A Phase 3 interventional study of Gilteritinib and Cytarabine in AML With FLT3 Mutation, sponsored by Astellas Pharma Inc. Completed at 49 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Astellas Pharma Inc · Phase 3, Interventional, and Treatment
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Among 31 studies testing gilteritinib in Leukemia, Myeloid, Acute — 13 open to participants now, 15 under way, 6 of them phase 3. gilteritinib · Leukemia, Myeloid, Acute.
Among the 31 studies testing gilteritinib in Leukemia, Myeloid, Acute, this one is the 10th largest by enrollment, is one of 3 that started in 2017, is one of 8 in phase 3, and was the 11th registered.
3,668 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
Browse Leukemia, Myeloid, Acute studies →13 of the 31 studies testing gilteritinib in Leukemia, Myeloid, Acute are open now, in 7 countries. Recruiting first, the newest first:
The First Affiliated Hospital of Soochow University · Sites in China
Ages 14–70
Technische Universität Dresden · Sites in Germany
Ages 18+
National Cancer Institute (NCI) · Sites in United States, Canada, Puerto Rico
Ages 18+
Institute of Hematology & Blood Diseases Hospital, China · Sites in China
Ages 14+
French Innovative Leukemia Organisation · Sites in France
Ages 18+
By country, each count listing its studies:
gilteritinib: first FDA approval 2018 (XOSPATA), first EMA approval 2019. What it is approved for, and how studies give it, is on its page →
Of the 625 Leukemia, Myeloid, Acute studies recruiting now, 363 (58%) set no upper age limit and 222 (36%) stop at 75 or younger; 145 take children under 18; 29 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
31 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 14 · Phase 2 16 · Phase 3 10
First in 2013; this study’s year is marked. 3 started in 2017.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 276 is above the median of 40 across 3,006 interventional studies indexed under Leukemia, Myeloid, Acute.
Median enrollment 97 across the 31 interventional studies. Of the 15 that completed or were terminated, 11 (73%) have results posted.
This tab as a report to print or save as PDF → With an account.
15 of 31 have an industry lead sponsor. The 10 most frequent:
Astellas Pharma Inc is the lead sponsor of 525 studies on the registry; 4 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 19 (68%) have results posted.
22 studies in Leukemia, Myeloid, Acute by Astellas Pharma Inc: none open now, 1 under way, none of them phase 3. Of 19 finished, 11 (58%) have results posted.
Among the 22 studies in Leukemia, Myeloid, Acute by Astellas Pharma Inc, this one is the 3rd largest by enrollment of the 17 interventional ones that give a count, is one of 3 that started in 2017, is one of 4 in phase 3, and was the 15th registered.
Astellas Pharma Inc’s Leukemia, Myeloid, Acute studies by the year they started, since 2006.
1 of the studies testing gilteritinib in Leukemia, Myeloid, Acute that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05024552H. Lee Moffitt Cancer Center and Research InstituteVyxeos Plus Gilteritinib in Relapsed or Refractory, FLT3-Mutated AML | 2027-02-15 |
Add it to your calendar (.ics) → With an account.
Among the 31 studies testing gilteritinib in Leukemia, Myeloid, Acute, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among Astellas Pharma Inc’s 22 in Leukemia, Myeloid, Acute:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The purpose of this study was to determine the clinical benefit of ASP2215 therapy in participants with FMS-like tyrosine kinase (FLT3) mutated AML who were refractory to or had relapse after first-line AML therapy as shown with overall survival (OS) compared to salvage chemotherapy. In addition, this study evaluated safety as well as determined the overall efficacy in event-free survival (EFS) and complete remission (CR) rate of ASP2215 compared to salvage chemotherapy.
Participants considered an adult according to local regulations at the time of signing informed consent were randomized in a 1:1 ratio and received ASP2215 or salvage chemotherapy. Participants entered the screening period up to 14 days prior to the start of treatment. Prior to randomization, the investigator preselected a salvage chemotherapy regimen for each participant; options included low-dose cytarabine (LoDAC), mitoxantrone, etoposide and intermediate-dose cytarabine (MEC) or fludarabine, high-dose cytarabine and granulocyte colony-stimulating factor (FLAG). The randomization was stratified by response to first-line therapy and preselected salvage chemotherapy. Participants were administered treatment over continuous 28-day cycles.
