A Phase 2 interventional study of Gilteritinib and Midostaurin in Acute Myeloid Leukemia, sponsored by PrECOG, LLC.. Completed at 44 sites in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-07-23.
Sponsored by PrECOG, LLC. · Phase 2, Interventional, and Treatment
Eligible untreated patients with FLT3 acute myeloid leukemia (AML) between the ages of 18 and 70 will be randomized to receive gilteritinib or midostaurin during induction and consolidation. Patients will also receive standard chemotherapy of daunorubicin and cytarabine during induction and high-dose cytarabine during consolidation.
Gilteritinib, is an oral drug that works by stopping the leukemia cells from making the FLT3 protein. This may help stop the leukemia cells from growing faster and thus may help make chemotherapy more effective. Gilteritinib has been approved by the Food and Drug Administration (FDA) for patients who have relapsed or refractory AML with a FLT3 mutation but is not approved by the FDA for newly diagnosed FLT3 AML, and its use in this setting is considered investigational.
Midostaurin is an oral drug that works by blocking several proteins on cancer cells, including FLT3 that can help leukemia cells grow. Blocking this pathway can cause death to the leukemic cells. Midostaurin is approved by the FDA for the treatment of FLT3 AML.
The purpose of this study is to compare the effectiveness of gilteritinib to midostaurin in patients receiving combination chemotherapy for FLT3 AML.
Approximately one third of patients with AML have a particular change in their leukemia cells (called a mutation) in a gene called FLT3. The presence of a FLT3 mutation can be used to direct treatment options.
This is an open-label phase II study. Patients will receive standard chemotherapy of daunorubicin and cytarabine during Induction and high-dose cytarabine during Consolidation. Patients will be randomized to gilteritinib or midostaurin. After approximately 90 patient's complete treatment, a review of the effectiveness of gliteritinib compared to midostaurin will be done. If gilteritinib is not as effective as midostaurin, the study may be stopped.
Bone marrow aspirate and biopsy will be done on Day 21 after start of Induction and after Induction to assess response. Patients with a complete response may proceed to consolidation chemotherapy. Another bone marrow aspirate and biopsy will be done after the first cycle of consolidation is complete.
Mandatory prescreening bone marrow and/or blood samples are required for FLT3 testing. Any left-over samples will be requested for future research (optional).
Mandatory bone marrow samples for research are required after Induction and if patient receives Consolidation, after the first cycle of Consolidation.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's enrollment of 181 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →PrECOG, LLC. is the lead sponsor of 19 studies on the registry; 3 are open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 11 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Registration Criteria:
Randomization Eligibility Criteria:
Patient must have previously untreated FLT3 mutated Non M3 AML (FLT3-TKD or FLT3-ITD allowed).
° Standard of care induction 7+3 chemotherapy may start prior to randomization using same regimen and doses as defined in the protocol while awaiting prescreening test results.
Patient must have had no prior systemic therapy for AML, except as noted below:
Patient may not have known active Central Nervous System (CNS) leukemia.
° Prophylaxis with intrathecal chemotherapy is allowed prior to or during induction/consolidation.
Patient must have adequate organ function as measured by the following criteria, obtained ≤ 48 hours prior to randomization except and (LVEF) which can be done ≤ 2 weeks prior to randomization:
A female patient is eligible to participate if she is not pregnant and at least one of the following conditions apply:
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
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.
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.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
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.
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.
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
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 other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
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.
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.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
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.
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.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
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.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Induction: Daunorubicin, cytarabine and gilteritinib. Depending on response, second cycle of Induction may be given. Consolidation: High-dose cytarabine and gilteritinib.
Drug: Gilteritinib · Drug: Daunorubicin · Drug: Cytarabine
Induction: Daunorubicin, cytarabine and midostaurin. Depending on response, second cycle of Induction may be given. Consolidation: High-dose cytarabine and midostaurin.
