acute leukemia (e.g., Auer rods) in the bone marrow with no evidence of extramedullary disease such as central nervous system involvement or granulocytic sarcoma.
  • Subject will not proceed with transplantation as either a decision not to proceed with transplantation has been made either on the recommendation of the treating physician or by the patient or a suitable donor could not be identified.
  • Subject is \< 2 months from the start of the last cycle of consolidation and should have completed the recommended number of consolidations per local practice.
  • Subject has had no use of investigational agents, with the exception of FLT3 inhibiting agents during induction and/or consolidation therapy, within the prior 4 weeks.
  • Subject has had presence of the FLT3/ITD activating mutation in the bone marrow or peripheral blood as determined by the local institution at diagnosis.
  • Subject has an ECOG performance status 0 to 2.

  • Subject must meet the following criteria as indicated on the clinical laboratory tests:

    • Serum creatinine ≤ 1.5 x institutional upper limit of normal (ULN), or if serum creatinine outside normal range, then glomerular filtration rate (GFR) > 40 mL/min/1.73m\^2 as calculated with the 4-parameter Modification of Diet in Renal Disease (MDRD) equation.
    • Serum total bilirubin ≤ 2.5 mg/dL (43 μmol/L), except for subjects with Gilbert's syndrome.
    • Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x ULN.
    • Serum potassium and serum magnesium ≥ institutional lower limit of normal (LLN).
    • Absolute neutrophil count (ANC) ≥ 500/μl and platelets ≥ 20000/μl (unsupported by transfusions).
  • Subject is suitable for oral administration of study drug.

  • Female subject must either:

    • Be of nonchildbearing potential:
    • Postmenopausal (defined as at least 1 year without any menses) prior to screening, or
    • Documented surgically sterile or status posthysterectomy (at least 1 month prior to screening)
    • Or, if of childbearing potential,
    • Agree not to try to become pregnant during the study and for 6 months after the final study drug administration
    • And have a negative urine or serum pregnancy test at screening
    • And, if heterosexually active, agree to consistently use highly effective contraception per locally accepted standards (in addition to a barrier method) starting at screening and throughout the study period and for 6 months after the final study drug administration.
  • Female subject must agree not to breastfeed starting at screening and throughout the study period, and for 2 months and 1 week after the final study drug administration.
  • Female subject must not donate ova starting at screening and throughout the study period, and for 6 months after the final study drug administration.
  • Male subject and subject's female partners who are of childbearing potential must be using highly effective contraception per locally accepted standards (in addition to a barrier method) starting at screening and continue throughout the study period and for 4 months and 1 week after the final study drug administration.
  • Male subject must not donate sperm starting at screening and throughout the study period and for 4 months and 1 week after the final study drug administration.
  • Subject agrees not to participate in another interventional study while on treatment.

Exclusion criteria

Exclusion Criteria:

  • Subject has had prior allogeneic transplant.
  • Subject has QTcF interval > 450 msec (average of triplicate determinations based on central reading).
  • Subject with Long QT Syndrome.
  • Subject with hypokalemia and hypomagnesemia at screening (defined as values below LLN).
  • Subject has clinically active central nervous system leukemia.
  • Subject is known to have human immunodeficiency virus infection.
  • Subject has active hepatitis B or C.
  • Subject has an uncontrolled infection. If a bacterial or viral infection is present, the subject must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to randomization. If a fungal infection is present, the subject must be receiving definitive systemic anti-fungal therapy and have no signs of progressing infection for 1 week prior to randomization.
  • Subject has progressing infection defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
  • Subject has uncontrolled angina, severe uncontrolled ventricular arrhythmias, electrocardiographic evidence of acute ischemia, congestive heart failure New York Heart Association (NYHA) class 3 or 4 or subject has a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 1 month prior to study entry results in a left ventricular ejection fraction that is ≥ 45%.
  • Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP) 3A.
  • Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of P-glycoprotein (P-gp) with the exception of drugs that are considered absolutely essential for the care of the subject.
  • Subject requires treatment with concomitant drugs that target serotonin 5-hydroxytryptamine receptor 1 (5HT1R) or 5-hydroxytryptamine receptor 2B (5HT2BR) or sigma nonspecific receptor with the exception of drugs that are considered absolutely essential for the care of the subject.
  • Subject has a serious medical or psychiatric illness likely to interfere with participation in this clinical study.
  • Subject has prior malignancies, except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent ≥ 5 years previously will be allowed. Cancer treated with curative intent \< 5 years previously will not be allowed.
  • Subject has any condition which makes the subject unsuitable for study participation.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
98 participants (actual)

Study arms

  • Experimental
    Gilteritinib

    Participants received gilteritinib 120 mg (three tablets of 40 mg) orally, once daily (QD) for up to 2 years or until a protocol-specified discontinuation criterion was met.

