NCT02421939

Brief Summary

The purpose of this study was to determine the clinical benefit of ASP2215 therapy in participants with FMS-like tyrosine kinase (FLT3) mutated acute myeloid leukemia (AML) who were refractory to or had relapsed after first-line AML therapy as shown with overall survival (OS) compared to salvage chemotherapy, and determined the efficacy of ASP2215 therapy as assessed by the rate of complete remission and complete remission with partial hematological recovery (CR/CRh) in these participants. This study also determined the overall efficacy in event-free survival (EFS) and complete remission (CR) rate of ASP2215 compared to salvage chemotherapy.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
371

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Oct 2015

Longer than P75 for phase_3

Geographic Reach
14 countries

126 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 16, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 21, 2015

Completed
6 months until next milestone

Study Start

First participant enrolled

October 20, 2015

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 17, 2018

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

October 17, 2019

Completed
5.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 25, 2025

Completed
Last Updated

December 4, 2025

Status Verified

November 1, 2025

Enrollment Period

2.9 years

First QC Date

April 16, 2015

Results QC Date

September 12, 2019

Last Update Submit

November 27, 2025

Conditions

Keywords

ASP2215Relapsed Acute Myeloid LeukemiaFLT3 MutationgilteritinibRefractory Acute Myeloid LeukemiaAcute Myeloid Leukemia (AML)XOSPATA®

Outcome Measures

Primary Outcomes (2)

  • Duration of Overall Survival (OS)

    Overall survival was defined as the time from the date of randomization until the date of death from any cause (death date - randomization date + 1). For a participant who was not known to have died by the end of study follow-up, OS was censored at the date of last contact (date of last contact - randomized date + 1). The date of last contact was the latest date that the participant was known to be alive. The last contact date was derived for participants alive at the analysis cutoff date. Survival rate and 95% CI were estimated using the Kaplan-Meier method and the Greenwood formula.

    From randomization to until the date of death from any cause (median time of follow-up for OS was 17.8 months)

  • Percentage of Participants With Complete Remission and Complete Remission With Partial Hematological Recovery (CR/CRh) in the Gilteritinib Arm

    The CR/CRh rate was defined as the number of participants who achieved either CR or CRh divided by the number of participants in the analysis population. CR: For participants to be classified as being in CR at, they must have had bone marrow regenerating normal hematopoietic cells and achieved a morphologic leukemia-free state and must had an ANC ≥ 1 x 10\^9/L and platelet count ≥ 100 x 10\^9/L and normal marrow differential with \< 5% blasts, and they were RBC and platelet transfusion independent (defined as 1 week without RBC transfusion and 1 week without platelet transfusion). There was no evidence of extramedullary leukemia. CRh: Participants were classified as CRh if they had marrow blasts \< 5%, partial hematologic recovery ANC ≥ 0.5 x 10\^9/L and platelets ≥ 50 x 10\^9/L, no evidence of extramedullary leukemia and could not be classified as CR.

    From the date of randomization up to at least 112 days

Secondary Outcomes (10)

  • Duration of Event-Free Survival (EFS)

    From the date of randomization until the date of documented relapse, treatment failure or death from any cause (median time of follow-up was 17.8 months)

  • Percentage of Participants With Complete Remission (CR) Rate

    From the date of randomization up to at least 6 months

  • Duration of Leukemia-Free Survival (LFS)

    From the date of first CRc until the date of documented relapse or death for participants who achieved CRc (median time of follow-up was 17.8 months)

  • Duration of Remission

    From the date of first response until the date of documented relapse for participants who achieved CRc or PR (median time of follow-up was 17.8 months)

  • Percentage of Participants With Composite Complete Remission (CRc Rate)

    From the date of randomization up to at least 6 months

  • +5 more secondary outcomes

Study Arms (2)

Gilteritinib

EXPERIMENTAL

Participants received 120 mg dose (3 tablets of 40 mg) orally once a day in continuous 28-day cycles, at least 2 hours after or 1 hour before food. Gilteritinib treatment continued until participants met one of the treatment discontinuation criteria.

