New hope for leukemia patients who Can't handle strong chemo

NCT ID NCT02752035

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

First seen Jun 25, 2026 · Last updated Jul 15, 2026 · Updated 3 times

Summary

This phase 3 study tested a new drug, gilteritinib, combined with azacitidine for people with a specific type of acute myeloid leukemia (AML) that has a FLT3 mutation. The participants were adults who could not receive standard intensive chemotherapy. The study compared the combination to azacitidine alone to see if it helped patients live longer.

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 azacitidine
What this could lead to: If successful, this combination could offer a new treatment option that helps people with this aggressive leukemia live longer without needing intensive chemotherapy.
What could go wrong: This is a phase 3 trial, but results may show only modest survival benefits or increased side effects. The combination may not work for everyone, and long-term outcomes are still uncertain.

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

183 people

The number who actually took part.

Started

Aug 2016

Finished

Dec 2024

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: * Subject is considered an adult according to local regulation at the time of obtaining informed consent. * Subject has a diagnosis of previously-untreated AML according to World Health Organization (WHO) classification \[Swerdlow et al, 2008\] as determined by pathology review at the treating institution. * Subject is positive for FLT3 mutation (internal tandem duplication \[ITD\] or tyrosine kinase domain \[TKD\] \[D835/I836\] mutation) (or for Korea only: ITD alone or ITD with concurrent TKD activating mutation) in bone marrow or whole blood as determined by central laboratory. Note: Only requirement of FLT3 mutation assessment by central laboratory is only applicable to the randomization portion of the study. * Subject is ineligible for intensive induction chemotherapy by meeting at least 1 of the following criteria: * Subject is ≥ 65 years of age and ineligible for intensive induction chemotherapy. * Subject is ≥ 18 to 64 years of age and has any of the following comorbidities: \[Ex-US Only\]: Congestive heart failure (New York Heart Association {NYHA} class ≤ 3) or ejection fraction (Ef) ≤ 50%; \[US Only\]: Severe cardiac disorder e.g. congestive heart failure (New York Heart Association \[NYHA\] class ≤ 3) requiring treatment, ejection fraction ≤ 50%, or chronic stable angina; \[Ex-US Only\]: Creatinine \> 2 mg/dL (177 µmol/L), dialysis or prior renal transplant; \[US Only\]: Creatinine clearance \< 45 mL/min; ECOG performance status ≥ 2; * \[Ex-US Only\]: Known pulmonary disease with decreased diffusion capacity of lung for carbon monoxide (DLCO) and/or requiring oxygen ≤ 2 liters per minute; \[US Only\] Severe pulmonary disorder (e.g., diffusion capacity of lung for carbon monoxide \[DLCO\] ≤ 65% or forced expiratory volume in the first second \[FEV1\] ≤ 65%); Prior or current malignancy that does not require concurrent treatment; Subject has received a cumulative anthracycline dose above 400 mg/m2 of doxorubicin (or cumulative maximum dose of another anthracycline). Any other comorbidity incompatible with intensive chemotherapy must be reviewed and approved by the Medical Monitor during screening and before randomization. * Subject must meet the following criteria as indicated on the clinical laboratory tests: * Serum AST and ALT ≤ 3.0 x Institutional upper limit of normal (ULN) * Serum total bilirubin ≤ 1.5 x Institutional ULN * Serum potassium ≥ Institutional lower limit of normal (LLN) * Serum magnesium ≥ Institutional LLN Repletion of potassium and magnesium levels during the screening period is allowed. * Subject is suitable for oral administration of study drug. * Female subject is eligible to participate if female subject is not pregnant and at least one of the following conditions applies: * Not a woman of childbearing potential (WOCBP); OR * WOCBP agrees to follow the contraceptive guidance starting at screening and continue throughout the study period, and for at least 180 days after the final study drug administration. * Female subject must agree not to breastfeed starting at screening and throughout the study period, and for 60 days after the final study drug administration. * Female subject must not donate ova starting at screening and throughout the study period, and for 180 days after the final study drug administration. * Male subject with female partners of childbearing potential must agree to use contraception as detailed in Contraception Requirements, starting at screening and continue throughout the study period, and for 120 days after the final study drug administration. * Male subject must not donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration. * Subject agrees not to participate in another interventional study while on treatment. Exclusion Criteria: * Subject was diagnosed as acute promyelocytic leukemia (APL). * Subject has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis). * Subject has received previous therapy for AML, with the exception of the following: * Emergency leukapheresis * Hydroxyurea * Preemptive treatment with retinoic acid prior to exclusion of APL ≤ 7 days * Growth factor or cytokine support * Steroids * Subject has clinically active central nervous system leukemia. * Subject has been diagnosed with another malignancy that requires concurrent treatment (with the exception of hormone therapy limited to those therapies that prevent recurrence and/or spread of cancer) or hepatic malignancy regardless of need for treatment. * Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 CYP3A/P-glycoprotein (P-gp). * Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of P-gp with the exception of drugs that are considered absolutely essential for the care of the subject. * Subject requires treatment with concomitant drugs that target serotonin 5-hydroxytryptamine receptor 2B (5HT2BR) or sigma nonspecific receptor with the exception of drugs that are considered absolutely essential for the care of the subject. * Subject has congestive heart failure classified as New York Heart Association Class IV. * Subject with mean Fridericia-corrected QT interval (QTcF) \> 480 ms at screening based on central reading. * Subject with a history of Long QT Syndrome at screening. * \[Ex-US Only\]: Subject has known pulmonary function tests with diffusion capacity of lung for carbon monoxide (DLCO) ≤ 50%, forced expiratory volume in the first second (FEV1) ≤ 60%, dyspnea at rest or requiring oxygen or any pleural neoplasm (Transient use of supplemental oxygen is allowed.) * Subject has active hepatitis B or C or other active hepatic disorder. * Subjects with positive hepatitis B surface antigen (HBsAg) or detectable hepatitis B DNA are not eligible. * Subjects with negative HBsAg, positive hepatitis B core antibody and negative hepatitis B surface antibody will be eligible if hepatitis B DNA is undetectable. * Subjects with antibodies to hepatitis C virus will be eligible if hepatitis C RNA is undetectable * Subject has any condition which makes the subject unsuitable for study participation, including any contraindications of azacitidine. * Subject has a known or suspected hypersensitivity to ASP2215, azacitidine or any components of the formulations used. * \[US Only\]: Subject is ≥ 65 to 74 years of age, suitable for and willing to receive intensive induction chemotherapy.