Among the participants, approximately 20 Chinese participants who were randomized into the ASP2215 arm were allocated to the pharmacokinetic (PK) cohort. Participants in the PK cohort were requested to be hospitalized from the date of randomization (Day 1) to at least the completion of all the assessments planned on Day 2. All participants in the PK cohort underwent blood sampling for PK measurement of ASP2215. Participants in PK cohort were administered the study drug in the same manner and underwent the same efficacy and safety assessments as other participants except for blood sampling for additional PK measurements.
Inclusion Criteria:
Participant is to or relapsed after AML therapy (with or without )
Refractory to first-line AML therapy is defined as:
a. Participant did not achieve CR/CRi/CRp under initial therapy. A participant eligible for standard therapy must receive at least 1 cycle of an anthracycline containing induction block in standard dose for the selected induction regimen. A participant not eligible for standard therapy must have received at least 1 complete block of induction therapy seen as the optimum choice of therapy to induce remission for this participant.
Untreated first hematologic relapse is defined as:
Participant must meet the following criteria as indicated on the clinical laboratory tests:
Inclusion Criteria for COE:
Participant is eligible for the COE if they continue to meet all inclusion criteria from the main protocol in addition to the following when the participant is evaluated for eligibility to participate in the COE portion of the study:
Exclusion Criteria:
Exclusion Criteria for COE:
Participant will be excluded from participation in the COE if they meet any of the exclusion criteria listed in the main protocol or when the participant is evaluated for eligibility to participate in the COE portion of the study.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received 120 milligrams (mg) gilteritinib (3 tablets of 40 mg) orally, once a day in continuous 28-day cycles until treatment discontinuation criteria were met. Participants who met the treatment discontinuation criteria, entered the long-term follow-up period. Participants remained in the long-term follow-up period for up to 3 years from the participant's end of treatment visit or until discontinuation from the study.
Drug: Gilteritinib
Participants received salvage chemotherapy in continuous 28-day cycles per institutional guidelines:LoDAC:20mg cytarabine twice daily SC/IV for 10 days. MEC:mitoxantrone 6 milligrams per square meter(mg/m\^2)/day IV for 5 days (days 1 to 5), etoposide 100mg/m\^2/day IV for 5 days (days 1 to 5), cytarabine 1000mg/m\^2/day IV for 5 days (days 1 to 5). FLAG: granulocyte colony-stimulating factor (G-CSF) 300 micrograms per square meter (μg/m\^2) per day SC/IV for 5 days (days 1 to 5), fludarabine 30mg/m\^2/day IV for 5 days (days 2 to 6), cytarabine 2000mg/m\^2/day IV for 5 days (days 2 to 6). Participants meeting treatment discontinuation criteria, entered long-term follow-up, for up to 3 years from end of treatment visit/until study discontinuation. Based on interim analysis outcome, at investigators discretion, treatment period participants had option to enter crossover extension to receive 120mg gilteritinib, orally, once daily in continuous 28-day cycles until treatment discontinuation.
Drug: Cytarabine · Drug: Mitoxantrone · Drug: Etoposide · Drug: G-CSF · Drug: Fludarabine
Tablet administered orally once daily.
Also known as: ASP2215
Once/twice daily Intravenously (IV)/subcutaneously (SC).
Once daily IV injection.
Once daily IV injection.
Once daily IV/SC injection.
Once daily IV injection.
Overall Survival (OS)
OS was defined as the time from the date of randomization to the date of death due to any cause. Participants who were still alive or lost to follow up were censored at the time they were last known to be alive. Kaplan-Meier (KM) estimates was used for analysis.
Time frame: From the date of randomization up to the date of death (up to approximatley 74 months)
Event-Free Survival (EFS)
EFS: time from the date of randomization until the date of documented relapse, treatment failure, death, reported off treatment relapse or new AML therapy start whichever occued first, including the long-term follow-up data. KM estimate was used for analysis. Relapse was defined as documentation of any of following events: * Bone marrow (BM) blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts Treatment failure: Treatment failure was defined as participant who ends treatment without having a previous response of CR, CR with incomplete platelet recovery (CRp) and CR with incomplete hematological recover (CRi).