Drug: gilteritinib

Salvage Chemotherapy

ACTIVE COMPARATOR

Participants received chemotherapy in 28-day cycles. Participants on Low-Dose Cytarabine (LoDAC) received 20 mg of cytarabine twice daily by subcutaneous (SC) or intravenous (IV) injection for 10 days. Participants on azacitidine received 75 mg/m\^2 daily by SC or IV injection for 7 days. Participants on LoDAC or azacitidine treatment continued until they met discontinuation criteria. Participants on MEC chemotherapy received mitoxantrone 8 mg/m\^2 daily by IV for 5 days, etoposide 100 mg/m\^2 daily by IV for 5 days and cytarabine 1000 mg/m\^2 daily by IV for 5 days (days 1-5). Participants on FLAG-IDA chemotherapy received G-CSF 300 μg/m\^2 daily by SC/IV for 5 days (days 1-5), fludarabine 30 mg/m\^2 daily by IV for 5 days (days 2-6), cytarabine 2000 mg/m\^2 daily by IV for 5 days (days 2-6) and idarubicin 10 mg/m\^2 daily by IV for 3 days (days 2-4). Participants receiving MEC or FLAG-IDA received 1 cycle of therapy and were assessed for response on or after day 15.

Drug: LoDAC (Low Dose Cytarabine)Drug: AzacitidineDrug: MEC (Mitoxantrone, Etoposide, Cytarabine)Drug: FLAG-IDA (Granulocyte-Colony Stimulating Factor (G-CSF), Fludarabine, Cytarabine, Idarubicin)

Interventions

tablet, oral

Also known as: ASP2215, XOSPATA®
Gilteritinib

subcutaneous (SC) or intravenous (IV) injection

Salvage Chemotherapy

SC or IV injection

Salvage Chemotherapy

IV injection

Salvage Chemotherapy

SC (G-CSF) and IV (Fludarabine, Cytarabine, Idarubicin) injection

Salvage Chemotherapy

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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:
  • \. 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:
  • 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:
  • +12 more criteria

You may not qualify if:

  • 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 \[LLN\]).
  • +8 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (126)

