AzTrials
AzTrials

Leukemia, Myeloid, AcutePhase 1/2NCT04988555

A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)

A Phase 1/2 study testing Enzomenib, azoles and Venetoclax in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Sumitomo Pharma America, Inc. at 104 sites in 12 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

A phase 1/2 dose escalation / dose expansion study of Enzomenib (DSP-5336) in patients with acute leukemia. Read more about this study

Who is it for?
Ages 12 years and older
What is being tested?
Enzomenib, azoles and Venetoclax
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
606 participants
Where
104 sitesin 12 countries
Study dates
28 Feb 2022to 31 Dec 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • For patients in Phase I:
  • 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):
  • Patients with MDS must have bone marrow blasts ≥ 5%
  • 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):
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  • 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
  • Have measurable disease as defined in the protocol
  • 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):
  • Have MLLr or NPM1m.
  • For patients with relapsed/refractory AML in the gilteritinib combination cohort (in countries and sites where permitted):
  • 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:
  • Must have ≥5% blasts in bone marrow by morphologic assessment
  • Must not have received prior treatment with a menin inhibitor
  • For patients with newly diagnosed AML:
  • 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)
  • Must not have received treatment for AML with the exception of hydroxyurea for control of white blood cell counts.
  • For patients in Phase 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.
  • 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:
  • 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.
  • Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
  • 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)
  • Clearance of creatinine level ≥ 50 ml/min, assessed by the CPK-EPI formula (2021 version and Cystatin C not required)
  • Total bilirubin ≤1.5 the upper limit of normal (ULN) (or ≤2.0 ULN for patients with known Gilbert's syndrome)
  • Aspartate aminotransferase (AST) ≤3.0 times ULN
  • Alanine aminotransferase (ALT) ≤3.0 times ULN
  • Any prior treatment-related toxicities resolved to Grade ≤1 prior to enrollment, with the exception of Grade ≤2 alopecia or neuropathy
  • Be willing to attend study visits as required by the protocol
  • Have an estimated life expectancy ≥3 months, based on the investigator's assessment
  • 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.
  • 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.
  • 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.

Who cannot join

  • Has a left ventricular ejection fraction (LVEF) <50%, as determined by ECHO
  • Histological diagnosis of acute promyelocytic leukemia
  • Received systemic calcineurin inhibitors within 2 weeks prior to the first dose of DSP 5336
  • 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.
  • 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.
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  • 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.
  • Had major surgery within 28 days prior to the first dose of DSP-5336
  • Has active central nervous system leukemia (prophylactic intrathecal chemotherapy is allowed).
  • 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.
  • 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
  • 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
  • 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
  • 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.
  • 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
  • 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
  • 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
  • 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.
  • Have a history of Torsades de Pointes
  • Received systemic calcineurin inhibitors within 4 weeks prior to the first dose of DSP-5336
  • Have plasma cell leukemia (>2.0 x 109 /L plasma cells in blood by standard differential) (for patients with MM)
  • 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)
  • Have a known intolerance of hypersensitivity reaction to components of the investigational medicinal product
  • 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

Trial locations and contacts

104 sites in 12 countries. Contact the study team or a site near you to ask about taking part.

Matt Hitron, MD contact

508-481-6700

matthew.hitron@us.sumitomo-pharma.com

Tomoko Kuwabara contact

tomoko.kuwabara@us.sumitomo-pharma.com

104 sites in 12 countries

United States33 sites, 30 recruiting

  • Sibley Memorial Hospital

    Baltimore, Maryland, 20016

    Mark Levis, MD, levisma@jhmi.edu

    Mark Levis

    Recruiting

  • University of Maryland

    Baltimore, Maryland, 21201

    Maria Baer, MD, mbaer@umm.edu

    Maria Baer, MD

    Recruiting

  • Johns Hopkins Main Center

    Baltimore, Maryland, 21287

    Mark Levis, MD, levisma@jhmi.edu

    Mark Levis, MD

    Recruiting

  • Tufts University

    Boston, Massachusetts, 02111

    Withdrawn

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Andrew Brunner, MD, abrunner@mgb.org