    Drug: Gilteritinib

  • Placebo comparator
    Placebo

    Participants received gilteritinib matching placebo orally, QD for up to 2 years or until a protocol-specified discontinuation criterion was met.

    Drug: Placebo

Interventions

  • DrugGilteritinib

    Oral tablet

    Also known as: ASP2215

  • DrugPlacebo

    Oral tablet

06

What researchers measure

Primary outcomes

  1. Relapse-free Survival (RFS) Per Independent Review Committee (IRC) Adjudication

    RFS was defined as the time from the date of randomization until the date of documented relapse or death from any cause, whichever occurred first. Relapse after complete remission (CR) \[including complete remission with incomplete platelet recovery (CRp) and Complete remission with incomplete hematologic recovery (CRi)\], was defined as bone marrow blasts 5% or higher (not attributable to regenerating bone marrow), any circulating blasts, any extra-medullary blast foci as per Revised International Working Group (IWG) criteria. Participants were classified as: CRi, if they fulfilled all the criteria for CR except for incomplete hematological recovery with residual neutropenia \< 1 × 10\^9/L with or without complete platelet recovery. RBC and platelet transfusion independence was not required. CRp, if they achieved CR except for incomplete platelet recovery (\< 100 × 10\^9/L). RFS was estimated using Kaplan-Meier estimates. hazard ratio (HR), cox proportional hazards model (CHM)

    Time frame: From the date of randomization until the date of documented relapse, or death; (Median time on study drug was 427 days for gilteritinib group and 212 days for placebo group)

Secondary outcomes

  1. Overall Survival (OS)

    OS was defined as the time from the date of randomization until the date of death from any cause. OS was estimated using Kaplan-Meiers method.

    Time frame: From the date of randomization until the date of death from any cause; (Median time on study drug was 427 days for gilteritinib group and 212 days for placebo group)

  2. Event-Free Survival (EFS)

    EFS was defined as the time from the date of randomization until the date of documented relapse or discontinuation of the treatment, or initiation of other anti-leukemic treatment or death from any cause, whichever occurred first. Relapse after CR (including CRp and CRi), was defined as bone marrow blasts 5% or higher (not attributable to regenerating bone marrow), any circulating blasts, any extra-medullary blast foci as per Revised IWG criteria. Participants were classified as: CRi, if they fulfilled all the criteria for CR except for incomplete hematological recovery with residual neutropenia \< 1 × 10\^9/L with or without complete platelet recovery. RBC and platelet transfusion independence was not required. CRp, if they achieved CR except for incomplete platelet recovery (\< 100 × 10\^9/L). EFS was estimated using Kaplan-Meier's method.

    Time frame: From date of randomization until the date of documented relapse or discontinuation of the treatment, or initiation of other anti-leukemic treatment or death from any cause; (Median time on study drug was 427 days for gilteritinib and 212 days for Placebo)

  3. Change From Baseline in Quantitative Minimal Residual Disease Measured as Log10-transformed Overall FLT3/ITD Mutation Ratio at Months 3, 6, 12, 24/End of Treatment (EoT)

    MRD was measured from bone marrow samples. FLT3/ITD mutation ratio was measured in relation to total FLT3. For a participant with multiple ITD mutations, the overall FLT3/ITD mutation ratio was calculated from the sum of all ITD mutations. Absence of Minimal Residual Disease (MRD) is defined as log10-transformed overall FLT3/ITD mutation ratio ≤ -4.

