New hope for Hard-to-Treat leukemia? enzomenib trial launches

NCT ID NCT04988555

Disease control Sponsor: Sumitomo Pharma America, Inc. Source: ClinicalTrials.gov ↗

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

Summary

This study tests a new oral drug called enzomenib (DSP-5336) in people with acute leukemia and certain other blood cancers that have not responded to standard treatments. The trial has two phases: first, finding the safest dose, and then checking how well it works alone or combined with other drugs. About 606 participants will be enrolled across multiple sites.

What this could mean

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

Active substance: Enzomenib (DSP-5336)
What this could lead to: If successful, this could lead to a new targeted treatment option for certain types of acute leukemia that have not responded to standard therapies.
What could go wrong: This is an early-phase trial (phase 1/2) with a small number of participants, so safety and effectiveness are not yet proven. Side effects are unknown, and the drug may not work for all patients.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 606 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2022

Expected to finish

Dec 2027

An estimate. End dates often move.

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

12 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: For patients in Phase I: 1. Have a diagnosis of relapsed or refractory AML, ALL or acute leukemia of ambiguous lineage according to World Health Organization (WHO) 2022 classification, or, in selected sites and regions, a diagnosis of MDS or MM as determined by pathology review at the treating institution, and whose disease has progressed after available standard therapies known to be active for their AML, ALL, or acute leukemia of ambiguous lineage or, in selected sites and regions, for MM or MDS. If acute leukemia patients are transformation from MDS or other hematologic malignancies, patients need to receive available standard therapies as acute leukemia after AML transformation and before enrolling this trial. In regions or countries where required by regulatory authorities, participants must have a documented KMT2A (MLL) fusion or NPM1 mutation, including those with coexisting FLT3 genomic alterations and/or IDH1/2 mutation. Participants who are candidates for stem cell transplantation must have been offered this therapeutic option. For patients with MDS (selected sites and regions): 1. Patients with MDS must have bone marrow blasts ≥ 5% 2. Patients with MDS must have relapsed or refractory disease and have exhausted available standard therapies including at least 2 cycles of treatment with HMA For patients with MM (selected sites and regions): 3. Have a confirmed diagnosis of multiple myeloma according to International Myeloma Working Group (IMWG) 2016 classification (Kumar, 2016) and whose disease has progressed after treatment with a minimum of 3 prior anti-myeloma regimens including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 monoclonal antibody (mAb); patients must not be candidates for available therapies with established clinical benefit 4. Have measurable disease as defined in the protocol 5. Meet the laboratory parameters set in the protocol For patients with relapsed/refractory AML in the venetoclax and azacitidine combination cohort (in countries and sites where permitted): 6. Have MLLr or NPM1m. For patients with relapsed/refractory AML in the gilteritinib combination cohort (in countries and sites where permitted): 7. Have MLLr or NPM1m AND any of the following FLT3 mutations: FLT3-ITD, FLT3-TKD/D835 or FLT3-TKD/I836. For patients with relapsed/refractory AML with NPM1 enrolled in the RP2D confirmation cohort: 8. Must have ≥5% blasts in bone marrow by morphologic assessment 9. Must not have received prior treatment with a menin inhibitor For patients with newly diagnosed AML: 10. Must have AML as defined by WHO 2022 criteria with a documented MLLr or NPM1m (patients with AML characterized by MLL partial tandem duplications, MLL deletions, or trisomy 11 are not eligible) 11. Must not have received treatment for AML with the exception of hydroxyurea for control of white blood cell counts. For patients in Phase 2: 2. Have a confirmed diagnosis of relapsed AML or ALL according to WHO 2022 classification, as determined by pathology review at the treating institution, and who have ≥5% blasts by morphologic assessment in the bone marrow. Patients with extramedullary disease or peripheral blasts as the only manifestation of relapse are not eligible. Patients must have received clinically applicable standard therapies with confirmed survival benefit. Patients must not have had prior exposure to a menin inhibitor. 