    Andrew Brunner, MD

    Recruiting

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203

    Eunice Wang, MD, Eunice.Wang@RoswellPark.org

    Eunice Wang, MD

    Recruiting

  • UNC Hospital

    Chapel Hill, North Carolina, 27514

    Joshua Zeidner, MD, joshua_zeidner@med.unc.edu

    Joshua Zeidner, MD

    Recruiting

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

    Praneeth Baratam, MD, baratamp@musc.edu

    Praneeth Baratam, MD

    Recruiting

  • University of Virginia

    Charlottesville, Virginia, 22908

    Michael Keng, MD, mk2pv@uvahealth.org

    Michael Keng, MD

    Recruiting

  • Northwestern

    Chicago, Illinois, 60611

    Yasmin Abaza, MD, yasmin.abaza@nm.org

    Yasmin Abaza, MD

    Recruiting

  • The Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

    Kristen Browning, MD, Kristen.Browning@osumc.edu

    Kristen Browning, MD

    Recruiting

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

    Alireza Eghtedar, MD, 720-754-4800, alireza.eghtedar@healthonecares.com

    Alireza Eghtedar, MD

    Recruiting

  • Duke University

    Durham, North Carolina, 27705

    Harry Erba, MD, 919-684-8964, harry.erba@duke.edu

    Harry Erba, MD

    Recruiting

  • Oncology Associates of Oregon

    Eugene, Oregon, 97401

    Luke Fletcher, MD, luke.fletcher@usoncology.com

    Luke Fletcher, MD

    Recruiting

  • Virginia Cancer Specialists

    Fairfax, Virginia, 22031

    Mitul Gandhi, MD, mitul.gandhi@usoncology.com

    Mitul Gandhi, MD

    Recruiting

  • MDACC

    Houston, Texas, 77030

    Naval Daver, MD, 7137944392, NDaver@mdanderson.org

    Naval Daver, MD

    Recruiting

  • University of Miami

    Miami, Florida, 33136

    Justin Watts, MD, 305-243-8986, jxw401@miami.edu

    Justin Watts, MD

    Recruiting

  • Miami Cancer Institute

    Miami, Florida, 33176

    SMPA Investigative Site, 508-481-6700

    Not yet recruiting

  • Atlantic Health

    Morristown, New Jersey, 07960

    Mohamad Cherry, MD, mohamad.cherry@atlantichealth.org

    Mohamad Cherry, MD

    Recruiting

  • TriStar Centennial Medical Center

    Nashville, Tennessee, 37203

    Stephen Strickland, MD, Stephen.Strickland@hcahealthcare.com

    Stephen Strickland, MD

    Recruiting

  • Rutgers Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08901

    Neil Palmisiano, MD, np938@cinj.rutgers.edu

    Neil Palmisiano, MD

    Recruiting

  • Mount Sinai Hospital

    New York, New York, 10029

    Completed

  • Columbia University

    New York, New York, 10032

    Completed

  • Hoag Family Cancer Center

    Newport Beach, California, 92663

    Benjamin Goldenson, MD, benjamin.goldenson@hoag.org

    Benjamin Goldenson, MD

    Recruiting

  • Virginia Oncology Associates

    Norfolk, Virginia, 23502

    Celeste Bremer, MD, celesteann.bremer@usoncology.com

    Celeste Bremer, MD

    Recruiting

  • Stanford University

    Palo Alto, California, 94304

    Gabriel Mannis, MD, gmannis@stanford.edu

    Not yet recruiting

  • Sidney Kimmel Comprehensive Cancer Center

    Philadelphia, Pennsylvania, 19107

    Gina Keiffer, MD, gina.keiffer@jefferson.edu

    Gina Keiffer, MD

    Recruiting

  • Allegheny Health Network

    Pittsburgh, Pennsylvania, 15224

    Salman Fazal, MD, salman.fazal@ahn.org

    Salman Fazal, MD