Drug: Midostaurin · Drug: Daunorubicin · Drug: Cytarabine
Induction: 120 mg orally daily x 14 days starting on day 8 Consolidation: 120 mg orally daily x 14 days starting on day 8 of each cycle (up to 4 cycles)
Also known as: ASP2215
Induction: 50 mg orally twice daily x 14 days beginning on day 8 Consolidation: 50 mg orally twice daily x 14 days beginning on day 8 of each cycle (up to 4 cycles)
Also known as: RYDAPT
First Induction: Daunorubicin 90 mg/m²/day IV Days 1,2,3 Second Induction, if needed: Daunorubicin 45 mg/m²/day IV Days 1,2,3
Also known as: Daunorubicin hydrochloride, Daunomycin, Rubidomycin, Cerubidine
Induction: Cytarabine 100 mg/m²/day continuous infusion x 7 days starting Day 1 Second Induction, if needed: Cytarabine 100 mg/m²/day continuous infusion x 7 days starting Day 1 Consolidation: Cytarabine 3 g/m² (recommend 1.5 g/m² for age ≥ 55 or patients with decreased creatinine clearance) every 12 hours IV Days 1,3,5 or Days 1-3 for 6 doses for up to 4 cycles
Also known as: Cytosar-U, Ara-C, Arabinosyl, Cytosine Arabinoside
FLT3 Mutation Negative Composite Complete Response (CRc) [Includes Complete Response (CR) or CR With Incomplete Hematologic Recovery (CRi)] at End of Induction
FLT3 mutation negative (evaluated by polymerase chain reaction \[PCR\]) Composite Complete Response (CRc) \[includes CR and CRi\] rate after induction treatment and complete MRD assessment. The cut points used for FLT3 mutation negative are 1% (equivalent to 10-2) for FLT3-TKD and 10-4 for FLT3-ITD.
Time frame: 3 months
FLT3 Mutation Negative Complete Response (CR) Rate at End of Induction
CR evaluated by FLT3 testing after Induction
Time frame: 3 months
Minimal Residual Disease (MRD)- CRc Rate at End of Induction
MRD- CRc evaluated by flow cytometry after Induction
Time frame: 3 months
CRc (CR or CRi) Rate at End of Induction
CRc assessed in accordance with 2017 European LeukemiaNet (ELN)
Time frame: 3 months
Event Free Survival (EFS)
EFS assessed in accordance with 2017 ELN
Time frame: 68 months
Overall Survival (OS)
OS assessed in accordance with 2017 ELN
Time frame: 68 months
Number of Participants Treatment-related Adverse Events as Assessed by CTCAE v5.0
Number of participants with abnormal laboratory values and/or adverse events
Time frame: 10 months
| Milestone | Arm A | Arm B |
|---|---|---|
| Started | 90 | 87 |
| Completed | 10 | 6 |
| Not completed | 80 | 81 |
| Withdrew: Adverse event | 5 | 2 |
| Withdrew: Death | 4 | 1 |
| Withdrew: Lack of efficacy | 7 | 10 |
| Withdrew: Physician decision | 7 | 11 |
| Withdrew: Protocol violation | 0 | 3 |
| Withdrew: Withdrawal by subject | 2 | 10 |
| Withdrew: Intercurrent illness, recurrence of disease, subject proceeded to transplant or non-protocol therapy | 55 | 44 |
FLT3 mutation negative (evaluated by polymerase chain reaction \[PCR\]) Composite Complete Response (CRc) \[includes CR and CRi\] rate after induction treatment and complete MRD assessment. The cut points used for FLT3 mutation negative are 1% (equivalent to 10-2) for FLT3-TKD and 10-4 for FLT3-ITD.
| Participants | Arm A | Arm B |
|---|---|---|
| FLT3 Mutation Negative Composite Complete Response (CRc) [Includes Complete Response (CR) or CR With Incomplete Hematologic Recovery (CRi)] at End of Induction | 36 | 41 |
CR evaluated by FLT3 testing after Induction
| Participants | Arm A | Arm B |
|---|---|---|
| FLT3 Mutation Negative Complete Response (CR) Rate at End of Induction | 33 | 37 |
MRD- CRc evaluated by flow cytometry after Induction
| Participants | Arm A | Arm B |
|---|---|---|
| Minimal Residual Disease (MRD)- CRc Rate at End of Induction | 58 | 52 |
CRc assessed in accordance with 2017 European LeukemiaNet (ELN)
| Participants | Arm A | Arm B |
|---|---|---|
| CRc (CR or CRi) Rate at End of Induction | 77 | 63 |
EFS assessed in accordance with 2017 ELN
Results for this outcome have not been posted.