Time frame: From the date of randomization up to the date of documented relapse, treatment failure or death from any cause, off-treatment relapse and start of new AML therapy (up to approximately 74 months)
Complete Remission (CR) Rate
Percentage of participants with CR were reported. CR: morphologically leukemia-free state, with absolute neutrophil count (ANC) \> 1x10\^9 per liter (1x10\^9/L), platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were red blood cell (RBC) and platelet transfusion independent and no evidence of extramedullary leukemia or Auer rods was necessary.
Time frame: From the date of randomization up to approximately 74 months
Duration of CR
Duration of CR: time from the date of achieving first CR until date of first documented relapse for participants who achieved CR. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. Relapse was defined as documentation of any of following events: * BM blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts
Time frame: From date of achieving CR until date of confirmed relapse (maximum duration was 53.4 months )
Duration of Composite Complete Remission (CRc)
Duration of CRc: time from date of achieving first CRc until date of first documented relapse for participants who achieved CRc. KM estimate was used for analysis. CRc: rate of all complete \& incomplete remissions \[CR + CRp + CRi\]. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
Time frame: From date of achieving CRc until date of confirmed relapse (maximum duration was 60 months)
Duration of CR/Complete Remission With Partial Hematologic Recovery (CRh)
Duration of CR/CRh: time from date of achieving first CR/CRh until date of first documented relapse for participants who achieved CR/CRh. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
Time frame: From date of achieving CR/CRh until date of confirmed relapse (maximum duration was 58.1 months)
Duration Of Response (DOR)
DOR: time from date of first CRc (CR+CRp+CRi)/PR until date of first documented relapse for participants who achieved CRc or PR. KM estimate used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts and increase in the percentage of blasts in the BM aspirate to \> 25%.
Time frame: From date of achieving CRc/PR until date of confirmed relapse (maximum duration was 52.1 months)
CR/CRh Rate
Percentage of participants with CR/CRh was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%.
Time frame: From the date of randomization up to approximately 74 months
Best Response Rate
Defined as percentage of participants with CR, CRp, CRi, PR, no response (NR) \& not estimable (NE). CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L \& normal marrow differential with \< 5% blasts. They were RBC \& platelet transfusion independent \& no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\<100x10\^9/L). CRi: met all CR criteria, except incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with/without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. \<=5% BM blasts if Auer rods are present, no evidence of extramedullary leukemia. Not Estimable (NE): No BM assessed/no myeloblast value, no blast value from peripheral blood or ≤2%, \& no extramedullary leukemia. No Response (NR): Response not categorized as CR, CRp, CRi, PR or NE.
Time frame: From the date of randomization up to approximately 74 months
Leukemia-Fee Survival (LFS)
LFS: time from the date of first CRc (CR+CRp+CRi) until the date of documented relapse or death for participants who achieved CRc. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
Time frame: From first day of achieving first CRc to the first day of confirmed relapse/death (maximum duration was 60.0 months)
Composite Complete Remission (CRc)
Percentage of participants with CRc (CR+CRp+CRi) was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.
Time frame: From the date of randomization up to approximately 74 months
Time to CRc
Time to CRc (TTCRc) was defined as the time from the date of randomization until the date of first CRc. CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.
Time frame: From randomization until date of first CRc (up to approximately 74 months)
Time to CR
Time to CR (TTCR) was defined as the time from the date of randomization until the date of first CR. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia.
Time frame: From randomization until date of first CR (up to approximately 74 months)
Time to Response
Time to Response (TTR) was defined as the time from the date of randomization until the date of either first response (CRc or PR). CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%.
Time frame: From randomization until date of first CRc or PR (up to approximately 74 months)
Time to CR/CRh
Time to CR/CRh (TTCRCRh) was defined as the time from the date of randomization until the date of first CR/CRh. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%.
Time frame: From randomization until date of first CR/CRh (up to approximately 74 months)
Percentage of Participants With Transfusion Conversion and Transfusion Maintenance
Transfusion conversion rate: Percentage of transfusion dependent participants at baseline period but became transfusion independent at post-baseline period divided by total participants who were transfusion dependent at baseline period. Transfusion maintenance rate: Percentage of transfusion independent participants at baseline period and still maintained transfusion independent at post-baseline period divided by total participants who were transfusion independent at baseline period. Baseline transfusion status: Participants were classified as transfusion independent if there were no RBC or platelet transfusions within 28 days prior to first dose to 28 days after first dose; otherwise classified as transfusion dependent. Post baseline transfusion status: Participants on treatment ≥ 84 days were classified as transfusion independent, if consecutive 56 days without any RBC or platelet transfusion; otherwise classified as transfusion dependent
Time frame: Baseline up to approximately 74 months
Percentage of Participants With Transplantation Rate
Transplantation rate is defined as the percentage of participants undergoing Hematopoietic stem cell transplant (HSCT) during the study period.