    Recruiting

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112

    Paul Shami, MD, paul.shami@utah.edu

    Paul Shami, MD

    Recruiting

  • Intermountain Healthcare

    Salt Lake City, Utah, 84143

    Bradley Hunter, MD, brad.hunter@imail.org

    Bradley Hunter, MD

    Recruiting

  • Moffitt Cancer Center

    Tampa, Florida, 33612

    SMPA Investigative Site, 508-481-6700

    Not yet recruiting

  • Georgetown Lombardi Comprehensive Cancer Center

    Washington D.C., District of Columbia, 20007

    SMPA Investigative Site, 508-481-6700

    Not yet recruiting

  • Atrium Wake Forest Baptist Medical Center

    Winston-Salem, North Carolina, 27157

    Timothy Pardee, MD, tspardee@wakehealth.edu

    Timothy Pardee, MD

    Recruiting

Other trials in United States

France12 sites, 12 recruiting

  • Centre Hospitalier Universitaire d'Angers

    Angers

    Corentin Orvain, MD, corentin.orvain@chu-angers.fr

    Corentin Orvain, MD

    Recruiting

  • Hopital Avicenne

    Bobigny, 93000

    Ramy Rahme, MD, ramy.rahme@aphp.fr

    Not yet recruiting

  • Centre Hospitalier Le Mans

    Le Mans

    Kamel Laribi, MD, klaribi@ch-lemans.fr

    Recruiting

  • Hopital Claude Huriez

    Lille, 59037

    Celine Berthon, MD, celine.berthon@chu-lille.fr

    Not yet recruiting

  • Centre Hospitalier Universitaire de Limoges

    Limoges

    Pascal Turture, MD, Pascal.Turlure@chu-limoges.fr

    Recruiting

  • Hospices Civils de Lyon

    Lyon

    Mael Heiblig, MD, mael.heiblig@chu-lyon.fr

    Recruiting

  • Institut Paoli-Calmettes

    Marseille

    Sylvain Garciaz, MD, garciazs@ipc.unicancer.fr

    Sylvain Garciaz, MD

    Recruiting

  • CHU de Nantes

    Nantes, 44093

    Pierre Peterlin, MD, Pierre.PETERLIN@chu-nantes.fr

    Not yet recruiting

  • CHU de Nice - Hôpital l'Archet 1

    Nice

    Thomas Cluzeau, MD, cluzeau.t@chu-nice.fr

    Thomas Cluzeau, MD

    Recruiting

  • Hopital Saint-Louis

    Paris

    Emmanuel Raffoux, MD, emmanuel.raffoux@aphp.fr

    Emmanuel Raffoux, MD

    Recruiting

  • CHU Bordeaux

    Talence, 33000

    Arnaud Pigneux, MD, Arnaud.pigneux@chu-bordeaux.fr

    Not yet recruiting

  • Institut Gustave Roussy

    Villejuif, 94800

    Stephane Debotton, MD, Stephane.DEBOTTON@gustaveroussy.fr

    Not yet recruiting

Other trials in France

Japan12 sites, 12 recruiting

  • Tokyo Metropolitan Komagome Hospital

    Bunkyō City, 113-8677

    Tomomi Toubai, MD, tomomi_tobai@tmhp.jp

    Not yet recruiting

  • Nippon Medical School Hospital

    Bunkyo-ku, Tokyo, 113-8603

    Hiroki Yamaguchi, MD, +81-3-3822-2131, y-hiroki@fd6.so-net.ne.jp

    Hiroki Yamaguchi, MD

    Recruiting

  • Kyushu University Hospital

    Fukuoka, 812-8582

    Takahiro Shima, MD, 81-92-641-1151, shima.takahiro.993@m.kyushu-u.ac.jp

    Takahiro Shima, MD

    Recruiting

  • Fukushima Medical University Hospital

    Fukushima, Fukushima, 960-1295

    Takayuki Ikezoe, +81-24-547-1111, ikezoet@fmu.ac.jp

    Takayuki Ikezoe, MD

    Recruiting

  • Hokkaido University Hospital

    Hokkaido, 060-8648

    Takanori Teshima, MD, +81-11-716-1161, teshima@med.hokudai.ac.jp

    Takanori Teshima, MD

    Not yet recruiting

  • Tokai University Hospital

    Isehara-shi, Kanagawa, 259-1193

    Yoshiaki Ogawa, +81-463-93-1121, yoshioga@is.icc.u-tokai.ac.jp

