New drug gilteritinib takes on standard therapy in FLT3 leukemia trial

NCT ID NCT03836209

Disease control Sponsor: PrECOG, LLC. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 2 times

Summary

This Phase 2 trial tests whether the oral drug gilteritinib works better than midostaurin for adults with a specific genetic subtype of acute myeloid leukemia (FLT3-mutated AML). All 181 participants also receive standard chemotherapy. The goal is to see which drug leads to more complete remissions without detectable leukemia cells. Gilteritinib is already approved for relapsed AML but is investigational for newly diagnosed patients.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Gilteritinib and midostaurin (oral drugs targeting FLT3 protein) plus standard chemotherapy (daunorubicin and cytarabine)
What this could lead to: If gilteritinib works better than midostaurin, it could become a new standard treatment for newly diagnosed FLT3-mutated AML, improving remission rates.
What could go wrong: This is a Phase 2 trial with only 181 participants, so results may not be definitive. Gilteritinib is not yet approved for newly diagnosed patients, and side effects from the drugs or chemotherapy could be serious.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

181 people

The number who actually took part.

Started

Dec 2019

Finished

Jun 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Registration Criteria: * Any patient undergoing bone marrow biopsy with suspicion of or known diagnosis of acute myeloid leukemia (AML) will be asked to sign a Prescreening Consent to allow for centralized testing of bone marrow/peripheral blood samples. 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: * Hydroxyurea and emergent leukapheresis or preemptive treatment with retinoic acid prior to exclusion of Acute Promyelocytic Leukemia (APL) allowed. * Prior therapy for myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPN) (e.g., thalidomide or lenalidomide, interferon, jakafi, cytokines, 5-azacytidine or decitabine, histone deacetylase inhibitors). * Initiation of standard of care 7+3 induction chemotherapy using same regimen and doses as defined in protocol while awaiting prescreening test results * Patient may not have received hypomethylating agent within 21 days. * Patient may not have M3 AML. * Patient may not have AML with known Core Binding Factor -t(8;21), inv(16), t(16;16). * 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 an Eastern Cooperative Oncology Group (ECOG) performance status of 0-3. * Patient must be age ≥ 18 years to ≤ 70 years. * Patient must be able to understand and willing to sign Institutional Review Board (IRB)-approved informed consent. * Patient must be willing to provide mandatory bone marrow and blood samples for research. * Patient must have adequate organ function as measured by the following criteria, obtained ≤ 48 hours prior to randomization except ECG and left ventricular ejection fraction (LVEF) which can be done ≤ 2 weeks prior to randomization: * Serum creatinine ≤ 1.5x institutional upper limit of normal (ULN), or if serum creatinine outside normal range, then glomerular filtration rate (GFR) \>40 mL/min as measured by Cockcroft-Gault formula. * Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) ≤ 3x ULN, unless secondary to leukemia. * Serum total or direct bilirubin \<2 mg/dL, unless due to Gilbert's, hemolysis or leukemic infiltration. * Fridericia-Corrected QT Interval (QTcF) interval ≤ 500 msec (using Friderica's correction). * Left Ventricular Ejection Fraction \>45%. * The patient may not be known to have hypokalemia and/or hypomagnesemia that does not respond to supplementation. * A female patient is eligible to participate if she is not pregnant and at least one of the following conditions apply: * Not a woman of childbearing potential (WOCBP) OR * WOCBP who agrees to follow the contraceptive guidance throughout the treatment period and for at least 180 days after the final study drug administration. * Female patient must agree not to breastfeed or donate ova starting at treatment and throughout the study period, and for at least 180 days after the final study drug administration. * A male patient must agree not to donate sperm starting at treatment and throughout the study period, and for at least 120 days after the final study drug administration. * A male patient with female partner(s) of child-bearing potential must agree to use contraception during the treatment period, and for at least 120 days after the final study drug administration. * Male patient with a pregnant or breastfeeding partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy or time partner is breastfeeding throughout the treatment period, and for at least 120 days after the final study drug administration. * Patient may not have another malignancy that could interfere with the evaluation of safety or efficacy of this combination. * Patient may not have a history of Long QT Syndrome. * Patient may not have evidence of uncontrolled angina, severe uncontrolled ventricular arrhythmias, electrocardiographic evidence of acute ischemia, or congestive heart failure (CHF) New York Heart Association (NYHA) Class 3 or 4. Patient may also not have a history of CHF NYHA Class 3 or 4 in the past, unless a prescreening echocardiogram (ECHO) or multigated acquisition scan (MUGA) performed within 2 weeks prior to study entry with results of left ventricular ejection fraction \>45%. * Patient may not have had major surgery or radiation therapy within 4 weeks of registration. * Patient may not require treatment with concomitant drugs that are strong inducers of CYP3A and P-gp. * Patient with a known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent are not eligible. * Patient with known gastrointestinal (GI) disease or prior GI procedure that could interfere with the oral absorption or tolerance of gilteritinib or midostaurin including difficulty swallowing are not eligible. * Patient with any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of the treatment according to the protocol are not eligible. * Patient may not participate in any other therapeutic clinical trials, including those with other investigational agents not included in this trial during treatment on this study without prior approval from PrECOG.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute myeloid leukemia