    Time frame: Baseline and months 3, 6, 12, 24/EoT

  4. Number of Participants With Adverse Events (AE)

    An AE is any untoward medical occurrence in a participant administered a study drug, which does not necessarily have to have a causal relationship with treatment. It can, be any unfavorable and unintended sign, symptom or disease (new or exacerbated) temporally associated with the use of a medicinal product whether considered related to the medicinal product. An AE is considered "serious" if, it results in results in death, is life-threatening (an AE is considered "life-threatening" if, its occurrence places the participant at immediate risk of death, results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly or birth defect, Requires inpatient hospitalization (except for planned procedures as allowed per study) or leads to prolongation of hospitalization. TEAE was defined as an AE observed after starting administration of the study drug through 30 days after the last dose.

    Time frame: From first dose date up to 30 days after last dose or data cut-off date 25-May 2021 (Maximum treatment duration was 744 days)

  5. Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) Score

    ECOG performance status was measured on an 6 point scale. 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. Number of participants with ECOG PS was reported. ECOG PS grades with zero participants were not reported.

    Time frame: Months 1, 2, 3, 4, 5, 6, 8, 10. 12, 14, 16, 18, 20, 22 and 24/EoT

07

Results

Posted Jun 27, 2022

Participant flow

Adult participants diagnosed with FMS-like tyrosine kinase 3 (FLT3)/internal tandem duplication (ITD) acute myeloid leukemia (AML) in first complete remission (CR1), including complete remission with incomplete platelet recovery (CRp) and complete remission with incomplete hematologic recovery (CRi) for whom a decision not to proceed with transplantation was made, or a suitable donor could not be identified, were enrolled in this study.

Treatment Period (Up to 744 Days)
Participant flow — Treatment Period (Up to 744 Days)
MilestoneGilteritinibPlacebo
Started6335
Completed2412
Not completed3923
Withdrew: Withdrawal by subject30
Withdrew: Death10
Withdrew: Treatment ended due to transplant procedure10
Withdrew: Physician decision10
Withdrew: Becomes eligible for and proceeds to transplant01
Withdrew: Participant relapsed and became eligible for and proceeded to transplant01
Withdrew: Randomized in error10
Withdrew: Participant was not eligible for the study10
Withdrew: Mrd positive01
Withdrew: Disease relapse2318
Withdrew: Adverse event71
Withdrew: Investigators decision to take participant off trial due to molecular relapse10
Withdrew: Clinicians decision as suspected relapse01
Long Term Follow-up (Up to 1201 Days)
Participant flow — Long Term Follow-up (Up to 1201 Days)
MilestoneGilteritinibPlacebo
Started6035
Did not enter long term follow up period30
Completed3521
Not completed2514
Withdrew: Withdrawal by subject02
Withdrew: Lost to follow-up20
Withdrew: Death2312

Outcome measures

PrimaryRelapse-free Survival (RFS) Per Independent Review Committee (IRC) Adjudication

RFS was defined as the time from the date of randomization until the date of documented relapse or death from any cause, whichever occurred first. Relapse after complete remission (CR) \[including complete remission with incomplete platelet recovery (CRp) and Complete remission with incomplete hematologic recovery (CRi)\], was defined as bone marrow blasts 5% or higher (not attributable to regenerating bone marrow), any circulating blasts, any extra-medullary blast foci as per Revised International Working Group (IWG) criteria. Participants were classified as: CRi, if they fulfilled all the criteria for CR except for incomplete hematological recovery with residual neutropenia \< 1 × 10\^9/L with or without complete platelet recovery. RBC and platelet transfusion independence was not required. CRp, if they achieved CR except for incomplete platelet recovery (\< 100 × 10\^9/L). RFS was estimated using Kaplan-Meier estimates. hazard ratio (HR), cox proportional hazards model (CHM)

Time frame:
From the date of randomization until the date of documented relapse, or death; (Median time on study drug was 427 days for gilteritinib group and 212 days for placebo group)
Reported as:
Median · Months
Relapse-free Survival (RFS) Per Independent Review Committee (IRC) Adjudication
MonthsGilteritinibPlacebo
Relapse-free Survival (RFS) Per Independent Review Committee (IRC) Adjudication24.02 (14.06 to NA)15.84 (3.02 to NA)
Statistical analysis
  • Gilteritinib vs Placebo · Log Rank · p = 0.163 · Hazard ratio (hr): 0.738 · 95% CI 0.407 to 1.336HR \& 95% CI are based on CHM. Assuming proportional hazards, HR \< 1 indicates a reduction in hazard rate in favor of gilteritinib arm. Stratification factors:age, geographic region, presence of MRD at screening, use of FLT3 inhibiting agents per IRT.
SecondaryOverall Survival (OS)

OS was defined as the time from the date of randomization until the date of death from any cause. OS was estimated using Kaplan-Meiers method.

