New pill shows promise for aggressive leukemia

NCT ID NCT02421939

Disease control Sponsor: Astellas Pharma Global Development, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new drug called gilteritinib against standard chemotherapy in 371 adults with a type of acute myeloid leukemia (AML) that has a specific genetic change (FLT3 mutation) and has either come back or not responded to initial treatment. The goal was to see if gilteritinib helps people live longer and achieve remission. Participants were randomly assigned to receive either gilteritinib pills or one of several chemotherapy regimens.

What this could mean

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

Active substance: gilteritinib (also known as ASP2215, XOSPATA®)
What this could lead to: If successful, this could provide a more effective treatment option for people with a specific type of AML that has come back or not responded to initial therapy.
What could go wrong: This is a completed Phase 3 trial, but results may not apply to all AML patients. Gilteritinib is not a cure and may have side effects; ongoing management is still needed.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

371 people

The number who actually took part.

Started

Oct 2015

Finished

Feb 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

18 years and older

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.

Inclusion Criteria: * Participant has a diagnosis of primary acute myeloid leukemia (AML) or AML secondary to myelodysplastic syndrome (MDS) according to WHO classification (2008) as determined by pathology review at the treating institute. * Participant is refractory to or relapsed after first-line AML therapy (with or without hematopoietic stem cell transplant (HSCT)). * Refractory to first-line AML therapy is defined as: 1\. Participant did not achieve complete remission/complete remission with incomplete hematologic recovery/complete remission with incomplete platelet recovery (CR/CRi/CRp) under initial therapy. A Participant eligible for standard therapy must receive at least one 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 one complete block of induction therapy seen as the optimum choice of therapy to induce remission for this subject. * Untreated first hematologic relapse is defined as: 1. Participant must have achieved a CR/CRi/CRp (criteria as defined by \[Cheson et al, 2003\], see Section 5.3) with first line treatment and has hematologic relapse. * Participant is positive for FLT3 mutation in bone marrow or whole blood as determined by the central lab. A Participant with rapidly proliferative disease and unable to wait for the central lab results can be enrolled based on a local test performed after completion of the last interventional treatment. Participants can be enrolled from a local test result if they have any of the following FLT3 mutations: FLT3 internal tandem duplication (ITD), FLT3 tyrosine kinase domain (TKD)/D835 or FLT3- TKD/I836. * Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2. * Participant is eligible for pre-selected salvage chemotherapy. * Participant must meet the following criteria as indicated on the clinical laboratory tests: * Serum aspartate aminotransferase and alanine aminotransferase ≤ 2.5 x upper limit of normal (ULN) * Serum total bilirubin ≤ 1.5 x ULN * Serum creatinine ≤ 1.5 x ULN or an estimated glomerular filtration rate of \> 50 mL/min as calculated by the Modification of Diet in Renal Disease equation. * Participant is suitable for oral administration of study drug. * Female Participant must either: * Be of non-child bearing potential: 1. post-menopausal (defined as at least 1 year without any menses) prior to Screening, or 2. documented as surgically sterile (at least 1 month prior to Screening) * Or, if of childbearing potential, 1. Agree not to try to become pregnant during the study and for 180 days after the final study administration 2. And have a negative urine pregnancy test at Screening 3. And, if heterosexually active, agree to consistently use highly effective contraception per locally accepted standards in addition to a barrier method starting at Screening and throughout the study period and for 180 days after the final study drug administration. * Female Participant must agree not to breastfeed at Screening and throughout the study period and for 60 days after the final study drug administration. * Female Participant must not donate ova starting at Screening and throughout the study period and for 180 days after the final study drug administration. * Male Participant and their female partners who are of childbearing potential must be using highly effective contraception per locally accepted standards in addition to a barrier method starting at Screening and continue throughout the study period and for 120 days after the final study drug administration. * Male Participant must not donate sperm starting at Screening and throughout the study period and 120 days after the final study drug administration. * Participant agrees not to participate in another interventional study while on treatment. Exclusion Criteria: * Participant was diagnosed as acute promyelocytic leukemia (APL). * Participant has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis). * Participant has AML secondary to prior chemotherapy for other neoplasms (except for MDS). * Participant is in second or later hematologic relapse or has received salvage therapy for refractory disease * Participant has clinically active central nervous system leukemia. * Participant has been diagnosed with another malignancy, unless disease-free for at least 5 years. Participants with treated nonmelanoma skin cancer, in situ carcinoma or cervical intraepithelial neoplasia, regardless of the disease-free duration, are eligible for this study if definitive treatment for the condition has been completed. Participants with organ-confined prostate cancer with no evidence of recurrent or progressive disease are eligible if hormonal therapy has been initiated or the malignancy has been surgically removed or treated with definitive radiotherapy. * Participant has received prior treatment with ASP2215 or other FLT3 inhibitors (with the exception of sorafenib and midostaurin used in first-line therapy regimen as part of induction, consolidation, and/or maintenance). * Participant has clinically significant abnormality of coagulation profile, such as disseminated intravascular coagulation (DIC). * Participant has had major surgery within 4 weeks prior to the first study dose. * Participant has radiation therapy within 4 weeks prior to the first study dose. * Participant has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or Participant with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram performed within 3 months prior to study entry results in a left ventricular ejection fraction that is ≥ 45%. * Participant requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP)3A. * Participants with mean of triplicate Fridericia-corrected QT interval (QTcF) \> 450 ms at Screening based on central reading. * Participants with Long QT Syndrome at Screening. * Participants with hypokalemia and hypomagnesemia at Screening (defined as values below lower limit of normal \[LLN\]). * Participant requires treatment with concomitant drugs that are strong inhibitors or inducers of P glycoprotein (P-gp) with the exception of drugs that are considered absolutely essential for the care of the subject. * Participant requires treatment with concomitant drugs that target serotonin 5-hydroxytryptamine receptor 1 (5HT1R) or 5-hydroxytryptamine receptor 2B (5HT2BR) or sigma nonspecific receptor with the exception of drugs that are considered absolutely essential for the care of the subject. * Participant has an active uncontrolled infection. * Participant is known to have human immunodeficiency virus infection. * Participant has active hepatitis B or C, or other active hepatic disorder. * Participant has any condition which makes the Participant unsuitable for study participation. * Participant has active clinically significant GVHD or is on treatment with systemic corticosteroids for GVHD. * Participant has an FLT3 mutation other than the following: FLT3-ITD, FLT3-TKD/D835 or FLT3-TKD/I836.