Time frame: Baseline up to approximately 74 months
Change From Baseline in Brief Fatigue Inventory (BFI)
The BFI is a screening tool designed to assess the severity and impact of fatigue on daily functioning of participants with cancer during the 24 hours. There are 9 items on the scale. The first three questions ask participants to rate their fatigues on a scale from 0 (no fatigue) - 10 (as bad as you can imagine), with higher scores indicating worse outcome. The remaining six questions ask participants to rate how much fatigue has interfered with their daily activities on a scale from 0 (Does not interfere) to 10 (Completely interferes). A global fatigue score can be obtained by averaging all the items on the BFI, ranging between 0 to 10; a higher BFI fatigue score indicates worse outcome. The global BFI scores were calculated only if at least 5 of the 9 items are answered.
Time frame: Baseline, End of treatment (63 months)
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE could therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. TEAE was defined as an adverse event observed after starting administration of the study drug.
Time frame: From the date of first dose up to approximately 74 months
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Scores
The ECOG Scale was used to assess performance status. Number of participants with each grade was reported. Grade Description: 0: Fully active, able to carry on all pre-disease performance without restriction. 1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work. 2. Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours. 3. Capable of only limited self-care, confined to bed or chair more than 50% of waking hours. 4. Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair. 5. Dead.
Time frame: Baseline, end of treatment visit (63 months)
Pharmacokinetics (PK) of Gilteritinib in Chinese PK Cohort: Area Under the Concentration Curve at 24 Hours (AUC24)
AUC24 was derived from the PK samples collected.
Time frame: Cycle 1 Day 1(C1D1): predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose, Cycle 1 Day 15(C1D15): predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose
PK of Gilteritinib in Chinese PK Cohort: Maximum Concentration (Cmax)
Cmax was derived from the PK samples collected.
Time frame: C1D1: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose, C1D15: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hour post dose
PK of Gilteritinib: Observed Trough Concentration (Ctrough)
Ctrough was derived from the PK samples collected.
Time frame: Predose on C1D15
PK of Gilteritinib in Chinese PK Cohort: Time to Maximum Concentration (Tmax)
tmax was derived from the PK samples collected.
Time frame: C1D1: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hours (+/- 20 minutes) post dose, C1D15: predose, 0.5, 1, 2, 3, 4, 6, 10, 24 hours (+/- 20 minutes) post dose
Ctrough Concentration of Gilteritinib
Concentrations below the lower limit of quantification (0.5 ng/mL) were set to zero.
Time frame: Predose C1D8, C1D15, Day 1 of each cycle from C2 to C65, C67D1,C68D1,C69D1,C70D1
Participants with FMS-like tyrosine kinase 3 (FLT3) mutations with relapsed or refractory Acute Myeloid Leukemia (AML) after first-line therapy were enrolled for this study. 21 participants from six china sites were included in Giltertinib PK cohort.
| Milestone | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Started | 137 | 139 |
| Lodac | 0 | 27 |
| Mec | 0 | 42 |
| Flag | 0 | 70 |
| Chinese pk cohort | 21 | 0 |
| Completed | 0 | 26 |
| Not completed | 137 | 113 |
| Withdrew: Adverse event | 8 | 9 |
| Withdrew: Death | 19 | 8 |
| Withdrew: Miscellaneous | 12 | 0 |
| Withdrew: Disease relapse | 41 | 5 |
| Withdrew: Physician decision | 5 | 10 |
| Withdrew: Withdrawal by subject | 10 | 28 |
| Withdrew: Protocol violation | 4 | 1 |
| Withdrew: Progressive disease | 18 | 9 |
| Withdrew: Lack of efficacy | 20 | 43 |
| Milestone | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Started | 100 | 105 |
| Completed | 9 | 12 |
| Not completed | 91 | 93 |
| Withdrew: Miscellaneous | 4 | 3 |
| Withdrew: Withdrawal by subject | 5 | 8 |
| Withdrew: Lost to follow-up | 6 | 7 |
| Withdrew: Death | 76 | 75 |
| Milestone | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Started | 0 | 0 |
| Completed | 0 | 0 |
| Not completed | 0 | 0 |
OS was defined as the time from the date of randomization to the date of death due to any cause. Participants who were still alive or lost to follow up were censored at the time they were last known to be alive. Kaplan-Meier (KM) estimates was used for analysis.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Overall Survival (OS) | 10.3 (8.8 to 12.7) | 5.4 (4.1 to 8.1) |
EFS: time from the date of randomization until the date of documented relapse, treatment failure, death, reported off treatment relapse or new AML therapy start whichever occued first, including the long-term follow-up data. KM estimate was used for analysis. Relapse was defined as documentation of any of following events: * Bone marrow (BM) blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts Treatment failure: Treatment failure was defined as participant who ends treatment without having a previous response of CR, CR with incomplete platelet recovery (CRp) and CR with incomplete hematological recover (CRi).