Time frame:
From the date of randomization until the date of death from any cause; (Median time on study drug was 427 days for gilteritinib group and 212 days for placebo group)
Reported as:
Median · Months
Overall Survival (OS)
MonthsGilteritinibPlacebo
Overall Survival (OS)NA (30.42 to NA)NA (43.56 to NA)
Statistical analysis
  • Gilteritinib vs Placebo · Log Rank · p = 0.627 · Hazard ratio (hr): 1.130 · 95% CI 0.540 to 2.364HR \& 95%CI are based on CHM. Assuming proportional hazards, HR \< 1 indicates a reduction in hazard rate in favor of gilteritinib arm. Stratification factors: age, geographic region, presence of MRD at screening, use of FLT3 inhibiting agents per IRT.
SecondaryEvent-Free Survival (EFS)

EFS was defined as the time from the date of randomization until the date of documented relapse or discontinuation of the treatment, or initiation of other anti-leukemic treatment or death from any cause, whichever occurred first. Relapse after CR (including CRp and CRi), was defined as bone marrow blasts 5% or higher (not attributable to regenerating bone marrow), any circulating blasts, any extra-medullary blast foci as per Revised IWG criteria. Participants were classified as: CRi, if they fulfilled all the criteria for CR except for incomplete hematological recovery with residual neutropenia \< 1 × 10\^9/L with or without complete platelet recovery. RBC and platelet transfusion independence was not required. CRp, if they achieved CR except for incomplete platelet recovery (\< 100 × 10\^9/L). EFS was estimated using Kaplan-Meier's method.

Time frame:
From date of randomization until the date of documented relapse or discontinuation of the treatment, or initiation of other anti-leukemic treatment or death from any cause; (Median time on study drug was 427 days for gilteritinib and 212 days for Placebo)
Reported as:
Median · Months
Event-Free Survival (EFS)
MonthsGilteritinibPlacebo
Event-Free Survival (EFS)14.06 (9.89 to 23.72)6.74 (2.86 to 21.95)
Statistical analysis
  • Gilteritinib vs Placebo · Log Rank · p = 0.296 · Hazard ratio (hr): 0.862 · 95% CI 0.510 to 1.455HR \& 95%CI are based on CHM. Assuming proportional hazards, HR \< 1 indicates a reduction in hazard rate in favor of gilteritinib arm. Stratification factors: age, geographic region, presence of MRD at screening, use of FLT3 inhibiting agents per IRT.
SecondaryChange From Baseline in Quantitative Minimal Residual Disease Measured as Log10-transformed Overall FLT3/ITD Mutation Ratio at Months 3, 6, 12, 24/End of Treatment (EoT)

MRD was measured from bone marrow samples. FLT3/ITD mutation ratio was measured in relation to total FLT3. For a participant with multiple ITD mutations, the overall FLT3/ITD mutation ratio was calculated from the sum of all ITD mutations. Absence of Minimal Residual Disease (MRD) is defined as log10-transformed overall FLT3/ITD mutation ratio ≤ -4.

Time frame:
Baseline and months 3, 6, 12, 24/EoT
Reported as:
Mean · Ratio
Change From Baseline in Quantitative Minimal Residual Disease Measured as Log10-transformed Overall FLT3/ITD Mutation Ratio at Months 3, 6, 12, 24/End of Treatment (EoT)
RatioGilteritinibPlacebo
Month 30.14 ± 0.960.14 ± 1.46
Month 60.07 ± 0.92-0.17 ± 0.79
Month 12-0.11 ± 0.60-0.34 ± 0.67
Month 24/EoT-0.12 ± 1.080.23 ± 1.63
Statistical analysis
  • Gilteritinib vs Placebo · ANCOVA · p = 0.970 (2-sided P-value from analysis of covariance (ANCOVA) including treatment, age group, geographic region and use of FLT3-inhibiting agents per IRT as fixed factors and baseline score as covariate.)
  • Gilteritinib vs Placebo · ANCOVA · p = 0.415 (2-sided P-value from ANCOVA including treatment, age group, geographic region and use of FLT3-inhibiting agents per IRT as fixed factors and baseline score as covariate.)
  • Gilteritinib vs Placebo · ANCOVA · p = 0.271 (2-sided P-value from ANCOVA including treatment, age group, geographic region and use of FLT3-inhibiting agents per IRT as fixed factors and baseline score as covariate.)
  • Gilteritinib vs Placebo · ANCOVA · p = 0.179 (2-sided P-value from ANCOVA including treatment, age group, geographic region and use of FLT3-inhibiting agents per IRT as fixed factors and baseline score as covariate.)
SecondaryNumber of Participants With Adverse Events (AE)