3. Have a documented KMT2A (MLL)-fusion assessed at relapse or immediately prior to the determination of refractory status. KMT2A genetic alterations other than fusions (eg, KMT2A-PTD, amplification, point mutation) are not permitted. For all patients: 4. Be \> 18 years of age. For countries and sites where approved, for DSP-5336 monotherapy, acute leukemia patients ≥12 years of age who weigh ≥40 kg may be enrolled. 5. Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤2. 6. For monotherapy, WBC below 30,000/μ at enrollment. For the combination arms, WBC count must be below 25,000/uL at enrollment and prior to starting treatment. (Hydroxyurea and steroids for cytoreduction purposes are allowed prior to enrollment and during study treatment) 7. Clearance of creatinine level ≥ 50 ml/min, assessed by the CPK-EPI formula (2021 version and Cystatin C not required) 8. Total bilirubin ≤1.5 the upper limit of normal (ULN) (or ≤2.0 ULN for patients with known Gilbert's syndrome) 9. Aspartate aminotransferase (AST) ≤3.0 times ULN 10. Alanine aminotransferase (ALT) ≤3.0 times ULN 11. Any prior treatment-related toxicities resolved to Grade ≤1 prior to enrollment, with the exception of Grade ≤2 alopecia or neuropathy 12. Be willing to attend study visits as required by the protocol 13. Have an estimated life expectancy ≥3 months, based on the investigator's assessment 14. Females of childbearing potential must have a negative serum pregnancy test. Females of childbearing potential are defined as women who have (1) experienced menarche and have not undergone sterilization procedures (hysterectomy, or bilateral oophorectomy), or have (2) not experienced menopause as defined in the protocol. 15. All men and all women of childbearing potential and male patients' partners who are women of childbearing potential are required to use a highly effective method of contraception during the study and for 6 months (for females and males alike) after the last dose of study drug. Further guidelines noted in protocol. 16. Have AML/ALL/MDS/MM bone marrow material suitable for genomic analysis of AML,ALL, MDS, or MM genetic alterations. Note: If a bone marrow material is insufficient, an alternative suitable tissue (ex: peripheral blood) must be provided. Exclusion Criteria: 1. Has a left ventricular ejection fraction (LVEF) \<50%, as determined by ECHO 2. Histological diagnosis of acute promyelocytic leukemia 3. Received systemic calcineurin inhibitors within 2 weeks prior to the first dose of DSP 5336 4. Have abnormal ECGs at screening that are clinically significant, such as (QTc \>480 msec, with QTc corrected according to Fridericia's formula (QTcF). For clinical sites in the UK, have abnormal ECGs at screening that are clinically significant, such as QTc ≥470 msec and ≥450 msec with QTc corrected according to Fridericia's formula (QTcF), for females and males, respectively. In addition, patients with a history of prolonged QT syndrome or who are required to take therapies associated with QT-interval prolongation are excluded. Note: In case of bundle branch block, QT interval correction can be performed. 5. Has an active and uncontrolled, bacterial, viral, or fungal infection requiring parenteral therapy. Note: Patients must be afebrile with negative blood cultures at least 72 hours prior to Cycle 1 Day 1. 6. Receives concurrent sensitive substrates with a narrow safety window or strong inhibitors or inducers of CYP3A4/5, including specifically: ketoconazole, isavuconazole and itraconazole. Other antifungals that are used as standard of care to prevent or treat infections are permitted. If a patient is on one of the excluded azole class antifungals, he/she can be taken off or switched to a permitted azole 7 or more days prior to first dose, then the patient could be allowed on study (Arm B) with approval of the medical monitor. 