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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 (AML) Acute myeloid leukemia with FMS-like tyrosine kinase (FLT3) mutation

Contacts and locations

Locations

GHS Cancer Institute

Greenville, South Carolina, 26615, United States
Hackensack University Medical Center - John Theurer Cancer Center

Hackensack, New Jersey, 07601, United States
Hematology-Oncology Associates of Northern NJ

Morristown, New Jersey, 07962, United States
LDS Hospital

Salt Lake City, Utah, 84143, United States
Memorial Sloan-Kettering Cancer Center

New York, New York, 10021, United States
Robert H. Lurie Comprehensive Cancer Center

Chicago, Illinois, 60611-5975, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Rush University Medical Center

Chicago, Illinois, 60612, United States
Site AU61004

Liverpool, New South Wales, 2170, Australia
Site AU61007

Geelong, Victoria, 3220, Australia
Site AU61008

Adelaide, South Australia, SA 5000, Australia
Site BE32003

Brussels, Bruxelles-Capitale, Region de, 1200, Belgium
Site BE32006

Ghent, 9000, Belgium
Site BE32007

Brussels, Brussels Capital, 1090, Belgium
Site CA15002

Toronto, Ontario, M5G 2M9, Canada
Site CA15006

Montreal, Quebec, H4A 3J1, Canada
Site CA15009

Edmonton, Alberta, T6G 2B7, Canada
Site CA15011

Toronto, Ontario, M4N 3M5, Canada
Site DE49002

Tübingen, Baden-Wurttemberg, 72076, Germany
Site DE49003

Berlin, 13353, Germany
Site DE49004

Hanover, Lower Saxony, 30625, Germany
Site DE49005

Frankfurt am Main, Hesse, 60590, Germany
Site DE49007

München, Bavaria, 81737, Germany
Site DE49009

Halle, Saxony-Anhalt, 06120, Germany
Site DE49011

Stuttgart, 70376, Germany
Site DE49012

Braunschweig, Lower Saxony, 38118, Germany
Site DE49015

Rostock, Mecklenburg-Vorpommern, 18057, Germany
Site ES34002

Cáceres, 10003, Spain
Site ES34003

Oviedo, Principality of Asturias, 33011, Spain
Site ES34004

Barcelona, 08035, Spain
Site ES34005

Valencia, 46026, Spain
Site ES34007

Palma de Mallorca, Balearic Islands, 07010, Spain
Site ES34008

Barcelona, 08003, Spain
Site ES34009

Barcelona, 8041, Spain
Site ES34010

Barcelona, 08036, Spain
Site ES34013

Madrid, Spain
Site FR33001

Nantes, Loire-Atlantique, 44093, France
Site FR33002

Pessac, Gironde, 33604, France
Site FR33003

Nîmes, Gard, 30029, France
Site FR33004

Lille, 59020, France
Site FR33006

Lille, 59037, France
Site FR33009

Angers, 49033, France
Site FR33012

Poitiers, Vienne, 86000, France
Site FR33013

Pierre-Bénite, Rhone, 69310, France
Site FR33015

Rouen, Haute-Normandie, 76038, France
Site FR33017

Le Mans, Sarthe, 72037, France
Site FR33018

Rennes, Ille-et-Vilaine, 35033, France
Site FR33019

Montpellier, Herault, 34295, France