Contacts and locations

Locations

Atlantic Health Systems/Morristown Medical Center

Morristown, New Jersey, 07962, United States
Augusta University Medical Center

Augusta, Georgia, 30912, United States
East Carolina University

Greenville, North Carolina, 27834, United States
Franciscan Health Indianapolis

Indianapolis, Indiana, 46237, United States
Geisinger Medical Center

Danville, Pennsylvania, 17822, United States
HonorHealth Research Institute

Scottsdale, Arizona, 85258, United States
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Kaiser Permanente Oakland

Oakland, California, 94611, United States
Kaiser Permanente Roseville

Roseville, California, 95661, United States
Kaiser Permanente Santa Clara

Santa Clara, California, 94115, United States
LDS Hospital

Salt Lake City, Utah, 84143, United States
Marshfield Medical Center

Marshfield, Wisconsin, 54449, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic- Jacksonville, FL

Jacksonville, Florida, 32224, United States
Mayo Clinic- Rochester, MN

Rochester, Minnesota, 55905, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Mount Sinai

New York, New York, 10029, United States
MultiCare

Spokane, Washington, 99218, United States
Northwell Health

Lake Success, New York, 11042, United States
Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611, United States
Ochsner Clinic Foundation

New Orleans, Louisiana, 70121, United States
Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States
SUNY Upstate Medical University

Syracuse, New York, 13210, United States
St. Joseph's Mercy Hospital

Ann Arbor, Michigan, 48106, United States
Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States
Tufts Medical Center

Boston, Massachusetts, 02111, United States
UC Irvine Health

Orange, California, 92868, United States
UCLA

Los Angeles, California, 90095, United States
UW Cancer Center at ProHealth Care

Waukesha, Wisconsin, 53188, United States
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University of California, San Francisco-Fresno (University Oncology Associates)

Clovis, California, 93611, United States
University of Chicago Medical Center

Chicago, Illinois, 60451, United States
University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States
University of Kentucky Markey Cancer Center

Lexington, Kentucky, 40536, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68105, United States
University of Oklahoma Stephenson Cancer Center

Oklahoma City, Oklahoma, 73104, United States
University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States
University of Rochester Medical Center

Rochester, New York, 14642, United States
University of Wisconsin Clinical Science Center

Madison, Wisconsin, 53792, United States
Vanderbilt University

Nashville, Tennessee, 37232, United States
Weill Cornell Medicine New York Presbyterian Hospital

New York, New York, 10065, United States
West Virginia University

Morgantown, West Virginia, 26506, United States

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