7. Had major surgery within 28 days prior to the first dose of DSP-5336 8. Has active central nervous system leukemia (prophylactic intrathecal chemotherapy is allowed). 9. Underwent HSCT or chimeric antigen receptor cell (CAR-T) therapy or other modified T-cell therapy within 60 days prior to the first dose of DSP-5336. For clinical sites in the UK, underwent CAR-T therapy or other modified T-cell therapy within 6 months prior to the first dose of DSP-5336. 10. Received a donor lymphocyte infusion within 28 days prior to the first dose of DSP-5336, or receiving immunosuppressive therapy post-HSCT at the time of screening, or with clinically active GVHD or GVHD requiring active medical intervention other than the use of topical steroids for ongoing cutaneous GVHD 11. Received antineoplastic agents (except hormonal therapies as adjuvant maintenance for breast or prostate cancers if a patient is taking before starting study treatment, and hydroxyurea given for controlling blast cells) or other investigational treatment within 7 days or 5 half-lives, whichever is shortest, prior to the first dose of DSP-5336 12. In the opinion of the treating investigator, have any concurrent conditions that could pose an undue medical hazard or interfere with interpretation of study results; these conditions include, but are not limited to: clinically significant non-healing or healing wounds; concurrent congestive heart failure (New York Heart Association Functional Classification Class III or IV; see Section 21.2); concurrent unstable angina; concurrent cardiac arrhythmia requiring treatment (excluding asymptomatic atrial fibrillation); recent (within the prior 6 months) myocardial infarction; acute coronary syndrome within the previous 6 months; significant pulmonary disease (shortness of breath at rest or on mild exertion), eg, due to concurrent severe obstructive pulmonary disease, concurrent hypertension not controlled with concomitant medication, or diabetes mellitus with more than 2 episodes of ketoacidosis in the prior 6 months 13. Have a known detectable viral load for human immunodeficiency virus or hepatitis C, or evidence of hepatitis B surface antigen, all being indicative of active infection. For sites in Japan, Taiwan, and Korea only: Hepatitis B core (HBc) antibody or hepatitis B surface (HBs) antibody test should be performed if HBsAg is negative. If HBc antibody or HBs antibody test is positive, HBV DNA quantification test should be performed to confirm that HBV DNA is negative. 14. Have severe dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally, including the inability to swallow oral medication 15. Have cognitive, psychological, or psychosocial impediment that would impair the ability of the patient to receive therapy according to the protocol, or adversely affect the ability of the patient to comply with the informed consent process, protocol, or protocol-required visits and procedures 16. Are pregnant or breastfeeding or planning to become pregnant. Note: Patients who are breastfeeding may be enrolled if they interrupt breastfeeding prior to the first dose of any study drugs and do not feed the baby with breast milk expressed after receiving the first dose of any study drugs. Breastfeeding should not be resumed for at least 6 months after the last dose of study drug 17. Have any history or complication of interstitial lung disease (for sites in Japan in Phase 1 dose escalation). For clinical sites in the EU, have a history of Grade ≥ 2 drug-induced interstitial lung disease or Grade ≥ 2 non-infectious pneumonitis within 6 months of starting study treatment. 18. Have a history of Torsades de Pointes 19. Received systemic calcineurin inhibitors within 4 weeks prior to the first dose of DSP-5336 20. Have plasma cell leukemia (\>2.0 x 109 /L plasma cells in blood by standard differential) (for patients with MM) 21. For patients intending to enroll into the combination cohort with gilteritinib: Patients must be gilteritinib-naïve or sensitive and have not received a FLT3 inhibitor in the relapsed refractory setting (prior FLT3 inhibitor in front line therapy is allowed) 22. Have a known intolerance of hypersensitivity reaction to components of the investigational medicinal product 23. For clinical sites in the UK: In Arm E (DSP-5336 + venetoclax/azacitidine), have received a live vaccine within 30 days prior to the first dose of DSP-5336