    Yoshiaki Ogawa, MD

    Recruiting

  • National Cancer Center Hospital East

    Kashiwa-shi, Chiba, 277-8577

    Junichiro Yuda, +81-4-7133-1111, jyuda@east.ncc.go.jp

    Junichiro Yuda, MD

    Recruiting

  • Tohoku University Hospital

    Miyagi, 980-8574

    Koichi Onodera, MD, +81-22-717-7165, koichi.onodera.d6@tohoku.ac.jp

    Hisayuki Yokoyama, MD

    Recruiting

  • Nagasaki University Hospital

    Nagasaki, Nagasaki, 852-8501

    Yasushi Miyazaki, +81-95-819-7200, y-miyaza@nagasaki-u.ac.jp

    Yasushi Miyazaki, MD

    Recruiting

  • Okayama University Hospital

    Okayama, 700-8558

    Noboru Asada, MD, +81-86-223-7151, nasada@okayama-u.ac.jp

    Noboru Asada, MD

    Recruiting

  • Osaka University Hospital

    Osaka, 565-0871

    Kentaro Fukushima, MD, +81-6-6879-5111, kfukushi@bldon.med.osaka-u.ac.jp

    Kentaro Fukushima, MD

    Recruiting

  • University of Fukui Hospital

    Yoshida-gun, Fukui, 910-1193

    Naoko Hosono, +81-776-61-3111, hosono@u-fukui.ac.jp

    Naoko Hosono, MD

    Recruiting

Other trials in Japan

Spain11 sites, 11 recruiting

  • Hospital General Universitario De Albacete

    Albacete

    Jesus Lorenzo Algarra, MD, jlalgarra@sescam.jccm.es

    Jesus Lorenzo Algarra, MD

    Recruiting

  • Hospital Universitari Vall D'Hebron

    Barcelona

    Olga Garcia, MD, osalamero@vhio.net

    Recruiting

  • Institut Catala d'Oncologia

    Barcelona

    Susana Vives Polo, MD, svives@iconcologia.net

    Susana Vives Polo, MD

    Recruiting

  • Hospital San Pedro de Alcantara

    Cáceres

    Juan Miguel Bergua Burgues, jmberguaburg@gmail.com

    Juan Miguel Bergua Burgues, MD

    Recruiting

  • Hospital Universitario De Gran Canaria Dr. Negrin

    Las Palmas

    Carlos Rodriguez Medina, MD, crodmedk@gobiernodecanarias.org

    Carlos Rodriguez Medina, MD

    Recruiting

  • MD Anderson Cancer Center

    Madrid

    Adolfo de la Fuente Burguera, MD, afuente@mdanderson.es

    Adolfo de la Fuente Burguera, MD

    Recruiting

  • Hospital Universitario Central De Asturias

    Oviedo

    Teresa Bernal del Castillo, MD, bernaldelcastillo@gmail.com

    Teresa Bernal del Castillo, MD

    Recruiting

  • Hospital Universitario de Salamanca

    Salamanca

    Maria-Belen Vidriales Vicente, MD, mbvidri@usal.es

    Maria-Belen Vidriales Vicente, MD

    Recruiting

  • Fundacion Instituto de Investigacion Marques de Valdecilla

    Santander

    Guillermo Sanchez, guillermo.martin@scsalud.es

    Recruiting

  • Complexo Hospitalario Universitario De Santiago

    Santiago de Compostela

    Manual Perez, MD, manualmateo.perez@usc.es

    Recruiting

  • Hospital Universitario y Politecnico La Fe

    Valencia

    Pau Montesinos, MD, montesinos_pau@gva.es

    Paul Montesinos, MD

    Recruiting

Other trials in Spain

United Kingdom10 sites, 10 recruiting

  • University Hospitals of Birmingham Centre for Clinical Hematology

    Birmingham

    Vidya Murthy, vidhya.murthy2@uhb.nhs.uk

    Recruiting

  • Bristol Hematology & Oncology Centre

    Bristol

    Katharine Hodby, MD, katharine.hodby@uhbw.nhs.uk

    Recruiting

  • NHS Lothian Western General

    Edinburgh, EH4 2XU

    Victoria Campbell, MD, victoria.campbell15@nhs.scot

    Not yet recruiting

  • King's College Hospital

    London