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Event-Free Survival (EFS) | 2.1 (0.1 to 3.2) | 0.6 (0.2 to 1.2) |
Percentage of participants with CR were reported. CR: morphologically leukemia-free state, with absolute neutrophil count (ANC) \> 1x10\^9 per liter (1x10\^9/L), platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were red blood cell (RBC) and platelet transfusion independent and no evidence of extramedullary leukemia or Auer rods was necessary.
| Percentage of participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Complete Remission (CR) Rate | 20.4 (14.0 to 28.2) | 11.5 (6.7 to 18.0) |
Duration of CR: time from the date of achieving first CR until date of first documented relapse for participants who achieved CR. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. Relapse was defined as documentation of any of following events: * BM blasts ≥ 5% (not attributable to regenerating BM) * Reappearance or new appearance of extramedullary leukemia * Reappearance of significant numbers of peripheral blasts
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Duration of CR | 24.0 (4.6 to NA) | NA (1.0 to NA) |
Duration of CRc: time from date of achieving first CRc until date of first documented relapse for participants who achieved CRc. KM estimate was used for analysis. CRc: rate of all complete \& incomplete remissions \[CR + CRp + CRi\]. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Duration of Composite Complete Remission (CRc) | 4.1 (2.8 to 5.6) | NA (NA to NA) |
Duration of CR/CRh: time from date of achieving first CR/CRh until date of first documented relapse for participants who achieved CR/CRh. KM estimate was used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Duration of CR/Complete Remission With Partial Hematologic Recovery (CRh) | 5.6 (2.8 to 24.0) | NA (NA to NA) |
DOR: time from date of first CRc (CR+CRp+CRi)/PR until date of first documented relapse for participants who achieved CRc or PR. KM estimate used for analysis. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts and increase in the percentage of blasts in the BM aspirate to \> 25%.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Duration Of Response (DOR) | 3.7 (2.5 to 4.7) | NA (1.2 to NA) |
Percentage of participants with CR/CRh was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and could be classified as CR. The blast counts in peripheral blood was ≤ 2%.
| Percentage of participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| CR/CRh Rate | 32.1 (24.4 to 40.6) | 14.4 (9.0 to 21.3) |
Defined as percentage of participants with CR, CRp, CRi, PR, no response (NR) \& not estimable (NE). CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L \& normal marrow differential with \< 5% blasts. They were RBC \& platelet transfusion independent \& no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\<100x10\^9/L). CRi: met all CR criteria, except incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with/without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%. \<=5% BM blasts if Auer rods are present, no evidence of extramedullary leukemia. Not Estimable (NE): No BM assessed/no myeloblast value, no blast value from peripheral blood or ≤2%, \& no extramedullary leukemia. No Response (NR): Response not categorized as CR, CRp, CRi, PR or NE.
| Percentage of participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| CR | 20.4 | 11.5 |
| CRp | 13.1 | 0.7 |
| CRi | 19.7 | 10.1 |
| PR | 14.6 | 5.0 |
| NR | 27.7 | 34.5 |
| NE | 4.4 | 38.1 |
LFS: time from the date of first CRc (CR+CRp+CRi) until the date of documented relapse or death for participants who achieved CRc. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. Relapse: BM blasts ≥ 5%, reappearance or new appearance of extramedullary leukemia, reappearance of significant numbers of peripheral blasts.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Leukemia-Fee Survival (LFS) | 3.9 (2.8 to 5.4) | 6.9 (2.9 to 10.4) |
Percentage of participants with CRc (CR+CRp+CRi) was reported. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.