An AE is any untoward medical occurrence in a participant administered a study drug, which does not necessarily have to have a causal relationship with treatment. It can, be any unfavorable and unintended sign, symptom or disease (new or exacerbated) temporally associated with the use of a medicinal product whether considered related to the medicinal product. An AE is considered "serious" if, it results in results in death, is life-threatening (an AE is considered "life-threatening" if, its occurrence places the participant at immediate risk of death, results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly or birth defect, Requires inpatient hospitalization (except for planned procedures as allowed per study) or leads to prolongation of hospitalization. TEAE was defined as an AE observed after starting administration of the study drug through 30 days after the last dose.

Time frame:
From first dose date up to 30 days after last dose or data cut-off date 25-May 2021 (Maximum treatment duration was 744 days)
Reported as:
Number · Participants
Number of Participants With Adverse Events (AE)
ParticipantsGilteritinibPlacebo
TEAE5833
Drug-Related TEAE5120
TEAE before Relapse5728
Drug-Related TEAE before Relapse5115
Serious TEAE2414
Drug-Related Serious TEAE103
TEAE Leading to Death11
Drug-Related TEAE Leading to Death01
TEAE Leading to Withdrawal of Treatment156
Drug-Related TEAE Leading to Withdrawal of Treatment52
TEAE Leading to Dose Reduction151
Drug-Related TEAE Leading to Dose Reduction141
TEAE Leading to Dose Interruption354
Drug-Related TEAE Leading to Dose Interruption311
Grade 3 or Higher Treatment-Emergent Adverse Events4218
Grade 3 or Higher Drug-related TEAE334
Death2011
SecondaryNumber of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) Score

ECOG performance status was measured on an 6 point scale. 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. Number of participants with ECOG PS was reported. ECOG PS grades with zero participants were not reported.

Time frame:
Months 1, 2, 3, 4, 5, 6, 8, 10. 12, 14, 16, 18, 20, 22 and 24/EoT
Reported as:
Number · Participants
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) Score
ParticipantsGilteritinibPlacebo
Baseline: Grade 03922
Baseline: Grade 12313
Month 2: Grade 03818
Month 2: Grade 1169
Month 2: Grade 201
Month 3: Grade 03320
Month 3: Grade 1194
Month 3: Grade 201
Month 4: Grade 03716
Month 4: Grade 1126
Month 5: Grade 03517
Month 5: Grade 1114
Month 6: Grade 03217
Month 6: Grade 1112
Month 8: Grade 03212
Month 8: Grade 186
Month 8: Grade 210
Month 10: Grade 02914
Month 10: Grade 183
Month 12: Grade 02414
Month 12: Grade 1113
Month 14: Grade 02113
Month 14: Grade 193
Month 16: Grade 02211
Month 16: Grade 142
Month 16: Grade 220
Month 18: Grade 01913
Month 18: Grade 181
Month 20: Grade 01813
Month 20: Grade 171
Month 22: Grade 02011
Month 22: Grade 142
Month 24/EoT: Grade 03420
Month 24/EoT: Grade 1146
Month 24/EoT: Grade 210
Month 24/EoT: Grade 311
Month 24/EoT: Grade 411