    Pramila Krishnamurthy, MD, pramila.krishnamurthy@kcl.ac.uk

    Recruiting

  • Sarah Cannon Research Institute

    London

    Richard Dillon, MD, richard.dillon@kcl.ac.uk

    Not yet recruiting

  • University College London Hospitals NHS Foundation Trust

    London

    Jenny O'nions, MD, jenny.o'nions@nhs.net

    Recruiting

  • Christie Hospital NHS Foundation Trust

    Manchester

    Emma Searle, MD, emma.searle9@nhs.net

    Recruiting

  • Churchill Hospital Oxford

    Oxford

    Connor Sweeney, connor.sweeney@hee.nhs.uk

    Not yet recruiting

  • University Hospitals of North Midlands NHS Foundation Trust

    Stoke-on-Trent

    Srinivas Pillai, MD, Srinivas.pillai@uhnm.nhs.uk

    Not yet recruiting

  • The Royal Marsden NHS Foundation Trust

    Sutton

    David Taussig, MD, david.taussig@rmh.mhs.uk

    Recruiting

Other trials in United Kingdom

Italy9 sites, 9 recruiting

  • IRCCS Azienda Ospedaliero-Universitaria Di Bologna, Policlinico Sant'Orsola

    Bologna

    Cristina Papayannidis, MD

    Cristina Papayannidis, MD

    Recruiting

  • Ospedale di Busto Arsizio

    Busto Arsizio, 21052

    Elisabetta Todisco, MD, elisabetta.todisco@asst-valleolona.it

    Not yet recruiting

  • Ospedale Policlinico San Martino, IRCCS

    Genoa

    Marica Laurino, MD, maurica.laurino@hsanmartino.it

    Recruiting

  • IRCCS istituto Romagnolo per lo studio dei tumori "Dino Amadori"

    Meldola

    Maria Giannini, MD, Maria.giannini@irst.emr.it

    Recruiting

  • Istituto Oncologico Veneto (IOV), IRCCS

    Padua

    Michele Gottardi, MD, michele.gottardi@iov.it

    Not yet recruiting

  • Università degli Studi di Perugia

    Perugia

    Maria Martelli, MD, maria.martelli@unipg.it

    Recruiting

  • Azienda USL della Romagna, Ospedale Santa Maria delle Croci di Ravenna

    Ravenna, 48121

    Giovanni Marconi, MD, giovanni.marconi2@unibo.it

    Not yet recruiting

  • PU A. Gemelli, Università Cattolica del Sacro Cuore

    Rome

    Patrizia Chiusolo, MD, Patrizia.Chiusolo@Unicatt.it

    Recruiting

  • Universita' Degli Studi Di Torino

    Turin

    Benedetto Bruno, MD, benedetto.bruno@unito.it

    Recruiting

Other trials in Italy

Belgium4 sites, 4 recruiting

  • ZNA Cadix

    Antwerp

    Dimitri Breems, MD, dimitri.breems@zna.be

    Recruiting

  • UZ Ghent

    Ghent, 9000

    Anke Delie, MD, anke.delie@uzgent.be

    Ine Moors, MD

    Recruiting

  • University Hospitals Leuven

    Leuven

    Johan Maertens, MD, johan.maertens@uzleuven.be

    Johan Maertens, MD

    Recruiting

  • AZ Delta

    Roeselare

    Dries Deeren, MD, dries.deeren@azdelta.be

    Dries Deeren, MD

    Recruiting

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South Korea4 sites, 4 recruiting

  • Chonnam National University Hwasun Hospital

    Hwasun

    Jae-Sook Ahn, MD, f0115@jnu.ac.kr

    Jae-Sook Ahn, MD

    Recruiting

  • Samsung Medical Center

    Seoul

    Dahee Jung, ddahee.jung@samsung.com

    Jun Jo Hang, MD

    Recruiting

  • Seoul National University Hospital

    Seoul

    Juhyun Lee, leejh8368@snuh.org

    Dong-Yeop Shin, MD

    Recruiting

  • The Catholic University of Korea

    Seoul

    HeeJe Kim, MD, cumckim@catholic.ac.kr

    HeeJe Kim, MD

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Switzerland3 sites, 3 recruiting