Site US10011

Birmingham, Alabama, 35294-0006, United States

Location

Site US10012

Los Angeles, California, 90095-1752, United States

Location

Site US10076

Orange, California, 92868, United States

Location

Site US10073

San Francisco, California, 94143, United States

Location

Site US10067

New Haven, Connecticut, 06504, United States

Location

Site US10045

Gainesville, Florida, 32610, United States

Location

Site US10081

Atlanta, Georgia, 30342, United States

Location

Site US10006

Chicago, Illinois, 60637, United States

Location

Site US10075

Westwood, Kansas, 66205, United States

Location

Site US10074

Louisville, Kentucky, 40202, United States

Location

Site US10048

New Orleans, Louisiana, 70112, United States

Location

Site US10005

Baltimore, Maryland, 21201, United States

Location

Site US10034

Boston, Massachusetts, 02114, United States

Location

Site US10022

Boston, Massachusetts, 02215, United States

Location

Site US10085

Boston, Massachusetts, 02215, United States

Location

Site US10087

Detroit, Michigan, 48201, United States

Location

Site US10057

Minneapolis, Minnesota, 55455, United States

Location

Site US10023

Lebanon, New Hampshire, 03756-1000, United States

Location

Site US10027

Hackensack, New Jersey, 07601, United States

Location

Site US10077

New Brunswick, New Jersey, 08903, United States

Location

Site US10001

Buffalo, New York, 14263, United States

Location

Site US10037

New York, New York, 10029, United States

Location

Site US10008

New York, New York, 10032, United States

Location

Site US10013

New York, New York, 10065, United States

Location

Site US10072

New York, New York, 10065, United States

Location

Site US10046

Syracuse, New York, 13210, United States

Location

Site US10024

Durham, North Carolina, 27710, United States

Location

Site US10078

Winston-Salem, North Carolina, 27157, United States

Location

Site US10044

Cleveland, Ohio, 44106, United States

Location

Site US10084

Columbus, Ohio, 43210, United States

Location

Site US10058

Oklahoma City, Oklahoma, 73104, United States

Location

Site US10041

Hershey, Pennsylvania, 17033, United States

Location

Site US10010

Philadelphia, Pennsylvania, 19104, United States

Location

Site US10080

Philadelphia, Pennsylvania, 19107, United States

Location

Site US10014

Charleston, South Carolina, 29425, United States

Location

Site US10063

Nashville, Tennessee, 37232-0656, United States

Location

Site US10035

Milwaukee, Wisconsin, 53226, United States

Location

Site BE32002

Yvoir, 5530, Belgium

Location

Site CA15004

Edmonton, Alberta, T6G 2G3, Canada

Location

Site CA15001

Hamilton, Ontario, L8V 1C3, Canada

Location

Site CA15015

Toronto, Ontario, M5G 2M9, Canada

Location

Site CA15003

Montreal, Quebec, H1T 2M4, Canada

Location

Site FR33013

Brest, 29609, France

Location

Site FR33002

Le Chesnay, 78157, France

Location

Site FR33010

Lille, 59037, France

Location

Site FR33009

Pessac, 33604, France

Location

Site FR33014

Rennes, 35033, France

Location

Site FR33008

Toulouse, 31059, France

Location

Site DE49009

Dresden, 01307, Germany

Location

Site DE49011

Leipzig, 04103, Germany

Location

Site DE49003

Marburg, 35043, Germany

Location

Site DE49002

München, 81737, Germany

Location

Site DE49010

Tübingen, 72076, Germany

Location

Site IL97201

Ashkelon, 78278, Israel

Location

Site IL97209

Haifa, 31096, Israel

Location

Site IL97203

Jerusalem, 91031, Israel

Location

Site IL97210

Jerusalem, 91120, Israel

Location

Site IL97206

Petah Tikva, 49100, Israel

Location

Site IL97208

Rehovot, 76100, Israel

Location

Site IT39005

Bologna, 40138, Italy

Location

Site IT39010

Brescia, 25126, Italy

Location

Site IT39001

Milan, 20132, Italy

Location

Site IT39004

Palermo, 90146, Italy

Location

Site IT39011

Pavia, 27100, Italy

Location

Site IT39007

Roma, 00189, Italy

Location

Site IT39002

Varese, 21100, Italy

Location

Site JP81002

Nagoya, Aichi-ken, Japan

Location

Site JP81010

Narita, Chiba, Japan

Location

Site JP81026

Yoshida-gun, Fukui, Japan

Location

Site JP81016

Sapporo, Hokkaido, Japan

Location

Site JP81018

Kobe, Hyōgo, Japan

Location

Site JP81017

Tsukuba, Ibaraki, Japan

Location

Site JP81009

Isehara, Kanagawa, Japan

Location

Site JP81006

Yokohama, Kanagawa, Japan

Location

Site JP81012

Sendai, Miyagi, Japan

Location

Site JP81007

Kurashiki, Okayama-ken, Japan

Location

Site JP81014

Sayama, Osaka, Japan

Location

Site JP81020

Kawagoe, Saitama, Japan

Location

Site JP81027

Shimotsuke, Tochigi, Japan

Location

Site JP81005

Chuo-ku, Tokyo, Japan

Location

Site JP81004

Shinagawa-ku, Tokyo, Japan

Location

Site JP81022

Shinjuku-ku, Tokyo, Japan

Location

Site JP81023

Akita, Japan

Location

Site JP81021

Aomori, Japan

Location

Site JP81013

Kumamoto, Japan

Location

Site JP81025

Kyoto, Japan

Location

Site JP81008

Nagasaki, Japan

Location

Site JP81024