Site FR33020

Bayonne, France
Site FR33023

Valenciennes, Nord, 59322, France
Site GB44007

Sheffield, S10 2JF, United Kingdom
Site IT39001

Naples, 80131, Italy
Site IT39004

Milan, 20162, Italy
Site IT39005

Pavia, Italy
Site IT39006

Palermo, 90146, Italy
Site IT39007

Monza, Italy
Site IT39009

Ancona, 60126, Italy
Site IT39011

San Giovanni Rotondo, 71013, Italy
Site IT39012

Florence, Italy
Site IT39014

Novara, Italy
Site IT39015

Bologna, 40138, Italy
Site JP81001

Isehara, Kanagawa, Japan
Site JP81004

Nagasaki, Japan
Site JP81005

Kumamoto, Japan
Site JP81007

Nagoya, Aichi-ken, Japan
Site JP81008

Fukuoka, Japan
Site JP81011

Kurashiki, Okayama-ken, Japan
Site JP81012

Sendai, Miyagi, Japan
Site JP81014

Shinagawa-ku, Tokyo, Japan
Site JP81015

Kobe, Hyōgo, Japan
Site JP81016

Kyoto, Japan
Site JP81017

Nagasaki, Japan
Site JP81018

Anjo, Aichi-ken, Japan
Site JP81019

Toyama, Japan
Site JP81021

Fukuyama, Hiroshima, Japan
Site JP81023

Kanazawa, Ishikawa-ken, Japan
Site JP81024

Gifu, Japan
Site JP81026

Tokushima, Japan
Site JP81027

Matsuyama, Ehime, Japan
Site JP81029

Shibuya-ku, Tokyo, Japan
Site JP81030

Osaka, Japan
Site JP81031

Sapporo, Hokkaido, Japan
Site JP81032

Yokohama, Kanagawa, Japan
Site JP81033

Sapporo, Hokkaido, Japan
Site JP81034

Hitachi, Ibaraki, Japan
Site JP81035

Chiba, Japan
Site JP81036

Osaka, Japan
Site KR82001

Ulsan, Ulsan Gwang'yeogsi, 682-714, South Korea
Site KR82002

Seoul, Seoul Teugbyeolsi, 137-701, South Korea
Site KR82003

Namdong, Incheon Gwang'yeogsiv, 405 760, South Korea
Site KR82006

Seoul, Seoul Teugbyeolsi, 110-744, South Korea
Site KR82010

Hwasun-gun, South Korea
Site KR82012

Seoul, 156-707, South Korea
Site KR82013

Seoul, Seoul Teugbyeolsi, 05505, South Korea
Site KR82014

Busan, 49241, South Korea
Site KR82015

Seongnam-si, South Korea
Site PL48001

Olsztyn, Warmian-Masurian Voivodeship, 10-228, Poland
Site PL48002

Opole, Opole Voivodeship, 45-061, Poland
Site PL48003

Lublin, Lublin Voivodeship, 20-081, Poland
Site PL48004

Warsaw, Masovian Voivodeship, 02-776, Poland
Site TW88601

Taipei, 10002, Taiwan
Site TW88602

Tainan, 704, Taiwan
Site TW88604

Kaohsiung City, 83301, Taiwan
Site TW88605

Kwei Shan Hsiang, Taiwan
Site TW88608

Taipei, 10449, Taiwan
Site TW88609

Tainan, 736, Taiwan
Site TW88610

Taipei, 11217, Taiwan
St. Louis University Cancer Center - Hematology/Oncology

St Louis, Missouri, 63110, United States
UCLA David Geffen School of Medicine

Los Angeles, California, 90095, United States
University of California, Irvine Medical Center

Orange, California, 92868, United States
Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, 10021, United States

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