| Percentage of participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Composite Complete Remission (CRc) | 53.3 (44.6 to 61.9) | 22.3 (15.7 to 30.1) |
Time to CRc (TTCRc) was defined as the time from the date of randomization until the date of first CRc. CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Time to CRc | 1.8 (1 to 8) | 1.0 (1 to 2) |
Time to CR (TTCR) was defined as the time from the date of randomization until the date of first CR. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \< 5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Time to CR | 3.7 (1 to 19) | 1.1 (1 to 2) |
Time to Response (TTR) was defined as the time from the date of randomization until the date of either first response (CRc or PR). CRc: Rate of all complete and incomplete remissions (CR + CRp +Cri) CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRp: met all CR criteria except incomplete platelet recovery (\< 100x10\^9/L). CRi: met all CR criteria, except for incomplete hematological recovery with residual neutropenia \< 1x10\^9/L with or without complete platelet recovery. PR: condition with regeneration of normal hematopoietic cells in BM, no detectable blasts, ≥ 50% decrease of blasts in BM aspirate \& total BM blasts of 5-25%.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Time to Response | 1.0 (1 to 7) | 1.0 (1 to 2) |
Time to CR/CRh (TTCRCRh) was defined as the time from the date of randomization until the date of first CR/CRh. CR: morphologically leukemia-free state, with ANC\> 1x10\^9/L, platelet count ≥ 100x10\^9/L and normal marrow differential with \<5% blasts. They were RBC and platelet transfusion independent and no evidence of extramedullary leukemia. CRh was defined as a condition at the post baseline visit, having bone marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5x10\^9/L and platelets ≥ 50x10\^9/L, no evidence of extramedullary leukemia and cannot be classified as CR. The blast counts in peripheral blood was ≤ 2%.
| months | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Time to CR/CRh | 2.8 (1 to 11) | 1.1 (1 to 2) |
Transfusion conversion rate: Percentage of transfusion dependent participants at baseline period but became transfusion independent at post-baseline period divided by total participants who were transfusion dependent at baseline period. Transfusion maintenance rate: Percentage of transfusion independent participants at baseline period and still maintained transfusion independent at post-baseline period divided by total participants who were transfusion independent at baseline period. Baseline transfusion status: Participants were classified as transfusion independent if there were no RBC or platelet transfusions within 28 days prior to first dose to 28 days after first dose; otherwise classified as transfusion dependent. Post baseline transfusion status: Participants on treatment ≥ 84 days were classified as transfusion independent, if consecutive 56 days without any RBC or platelet transfusion; otherwise classified as transfusion dependent
| Percentage of participants | Gilteritinib |
|---|---|
| Transfusion Conversion Rate | 42.7 |
| Transfusion Maintenance Rate | 70.8 |
Transplantation rate is defined as the percentage of participants undergoing Hematopoietic stem cell transplant (HSCT) during the study period.
| Percentage of participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Percentage of Participants With Transplantation Rate | 22.6 (15.9 to 30.6) | 7.9 (4.0 to 13.7) |
The BFI is a screening tool designed to assess the severity and impact of fatigue on daily functioning of participants with cancer during the 24 hours. There are 9 items on the scale. The first three questions ask participants to rate their fatigues on a scale from 0 (no fatigue) - 10 (as bad as you can imagine), with higher scores indicating worse outcome. The remaining six questions ask participants to rate how much fatigue has interfered with their daily activities on a scale from 0 (Does not interfere) to 10 (Completely interferes). A global fatigue score can be obtained by averaging all the items on the BFI, ranging between 0 to 10; a higher BFI fatigue score indicates worse outcome. The global BFI scores were calculated only if at least 5 of the 9 items are answered.
| Scores on scale | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Change From Baseline in Brief Fatigue Inventory (BFI) | 1.42 ± 2.99 | 0.75 ± 2.92 |
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.
| Participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 134 | 119 |
The ECOG Scale was used to assess performance status. Number of participants with each grade was reported. Grade Description: 0: Fully active, able to carry on all pre-disease performance without restriction. 1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work. 2. Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours. 3. Capable of only limited self-care, confined to bed or chair more than 50% of waking hours. 4. Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair. 5. Dead.