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Conditions

The condition(s) this trial relates to.

acute leukemia of ambiguous lineage acute lymphoblastic leukemia acute myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes plasma cell myeloma Precursor Cell Lymphoblastic Leukemia-Lymphoma Recurrence

As listed by the trial registrant

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

Leukemia, lymphocytic, acute Leukemia, myeloid, acute Multiple myeloma Myelodysplastic syndromes

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

101 sites in 12 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

AZ Delta
RECRUITING

Roeselare, Belgium
Allegheny Health Network
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Atlantic Health
RECRUITING

Morristown, New Jersey, 07960, United States
Atrium Wake Forest Baptist Medical Center
RECRUITING

Winston-Salem, North Carolina, 27157, United States
Azienda USL della Romagna, Ospedale Santa Maria delle Croci di Ravenna
NOT_YET_RECRUITING

Ravenna, 48121, Italy
Bristol Hematology & Oncology Centre
RECRUITING

Bristol, United Kingdom
CHU Bordeaux
NOT_YET_RECRUITING

Talence, 33000, France
CHU de Nantes
NOT_YET_RECRUITING

Nantes, 44093, France
CHU de Nice - Hôpital l'Archet 1
RECRUITING

Nice, France
Centre Hospitalier Le Mans
RECRUITING

Le Mans, France
Centre Hospitalier Universitaire d'Angers
RECRUITING

Angers, France
Centre Hospitalier Universitaire de Limoges
RECRUITING

Limoges, France
Chonnam National University Hwasun Hospital
RECRUITING

Hwasun, South Korea
Christie Hospital NHS Foundation Trust
RECRUITING

Manchester, United Kingdom
Churchill Hospital Oxford
NOT_YET_RECRUITING

Oxford, United Kingdom
Colorado Blood Cancer Institute
RECRUITING

Denver, Colorado, 80218, United States
Columbia University
COMPLETED

New York, New York, 10032, United States
Complexo Hospitalario Universitario De Santiago
RECRUITING

Santiago de Compostela, Spain
Duke University
RECRUITING

Durham, North Carolina, 27705, United States
Fukushima Medical University Hospital
RECRUITING

Fukushima, Fukushima, 960-1295, Japan
Fundacion Instituto de Investigacion Marques de Valdecilla
RECRUITING

Santander, Spain
Georgetown Lombardi Comprehensive Cancer Center
NOT_YET_RECRUITING

Washington D.C., District of Columbia, 20007, United States
Hoag Family Cancer Center
RECRUITING

Newport Beach, California, 92663, United States
Hokkaido University Hospital
NOT_YET_RECRUITING

Hokkaido, 060-8648, Japan
Hopital Avicenne
NOT_YET_RECRUITING

Bobigny, 93000, France
Hopital Claude Huriez
NOT_YET_RECRUITING

Lille, 59037, France
Hopital Saint-Louis
RECRUITING

Paris, France
Hospices Civils de Lyon
RECRUITING

Lyon, France
Hospital General Universitario De Albacete
RECRUITING

Albacete, Spain
Hospital San Pedro de Alcantara
RECRUITING

Cáceres, Spain
Hospital Universitari Vall D'Hebron
RECRUITING

Barcelona, Spain
Hospital Universitario Central De Asturias
RECRUITING

Oviedo, Spain
Hospital Universitario De Gran Canaria Dr. Negrin
RECRUITING

Las Palmas, Spain
Hospital Universitario de Salamanca
RECRUITING

Salamanca, Spain
Hospital Universitario y Politecnico La Fe
RECRUITING

Valencia, Spain
Huntsman Cancer Institute
RECRUITING

Salt Lake City, Utah, 84112, United States
IRCCS Azienda Ospedaliero-Universitaria Di Bologna, Policlinico Sant'Orsola
RECRUITING

Bologna, Italy
IRCCS istituto Romagnolo per lo studio dei tumori "Dino Amadori"
RECRUITING

Meldola, Italy
Institut Catala d'Oncologia
RECRUITING

Barcelona, Spain
Institut Gustave Roussy
NOT_YET_RECRUITING

Villejuif, 94800, France
Institut Paoli-Calmettes
RECRUITING

Marseille, France
Intermountain Healthcare
RECRUITING

Salt Lake City, Utah, 84143, United States
Istituto Oncologico Veneto (IOV), IRCCS
NOT_YET_RECRUITING

Padua, Italy
Johns Hopkins Main Center
RECRUITING

Baltimore, Maryland, 21287, United States
King's College Hospital
RECRUITING

London, United Kingdom
Kyushu University Hospital
RECRUITING

Fukuoka, 812-8582, Japan
MD Anderson Cancer Center
RECRUITING

Madrid, Spain
MDACC
RECRUITING

Houston, Texas, 77030, United States
Massachusetts General Hospital
RECRUITING

Boston, Massachusetts, 02114, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
Miami Cancer Institute
NOT_YET_RECRUITING

Miami, Florida, 33176, United States
Moffitt Cancer Center
NOT_YET_RECRUITING

Tampa, Florida, 33612, United States
Mount Sinai Hospital
COMPLETED

New York, New York, 10029, United States
NHS Lothian Western General
NOT_YET_RECRUITING

Edinburgh, EH4 2XU, United Kingdom
Nagasaki University Hospital
RECRUITING

Nagasaki, Nagasaki, 852-8501, Japan
National Cancer Center Hospital East
RECRUITING

Kashiwa-shi, Chiba, 277-8577, Japan
National Cheng Kung University Hospital
RECRUITING