Adverse events

Collected over From the date of randomization up to end of study (85 months and 9 days). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Gilteritinib24/63 (38.1%)24/62 (38.7%)53/62 (85.5%)
Placebo12/35 (34.3%)14/35 (40%)32/35 (91.4%)
Most frequent serious events
Showing 10 of 41
Most frequent serious events
EventGilteritinibPlacebo
Acute myeloid leukaemia recurrentNeoplasms benign, malignant and unspecified (incl cysts and polyps)7/625/35
Febrile neutropeniaBlood and lymphatic system disorders2/621/35
HyperleukocytosisBlood and lymphatic system disorders0/621/35
NeutropeniaBlood and lymphatic system disorders1/621/35
Anal haemorrhageGastrointestinal disorders0/621/35
NauseaGastrointestinal disorders0/621/35
VomitingGastrointestinal disorders0/621/35
PyrexiaGeneral disorders0/621/35
InfluenzaInfections and infestations0/621/35
Back painMusculoskeletal and connective tissue disorders0/621/35
Most frequent other events
Showing 10 of 57
Most frequent other events
EventGilteritinibPlacebo
Blood creatine phosphokinase increasedInvestigations18/621/35
NauseaGastrointestinal disorders8/627/35
Neutrophil count decreasedInvestigations12/623/35
Platelet count decreasedInvestigations12/624/35
ThrombocytopeniaBlood and lymphatic system disorders11/624/35
NasopharyngitisInfections and infestations7/626/35
NeutropeniaBlood and lymphatic system disorders10/621/35
FatigueGeneral disorders10/623/35
CoughRespiratory, thoracic and mediastinal disorders9/622/35
AnaemiaBlood and lymphatic system disorders5/625/35

Baseline characteristics

The full analysis set (FAS) consisted of all participants who were randomized.

Age, Continuous
Age, Continuous(Years)GilteritinibPlaceboTotal
Mean61.4 ± 11.059.9 ± 13.960.9 ± 12.1
Sex: Female, Male
Sex: Female, Male(Participants)GilteritinibPlaceboTotal
Female312051
Male321547
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)GilteritinibPlaceboTotal
Hispanic or Latino639
Not Hispanic or Latino502979
Unknown or Not Reported7310
Race (NIH/OMB)
Race (NIH/OMB)(Participants)GilteritinibPlaceboTotal
American Indian or Alaska Native000
Asian171027
Native Hawaiian or Other Pacific Islander000
Black or African American000
White382260
More than one race000
Unknown or Not Reported8311
Age (<60 or ≥60 years)
Age (<60 or ≥60 years)(Participants)GilteritinibPlaceboTotal
<60 years241337
≥60 years392261
Geographic Region
Geographic Region(Participants)GilteritinibPlaceboTotal
North America549
Europe402060
Rest of the world181129
Presence of MRD
Presence of MRD(Participants)GilteritinibPlaceboTotal
MRD = Yes8614
MRD = No552984
Use of FLT3-inhibitors
Use of FLT3-inhibitors(Participants)GilteritinibPlaceboTotal
Use of FLT3 Inhibitor = Yes121022
Use of FLT3 Inhibitor = No512576
08