| Participants | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Grade 0 at baseline | 35 | 37 |
| Grade 1 at baseline | 65 | 64 |
| Grade 2 at baseline | 34 | 18 |
| Grade 3 at baseline | 0 | 0 |
| Grade 4 at baseline | 0 | 0 |
| Grade 5 at baseline | 0 | 0 |
| Grade 0 at End of Treatment | 17 | 26 |
| Grade 1 at End of Treatment | 36 | 39 |
| Grade 2 at End of Treatment | 19 | 21 |
| Grade 3 at End of Treatment | 10 | 6 |
| Grade 4 at End of Treatment | 1 | 2 |
| Grade 5 at End of Treatment | 0 | 0 |
AUC24 was derived from the PK samples collected.
| nanogram*hour per milliliters(ng*h/mL) | Gilteritinib |
|---|---|
| C1D1 | 3230 ± 1970 |
| C1D15 | 10000 ± 6670 |
Cmax was derived from the PK samples collected.
| ng/mL | Gilteritinib |
|---|---|
| C1D1 | 206 ± 112 |
| C1D15 | 536 ± 323 |
Ctrough was derived from the PK samples collected.
| ng/mL | Gilteritinib |
|---|---|
| PK of Gilteritinib: Observed Trough Concentration (Ctrough) | 323 ± 222 |
tmax was derived from the PK samples collected.
| hours | Gilteritinib |
|---|---|
| Cycle 1 Day 1 | 4.00 (2.00 to 24.1) |
| Cycle 1 Day 15 | 3.85 (1.00 to 24.3) |
Concentrations below the lower limit of quantification (0.5 ng/mL) were set to zero.
| ng/mL | Gilteritinib |
|---|---|
| C1D8 | 310 ± 177 |
| CID15 | 367 ± 243 |
| C2D1 | 425 ± 305 |
| C3D1 | 457 ± 358 |
| C4D1 | 420 ± 428 |
| C5D1 | 407 ± 487 |
| C6D1 | 284 ± 275 |
| C7D1 | 370 ± 477 |
| C8D1 | 328 ± 323 |
| C9D1 | 351 ± 381 |
| C10D1 | 322 ± 383 |
| C11D1 | 336 ± 426 |
| C12D1 | 460 ± 703 |
| C13D1 | 368 ± 409 |
| C14D1 | 386 ± 449 |
| C15D1 | 396 ± 541 |
| C16D1 | 200 ± 116 |
| C17D1 | 228 ± 138 |
| C18D1 | 201 ± 143 |
| C19D1 | 231 ± 270 |
| C20D1 | 241 ± 190 |
| C21D1 | 221 ± 112 |
| C22D1 | 238 ± 149 |
| C23D1 | 152 ± 137 |
| C24D1 | 188 ± 141 |
| C25D1 | 192 ± 137 |
| C26D1 | 264 ± 138 |
| C27D1 | 199 ± 145 |
| C28D1 | 243 ± 141 |
| C29D1 | 184 ± 138 |
| C30D1 | 340 ± 472 |
| C31D1 | 426 ± 606 |
| C32D1 | 301 ± 395 |
| C33D1 | 431 ± 646 |
| C34D1 | 405 ± 590 |
| C35D1 | 298 ± 420 |
| C36D1 | 264 ± 343 |
| C37D1 | 437 ± 823 |
| C38D1 | 78.0 ± 76.3 |
| C39D1 | 107 ± 91.3 |
| C40D1 | 114 ± 59.2 |
| C41D1 | 152 ± 123 |
| C42D1 | 128 ± 150 |
| C43D1 | 96.9 ± 114 |
| C44D1 | 148 ± 145 |
| C45D1 | 166 ± 97.9 |
| C46D1 | 105 ± 127 |
| C47D1 | 116 ± 104 |
| C48D1 | 131 ± 107 |
| C49D1 | 74.4 ± 82.0 |
| C50D1 | 134 ± 119 |
| C51D1 | 130 ± 136 |
| C52D1 | 93.4 ± 114 |
| C53D1 | 148 ± 188 |
| C54D1 | 166 ± 246 |
| C55D1 | 129 ± 169 |
| C56D1 | 157 ± 227 |
| C57D1 | 380 |
| C58D1 | 338 |
| C59D1 | 451 |
| C60D1 | 362 |
| C61D1 | 292 |
| C62D1 | 287 |
| C63D1 | 411 |
| C65D1 | 362 |
| C67D1 | 326 |
| C68D1 | 584 |
| C69D1 | 345 |
| C70D1 | 313 |
Collected over All-cause mortality: From randomization up to 74 months AEs: From first dose up to 74 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Gilteritinib | 97/137 (70.8%) | 102/134 (76.1%) | 133/134 (99.3%) |
| Salvage Chemotherapy | 85/139 (61.2%) | 71/119 (59.7%) | 113/119 (95%) |
| Event | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| Febrile neutropeniaBlood and lymphatic system disorders | 29/134 | 25/119 |
| PneumoniaInfections and infestations | 24/134 | 8/119 |
| SepsisInfections and infestations | 9/134 | 15/119 |
| AnaemiaBlood and lymphatic system disorders | 14/134 | 1/119 |
| PyrexiaGeneral disorders | 12/134 | 0/119 |