OS assessed in accordance with 2017 ELN
Results for this outcome have not been posted.
Number of participants with abnormal laboratory values and/or adverse events
| Participants | Arm A | Arm B |
|---|---|---|
| Number of Participants Treatment-related Adverse Events as Assessed by CTCAE v5.0 | 82 | 73 |
Collected over Adverse events (AEs) are monitored during the 9 month treatment period for each patient, and for 30 days post-final dose, until resolution to ≤ grade 1 or stabilization, for a total of 10 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Arm A | 23/90 (25.6%) | 48/90 (53.3%) | 83/90 (92.2%) |
| Arm B | 27/87 (31%) | 39/87 (44.8%) | 81/87 (93.1%) |
| Event | Arm A | Arm B |
|---|---|---|
| Febrile neutropeniaBlood and lymphatic system disorders | 24/90 | 18/87 |
| SepsisInfections and infestations | 10/90 | 2/87 |
| Platelet count decreasedInvestigations | 1/90 | 4/87 |
| PyrexiaGeneral disorders | 3/90 | 1/87 |
| PneumoniaInfections and infestations | 1/90 | 2/87 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 2/90 | 2/87 |
| StomatitisGastrointestinal disorders | 1/90 | 2/87 |
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 0/90 | 2/87 |
| BacteraemiaInfections and infestations | 2/90 | 1/87 |
| Cardiac failureCardiac disorders | 2/90 | 0/87 |
| Event | Arm A | Arm B |
|---|---|---|
| Febrile neutropeniaBlood and lymphatic system disorders | 48/90 | 43/87 |
| White blood cell count decreasedInvestigations | 31/90 | 37/87 |
| Platelet count decreasedInvestigations | 38/90 | 33/87 |
| DiarrhoeaGastrointestinal disorders | 36/90 | 25/87 |
| AnaemiaBlood and lymphatic system disorders | 28/90 | 34/87 |
| Neutrophil count decreasedInvestigations | 28/90 | 34/87 |
| StomatitisGastrointestinal disorders | 29/90 | 25/87 |
| HypokalaemiaMetabolism and nutrition disorders | 18/90 | 28/87 |
| Lymphocyte count decreasedInvestigations | 24/90 | 26/87 |
| Aspartate aminotransferase increasedInvestigations | 17/90 | 24/87 |
| Age, Continuous(years) | Arm A | Arm B | Total |
|---|---|---|---|
| Mean | 50 ± 13 | 51 ± 12 | 51 ± 13 |
| Sex: Female, Male(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| Female | 49 | 54 | 103 |
| Male | 41 | 33 | 74 |
| Ethnicity (NIH/OMB)(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| Hispanic or Latino | 9 | 6 | 15 |
| Not Hispanic or Latino | 75 | 77 | 152 |
| Unknown or Not Reported | 6 | 4 | 10 |
| Race (NIH/OMB)(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 3 | 2 | 5 |
| Native Hawaiian or Other Pacific Islander | 0 | 1 | 1 |
| Black or African American | 7 | 8 | 15 |
| White | 69 | 68 | 137 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 11 | 8 | 19 |
| ECOG (Eastern Cooperative Oncology Group) Performance Score(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| 0 | 22 | 22 | 44 |
| 1 | 58 | 56 | 114 |
| 2 | 8 | 7 | 15 |
| 3 | 2 | 1 | 3 |
| Unknown | 0 | 1 | 1 |
| AML Category(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| AML with Myelodysplasia-Related Changes | 7 | 4 | 11 |
| De Novo AML | 80 | 79 | 159 |
| Therapy-Related AML | 3 | 4 | 7 |
| WHO Classification(Participants) | Arm A | Arm B | Total |
|---|---|---|---|
| AML related to previous chemotherapy or radiation | 1 | 1 | 2 |
| AML with certain genetic abnormalities | 34 | 34 | 68 |
| AML with myelodysplasia-related changes | 5 | 2 | 7 |
| AML not otherwise specified | 50 | 50 | 100 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Data is proprietary.
This study is completed, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Gilteritinib
PrECOG, LLC.