Study locations

73 sites
  • Site US10017
    Gainesville, Florida 32610-0278, United States
  • Site US10030
    Jacksonville, Florida 32204, United States
  • Site US10012
    Chicago, Illinois 60612, United States
  • Site US10025
    Syracuse, New York 13210, United States
  • Site US10007
    Portland, Oregon 97239, United States
  • Site US10029
    Greenville, South Carolina 26615, United States
  • Site BR55002
    Goiania, Goias 74605-020, Brazil
  • Site CA15001
    Halifax, Nova Scotia B3H2Y9, Canada
  • Site CA15003
    Toronto, Ontario M5G 2M9, Canada
  • Site CZ42001
    Ostrava-Poruba, 70852, Czechia
  • Site DK45002
    Arhus, Region Midtjylland DK 8000, Denmark
  • Site FR33004
    Brest, Finistere 29609, France
  • Site FR33002
    Tours cedex 01, Indre-et-Loire 37044, France
  • Site FR33014
    Vandoeuvre les Nancy, Meurthe-et-Moselle 54511, France
  • Site FR33007
    Pierre-Benite, Rhone 69310, France
  • Site FR33001
    Bayonne, 64100, France
  • Site FR33009
    Mulhouse, 68070, France
  • Site FR33008
    Nice Cedex 2, 06189, France
  • Site DE49001
    Duisburg, Nordrhein-Westfalen 47166, Germany
  • Site DE49008
    Stuttgart, 70376, Germany
  • Site GR30010
    Athens, Attiki 10676, Greece
  • Site GR30004
    Thessaloniki, Kentriki Makedonia 57010, Greece
  • Site GR30009
    Athens, Greece
  • Site GR30007
    Larissa, Greece
  • Site HU36003
    Nyiregyhaza, Szabolcs-Szatmar-Bereg H-4400, Hungary
  • Site IL97205
    Jerusalem, Yerushalayim 91031, Israel
  • Site IT39011
    Milano, Lombardia 20141, Italy
  • Site IT39004
    Castelfranco Veneto (TV), Treviso 31033, Italy
  • Site IT39008
    Bergamo, 24127, Italy
  • Site IT39005
    Parma, Italy
  • Site IT39010
    Reggio Emilia, 42100, Italy
  • Site IT39002
    Roma, 161, Italy
  • Site JP81018
    Nagoya, Aichi, Japan
  • Site JP81025
    Matsuyama, Ehime, Japan
  • Site JP81002
    Yoshida-gun, Fukui, Japan
  • Site JP81024
    Sapporo, Hokkaido, Japan
  • Site JP81012
    Kobe, Hyogo, Japan
  • Site JP81004
    Kanazawa, Ishikawa, Japan
  • Site JP81009
    Yokohama, Kanagawa, Japan
  • Site JP81014
    Sendai, Miyagi, Japan
  • Site JP81023
    Shimotsuke, Tochigi, Japan
  • Site JP81011
    Tachikawa, Tokyo, Japan
  • Site JP81010
    Aomori, Japan
  • Site JP81017
    Okayama, Japan
  • Site KR82005
    Suwon-si, Gyeonggi-do 16499, Korea, Republic of
  • Site KR82014
    Bucheon-Si, Gyeonggido 14584, Korea, Republic of
  • Site KR82013
    Goyang, Gyeonggido 10408, Korea, Republic of
  • Site KR82008
    Namdong, Incheon Gwang'yeogsiv 405 760, Korea, Republic of
  • Site KR82003
    Hwasungun, Jeonranamdo 58128, Korea, Republic of
  • Site KR82012
    Seoul, Seoul Teugbyeolsi 06351, Korea, Republic of
  • Site KR82006
    Busan, 49241, Korea, Republic of
  • Site KR82009
    Seoul, 120-752, Korea, Republic of
  • Site PL48001
    Olsztyn, Warmińsko-mazurskie 10-228, Poland
  • Site PL48002
    Bydgoszcz, 85-168, Poland
  • Site PL48007
    Poznan, Poland
  • Site PT35106
    Coimbra, 3000, Portugal
  • Site PT35101
    Porto, 4200-072, Portugal
  • Site RO40005
    București, Romania
  • Site RS38102
    Belgrade, 11000, Serbia
  • Site ES34009
    Vitoria, Alava 01009, Spain
  • Site SE46003
    Stockholm, Stockholms Lan 171 76, Sweden
  • Site SE46002
    Lund, 221 85, Sweden
  • Site TW88605
    Kaohsiung, 112, Taiwan
  • Site TW88604
    Kaohsiung, 83301, Taiwan
  • Site TW88603
    Taipei, 114, Taiwan
  • Site GB44007
    Exeter, Devon EX2 5DW, United Kingdom
  • Site GB44019
    Plymouth, Devon PL6 8DH, United Kingdom
  • Site GB44006
    Cottingham, East Riding Of Yorkshire HU165JQ, United Kingdom
  • Site GB44018
    London, London, City Of WC1E 6BT, United Kingdom
  • Site GB44002
    Birmingham, B95SS, United Kingdom
  • Site GB44015
    Cardiff, CF4 4XN, United Kingdom
  • Site GB44020
    Leeds, LS9 7TF, United Kingdom
  • Site GB44004
    Nottingham, NG5 1PB, United Kingdom
09

References and documents

Study documents

  • Study protocol · Apr 25, 2019
  • Statistical analysis plan · Aug 12, 2021

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

Individual participant data

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

Supporting information: Study protocol, Sap, Csr

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 26, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02927262
Lead sponsor
Astellas Pharma Global Development, Inc.
Responsible party
Sponsor
First posted
Oct 7, 2016
Start date
Jan 10, 2017
Primary completion
May 25, 2021
Completion
Feb 19, 2024
Results posted
Jun 27, 2022
Last update
Nov 26, 2024

Study contacts

Executive Medical Director
study director · Astellas Pharma Global Development, Inc.

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.

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Discussion

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