| ThrombocytopeniaBlood and lymphatic system disorders | 11/134 | 1/119 |
| Neutropenic sepsisInfections and infestations | 8/134 | 4/119 |
| Skin infectionInfections and infestations | 7/134 | 0/119 |
| Septic shockInfections and infestations | 5/134 | 5/119 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 0/134 | 5/119 |
| Event | Gilteritinib | Salvage Chemotherapy |
|---|---|---|
| AnaemiaBlood and lymphatic system disorders | 99/134 | 79/119 |
| Blood lactate dehydrogenase increasedInvestigations | 61/134 | 22/119 |
| White blood cell count decreasedInvestigations | 55/134 | 51/119 |
| HypokalaemiaMetabolism and nutrition disorders | 57/134 | 45/119 |
| Platelet count decreasedInvestigations | 55/134 | 50/119 |
| ThrombocytopeniaBlood and lymphatic system disorders | 56/134 | 44/119 |
| Aspartate aminotransferase increasedInvestigations | 56/134 | 19/119 |
| PyrexiaGeneral disorders | 54/134 | 45/119 |
| Neutrophil count decreasedInvestigations | 51/134 | 44/119 |
| Alanine aminotransferase increasedInvestigations | 49/134 | 23/119 |
Intent-to-treat analysis Set (ITT) : The ITT consisted of all participants who were randomized.
| Age, Continuous(Years) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| Mean | 46.9 ± 16.2 | 46.2 ± 15.1 | 46.6 ± 15.7 |
| Sex: Female, Male(Participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| Female | 76 | 70 | 146 |
| Male | 61 | 69 | 130 |
| Ethnicity (NIH/OMB)(Participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 136 | 139 | 275 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 123 | 120 | 243 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 14 | 19 | 33 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| China | 90 | 92 | 182 |
| Russia | 14 | 19 | 33 |
| Malaysia | 15 | 14 | 29 |
| Thailand | 14 | 10 | 24 |
| Singapore | 4 | 4 | 8 |
| Response to First-Line Therapy(Participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| Relapse within 6 months after allogeneic HSCT | 1 | 4 | 5 |
| Relapse after 6 months after allogeneic HSCT | 2 | 2 | 4 |
| Primary refractory without HSCT | 80 | 81 | 161 |
| Relapse within 6 months after CRc and no HSCT | 31 | 29 | 60 |
| Relapse after 6 months after CRc and no HSCT | 23 | 23 | 46 |
| Preselected Salvage Chemotherapy(Participants) | Gilteritinib | Salvage Chemotherapy | Total |
|---|---|---|---|
| High intensity chemotherapy | 110 | 112 | 222 |
| Low intensity chemotherapy | 27 | 27 | 54 |
PDFs hosted by the registry, ClinicalTrials.gov.
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, Statistical analysis plan, Clinical study report
Time frame: Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
Access criteria: Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
Data sharing details ↗ · www.clinicaltrials.astellas.com
Ran from Oct 2017 to primary completion in Dec 2023, 74 months.
Results were first posted in Jan 2025, 1 year after primary completion.
Of 43 industry-sponsored phase 3 interventional studies under Leukemia, Myeloid, Acute, half reached primary completion within 42 months of starting (the median), counting those still running; a quarter within 30 and three quarters within 62. This study's 74 months is above that.
Counting every sponsor, half of 121 studies reached it within 74 months; a quarter within 39 and three quarters within 99.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Gilteritinib