Okayama, Japan

Location

Site JP81011

Osaka, Japan

Location

Site PL48002

Gdansk, 80-952, Poland

Location

Site PL48005

Opole, 45-372, Poland

Location

Site PL48004

Wroclaw, 50-367, Poland

Location

Site KR82005

Suwon, Gyeonggi-do, 443380, South Korea

Location

Site KR82010

Busan, 602739, South Korea

Location

Site KR82009

Goyang, 602-715, South Korea

Location

Site KR82003

Jeollanam-do, 519-809, South Korea

Location

Site KR82007

Seoul, 110-744, South Korea

Location

Site KR82004

Seoul, 120-752, South Korea

Location

Site KR82001

Seoul, 135710, South Korea

Location

Site KR82002

Seoul, 137701, South Korea

Location

Site KR82008

Seoul, 138-736, South Korea

Location

Site KR82011

Seoul, 156-707, South Korea

Location

Site ES34009

Badalona, 08025, Spain

Location

Site ES34011

Barcelona, 08035, Spain

Location

Site ES34012

Barcelona, 08036, Spain

Location

Site ES34010

Barcelona, 08916, Spain

Location

Site ES34016

Girona, 17007, Spain

Location

Site ES34005

L'Hospitalet de Llobregat, 08907, Spain

Location

Site ES34014

Salamanca, 37007, Spain

Location

Site ES34017

Valencia, 46026, Spain

Location

Site TW88606

Kaohsiung City, 112, Taiwan

Location

Site TW88604

Kaohsiung City, 83301, Taiwan

Location

Site TW88608

Taichung, 404, Taiwan

Location

Site TW88609

Taichung, 40705, Taiwan

Location

Site TW88601

Tainan, 704, Taiwan

Location

Site TW88603

Taipei, 10002, Taiwan

Location

Site TW88610

Taipei, 10449, Taiwan

Location

Site TW88611

Taipei, 112, Taiwan

Location

Site TW88602

Taipei, 114, Taiwan

Location

Site TW88605

Taoyuan District, 33305, Taiwan

Location

Site TR90001

Ankara, 06100, Turkey (Türkiye)

Location

Site TR90004

Ankara, 06500, Turkey (Türkiye)

Location

Site GB44014

Bournemouth, BH7 7DW, United Kingdom

Location

Site GB44013

Harrow, HA1 3UJ, United Kingdom

Location

Site GB44003

Manchester, M13 9WL, United Kingdom

Location

Site GB44015

Plymouth, PL6 8DH, United Kingdom

Location

Related Publications (4)

  • Ritchie EK, Cella D, Fabbiano F, Pigneux A, Kanda Y, Ivanescu C, Pandya BJ, Shah MV. Patient-reported outcomes from the phase 3 ADMIRAL trial in patients with FLT3-mutated relapsed/refractory AML. Leuk Lymphoma. 2023 May;64(5):938-950. doi: 10.1080/10428194.2023.2186731. Epub 2023 Apr 5.

  • Smith CC, Levis MJ, Perl AE, Hill JE, Rosales M, Bahceci E. Molecular profile of FLT3-mutated relapsed/refractory patients with AML in the phase 3 ADMIRAL study of gilteritinib. Blood Adv. 2022 Apr 12;6(7):2144-2155. doi: 10.1182/bloodadvances.2021006489.

  • Perl AE, Larson RA, Podoltsev NA, Strickland S, Wang ES, Atallah E, Schiller GJ, Martinelli G, Neubauer A, Sierra J, Montesinos P, Recher C, Yoon SS, Hosono N, Onozawa M, Chiba S, Kim HJ, Hasabou N, Lu Q, Tiu R, Levis MJ. Follow-up of patients with R/R FLT3-mutation-positive AML treated with gilteritinib in the phase 3 ADMIRAL trial. Blood. 2022 Jun 9;139(23):3366-3375. doi: 10.1182/blood.2021011583.

  • Perl AE, Martinelli G, Cortes JE, Neubauer A, Berman E, Paolini S, Montesinos P, Baer MR, Larson RA, Ustun C, Fabbiano F, Erba HP, Di Stasi A, Stuart R, Olin R, Kasner M, Ciceri F, Chou WC, Podoltsev N, Recher C, Yokoyama H, Hosono N, Yoon SS, Lee JH, Pardee T, Fathi AT, Liu C, Hasabou N, Liu X, Bahceci E, Levis MJ. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. N Engl J Med. 2019 Oct 31;381(18):1728-1740. doi: 10.1056/NEJMoa1902688.

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

gilteritinibCytarabineAzacitidineMitoxantroneEtoposideGranulocyte Colony-Stimulating FactorfludarabineIdarubicin

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

CytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsArabinonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesAza CompoundsOrganic ChemicalsRibonucleosidesAnthraquinonesAnthronesAnthracenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsQuinonesPolycyclic CompoundsPodophyllotoxinTetrahydronaphthalenesNaphthalenesGlucosidesGlycosidesCarbohydratesColony-Stimulating FactorsGlycoproteinsGlycoconjugatesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological FactorsDaunorubicinAnthracyclinesNaphthacenesAminoglycosides

Results Point of Contact

Title
Clinical Trial Disclosure
Organization
Astellas Pharma Global Development, Inc.

Study Officials

  • Executive Medical Director

    Astellas Pharma Global Development, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

April 16, 2015

First Posted

April 21, 2015

Study Start

October 20, 2015

Primary Completion

September 17, 2018

Study Completion

February 25, 2025

Last Updated

December 4, 2025

Results First Posted

October 17, 2019

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will share

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/.

Shared Documents
STUDY PROTOCOL, SAP, CSR
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.
More information

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