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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.

Leukemia acute myeloid - AML

Contacts and locations

Locations

Site BE32002

Yvoir, 5530, Belgium
Site CA15001

Hamilton, Ontario, L8V 1C3, Canada
Site CA15003

Montreal, Quebec, H1T 2M4, Canada
Site CA15004

Edmonton, Alberta, T6G 2G3, Canada
Site CA15015

Toronto, Ontario, M5G 2M9, Canada
Site DE49002

München, 81737, Germany
Site DE49003

Marburg, 35043, Germany
Site DE49009

Dresden, 01307, Germany
Site DE49010

Tübingen, 72076, Germany
Site DE49011

Leipzig, 04103, Germany
Site ES34005

L'Hospitalet de Llobregat, 08907, Spain
Site ES34009

Badalona, 08025, Spain
Site ES34010

Barcelona, 08916, Spain
Site ES34011

Barcelona, 08035, Spain
Site ES34012

Barcelona, 08036, Spain
Site ES34014

Salamanca, 37007, Spain
Site ES34016

Girona, 17007, Spain
Site ES34017

Valencia, 46026, Spain
Site FR33002

Le Chesnay, 78157, France
Site FR33008

Toulouse, 31059, France
Site FR33009

Pessac, 33604, France
Site FR33010

Lille, 59037, France
Site FR33013

Brest, 29609, France
Site FR33014

Rennes, 35033, France
Site GB44003

Manchester, M13 9WL, United Kingdom
Site GB44013

Harrow, HA1 3UJ, United Kingdom
Site GB44014

Bournemouth, BH7 7DW, United Kingdom
Site GB44015

Plymouth, PL6 8DH, United Kingdom
Site IL97201

Ashkelon, 78278, Israel
Site IL97203

Jerusalem, 91031, Israel
Site IL97206

Petah Tikva, 49100, Israel
Site IL97208

Rehovot, 76100, Israel
Site IL97209

Haifa, 31096, Israel
Site IL97210

Jerusalem, 91120, Israel
Site IT39001

Milan, 20132, Italy
Site IT39002

Varese, 21100, Italy
Site IT39004

Palermo, 90146, Italy
Site IT39005

Bologna, 40138, Italy
Site IT39007

Roma, 00189, Italy
Site IT39010

Brescia, 25126, Italy
Site IT39011

Pavia, 27100, Italy
Site JP81002

Nagoya, Aichi-ken, Japan
Site JP81004

Shinagawa-ku, Tokyo, Japan
Site JP81005

Chuo-ku, Tokyo, Japan
Site JP81006

Yokohama, Kanagawa, Japan
Site JP81007

Kurashiki, Okayama-ken, Japan
Site JP81008

Nagasaki, Japan
Site JP81009

Isehara, Kanagawa, Japan
Site JP81010

Narita, Chiba, Japan
Site JP81011

Osaka, Japan
Site JP81012

Sendai, Miyagi, Japan
Site JP81013

Kumamoto, Japan
Site JP81014

Sayama, Osaka, Japan
Site JP81016

Sapporo, Hokkaido, Japan
Site JP81017

Tsukuba, Ibaraki, Japan
Site JP81018

Kobe, Hyōgo, Japan
Site JP81020

Kawagoe, Saitama, Japan
Site JP81021

Aomori, Japan
Site JP81022

Shinjuku-ku, Tokyo, Japan
Site JP81023

Akita, Japan
Site JP81024

Okayama, Japan
Site JP81025

Kyoto, Japan
Site JP81026

Yoshida-gun, Fukui, Japan
Site JP81027

Shimotsuke, Tochigi, Japan
Site KR82001

Seoul, 135710, South Korea
Site KR82002

Seoul, 137701, South Korea
Site KR82003

Jeollanam-do, 519-809, South Korea
Site KR82004

Seoul, 120-752, South Korea
Site KR82005

Suwon, Gyeonggi-do, 443380, South Korea
Site KR82007

Seoul, 110-744, South Korea
Site KR82008

Seoul, 138-736, South Korea