Tainan, Taiwan
National Taiwan University Hospital
RECRUITING

Taipei, Taiwan
National University Cancer Institute
RECRUITING

Singapore, 119074, Singapore
Nippon Medical School Hospital
RECRUITING

Bunkyo-ku, Tokyo, 113-8603, Japan
Northwestern
RECRUITING

Chicago, Illinois, 60611, United States
Okayama University Hospital
RECRUITING

Okayama, 700-8558, Japan
Oncology Associates of Oregon
RECRUITING

Eugene, Oregon, 97401, United States
Osaka University Hospital
RECRUITING

Osaka, 565-0871, Japan
Ospedale Policlinico San Martino, IRCCS
RECRUITING

Genoa, Italy
Ospedale di Busto Arsizio
NOT_YET_RECRUITING

Busto Arsizio, 21052, Italy
PU A. Gemelli, Università Cattolica del Sacro Cuore
RECRUITING

Rome, Italy
Roswell Park Comprehensive Cancer Center
RECRUITING

Buffalo, New York, 14203, United States
Rutgers Cancer Institute of New Jersey
RECRUITING

New Brunswick, New Jersey, 08901, United States
Samsung Medical Center
RECRUITING

Seoul, South Korea
Sarah Cannon Research Institute
NOT_YET_RECRUITING

London, United Kingdom
Seoul National University Hospital
RECRUITING

Seoul, South Korea
Sibley Memorial Hospital
RECRUITING

Baltimore, Maryland, 20016, United States
Sidney Kimmel Comprehensive Cancer Center
RECRUITING

Philadelphia, Pennsylvania, 19107, United States
Stanford University
NOT_YET_RECRUITING

Palo Alto, California, 94304, United States
Taichung Veterans General Hospital
RECRUITING

Taichung, Taiwan
The Catholic University of Korea
RECRUITING

Seoul, South Korea
The Ohio State University Comprehensive Cancer Center
RECRUITING

Columbus, Ohio, 43210, United States
The Royal Marsden NHS Foundation Trust
RECRUITING

Sutton, United Kingdom
Tohoku University Hospital
RECRUITING

Miyagi, 980-8574, Japan
Tokai University Hospital
RECRUITING

Isehara-shi, Kanagawa, 259-1193, Japan
Tokyo Metropolitan Komagome Hospital
NOT_YET_RECRUITING

Bunkyō City, 113-8677, Japan
Tom Baker Cancer Center
RECRUITING

Calgary, Alberta, T2N 5G2, Canada
TriStar Centennial Medical Center
RECRUITING

Nashville, Tennessee, 37203, United States
Tufts University
WITHDRAWN

Boston, Massachusetts, 02111, United States
UNC Hospital
RECRUITING

Chapel Hill, North Carolina, 27514, United States
UZ Ghent
RECRUITING

Ghent, 9000, Belgium
Universita' Degli Studi Di Torino
RECRUITING

Turin, Italy
Universitaetsspital Zuerich - Haematology
NOT_YET_RECRUITING

Zurich, 8091, Switzerland
University College London Hospitals NHS Foundation Trust
RECRUITING

London, United Kingdom
University Hospital Basel
NOT_YET_RECRUITING

Basel, 4031, Switzerland
University Hospital Bern Inselspital
NOT_YET_RECRUITING

Bern, 3010, Switzerland
University Hospitals Leuven
RECRUITING

Leuven, Belgium
University Hospitals of Birmingham Centre for Clinical Hematology
RECRUITING

Birmingham, United Kingdom
University Hospitals of North Midlands NHS Foundation Trust
NOT_YET_RECRUITING

Stoke-on-Trent, United Kingdom
University of Alberta
RECRUITING

Edmonton, T6G 2R3, Canada
University of Fukui Hospital
RECRUITING

Yoshida-gun, Fukui, 910-1193, Japan
University of Maryland
RECRUITING

Baltimore, Maryland, 21201, United States
University of Miami
RECRUITING

Miami, Florida, 33136, United States
University of Virginia
RECRUITING

Charlottesville, Virginia, 22908, United States
Università degli Studi di Perugia
RECRUITING

Perugia, Italy
Virginia Cancer Specialists
RECRUITING

Fairfax, Virginia, 22031, United States
Virginia Oncology Associates
RECRUITING

Norfolk, Virginia, 23502, United States
ZNA Cadix
RECRUITING

Antwerp, Belgium

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