Site KR82009

Goyang, 602-715, South Korea
Site KR82010

Busan, 602739, South Korea
Site KR82011

Seoul, 156-707, South Korea
Site PL48002

Gdansk, 80-952, Poland
Site PL48004

Wroclaw, 50-367, Poland
Site PL48005

Opole, 45-372, Poland
Site TR90001

Ankara, 06100, Turkey (Türkiye)
Site TR90004

Ankara, 06500, Turkey (Türkiye)
Site TW88601

Tainan, 704, Taiwan
Site TW88602

Taipei, 114, Taiwan
Site TW88603

Taipei, 10002, Taiwan
Site TW88604

Kaohsiung City, 83301, Taiwan
Site TW88605

Taoyuan, 33305, Taiwan
Site TW88606

Kaohsiung City, 112, Taiwan
Site TW88608

Taichung, 404, Taiwan
Site TW88609

Taichung, 40705, Taiwan
Site TW88610

Taipei, 10449, Taiwan
Site TW88611

Taipei, 112, Taiwan
Site US10001

Buffalo, New York, 14263, United States
Site US10005

Baltimore, Maryland, 21201, United States
Site US10006

Chicago, Illinois, 60637, United States
Site US10008

New York, New York, 10032, United States
Site US10010

Philadelphia, Pennsylvania, 19104, United States
Site US10011

Birmingham, Alabama, 35294-0006, United States
Site US10012

Los Angeles, California, 90095-1752, United States
Site US10013

New York, New York, 10065, United States
Site US10014

Charleston, South Carolina, 29425, United States
Site US10022

Boston, Massachusetts, 02215, United States
Site US10023

Lebanon, New Hampshire, 03756-1000, United States
Site US10024

Durham, North Carolina, 27710, United States
Site US10027

Hackensack, New Jersey, 07601, United States
Site US10034

Boston, Massachusetts, 02114, United States
Site US10035

Milwaukee, Wisconsin, 53226, United States
Site US10037

New York, New York, 10029, United States
Site US10041

Hershey, Pennsylvania, 17033, United States
Site US10044

Cleveland, Ohio, 44106, United States
Site US10045

Gainesville, Florida, 32610, United States
Site US10046

Syracuse, New York, 13210, United States
Site US10048

New Orleans, Louisiana, 70112, United States
Site US10057

Minneapolis, Minnesota, 55455, United States
Site US10058

Oklahoma City, Oklahoma, 73104, United States
Site US10063

Nashville, Tennessee, 37232-0656, United States
Site US10067

New Haven, Connecticut, 06504, United States
Site US10072

New York, New York, 10065, United States
Site US10073

San Francisco, California, 94143, United States
Site US10074

Louisville, Kentucky, 40202, United States
Site US10075

Westwood, Kansas, 66205, United States
Site US10076

Orange, California, 92868, United States
Site US10077

New Brunswick, New Jersey, 08903, United States
Site US10078

Winston-Salem, North Carolina, 27157, United States
Site US10080

Philadelphia, Pennsylvania, 19107, United States
Site US10081

Atlanta, Georgia, 30342, United States
Site US10084

Columbus, Ohio, 43210, United States
Site US10085

Boston, Massachusetts, 02215, United States
Site US10087

Detroit, Michigan, 48201, United States

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