  • University Hospital Basel

    Basel, 4031

    Not yet recruiting

  • University Hospital Bern Inselspital

    Bern, 3010

    Not yet recruiting

  • Universitaetsspital Zuerich - Haematology

    Zurich, 8091

    Not yet recruiting

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Taiwan3 sites, 3 recruiting

  • Taichung Veterans General Hospital

    Taichung

    Huey-En Tzeng, MD, tzenghueyen@gmail.com

    Huey-En Tzeng, MD

    Recruiting

  • National Cheng Kung University Hospital

    Tainan

    Tsai-Yun Chen, MD, teresa@mail.ncku.edu.tw

    Tsai-Yun Chen, MD

    Recruiting

  • National Taiwan University Hospital

    Taipei

    Yi Hsuan Shao, bakeyshao248@gmail.com

    Shang-Ju Wu, MD

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Canada2 sites, 2 recruiting

  • Tom Baker Cancer Center

    Calgary, Alberta, T2N 5G2

    Lynn Savoie, MD, lynn.savoie@albertahealthservices.ca

    Lynn Savoie, MD

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  • University of Alberta

    Edmonton, T6G 2R3

    Joseph Brandwein, MD, jbrandwe@ualberta.ca

    Joseph Brandwein, MD

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Singapore1 site, 1 recruiting

  • National University Cancer Institute

    Singapore, 119074

    Melissa Ooi, MD, melissa_ooi@nuhs.edu.sg

    Melissa Ooi, MD

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About this study

Official title: A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia and Other Selected Hematologic Malignancies, With and Without Mixed Lineage Leukemia (MLL) Rearrangement or Nucleophosmin 1 (NPM1) Mutation (Horizen-1)

A phase 1/2 dose escalation / dose expansion study of Enzomenib (DSP-5336) in patients with acute leukemia.

Read the detailed description

DSP-5336-101 is a phase 1/2 open-label, dose escalation, dose expansion study in which the safety, PK, pharmacodynamics, and clinical activity of orally administered DSP-5336 will be evaluated in patients with relapsed or refractory AML, ALL, or acute leukemia of ambiguous lineage, and in selected sites and regions, in adult patients with high-risk relapsed or refractory MDS or relapsed MM.

Additionally, the safety and clinical activity of orally administered DSP-5336 will be evaluated in combination with Standard-of-Care (SOC) AML treatments including: (a) the SOC nonintensive regimen (venetoclax + azacitidine) or (b) the SOC intensive regimen (cytarabine + daunorubicin induction, 7+3) in patients with newly diagnosed AML who have MLLr or NPM1m.

Being tested
Enzomenib, azoles, Venetoclax, Gilteritinib, Azacitidine (AZA)
Keywords
Relapsed or refractory AML, MLLr, Menin, NPM1m, KMT2A, MDS, MM
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Multiple Myeloma, Myelodysplastic Syndromes, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EnzomenibDrug

    DSP-5336 orally

  • azolesDrug

    Posaconazole, Voriconazole, or Fluconazole

  • VenetoclaxDrug

    Venetoclax orally

  • GilteritinibDrug

    Gilteritinib orally

  • Azacitidine (AZA)Drug

    Azacitidine orally

  • Intensive chemotherapy with 7 + 3Drug

    chemotherapy

Study groups in NCT04988555
Study groupRoleWhat they receive
Phase 1 - Arm AExperimentalR/R acute leukemia without CYP3A4 inhibitor azoles
Receives: Drug: Enzomenib
Phase 1 - Arm BExperimentalR/R acute leukemia with CYP3A4 inhibitor azoles
Receives: Drug: Enzomenib; Drug: azoles
Phase 1 - Arm CExperimentalHigh-risk MDS after HMA
Receives: Drug: Enzomenib
Phase 1 - Arm DExperimentalPatients with refractory MM
Receives: Drug: Enzomenib
Phase 1 - Arm EExperimentalR/R AML with NPM1m or KMT2Ar
Receives: Drug: Enzomenib; Drug: Venetoclax; Drug: Azacitidine (AZA)
Phase 1 - Arm FExperimentalR/R AML with FLT3m + NPM1m or KMT2Ar
Receives: Drug: Enzomenib; Drug: Gilteritinib
Phase 2 - Arm GExperimentalR/R AML with MLLr
Receives: Drug: Enzomenib
Phase 2 - Arm HExperimentalR/R AML with NPM1m
Receives: Drug: Enzomenib
Phase 2 - Arm IExperimentalR/R ALL with MLLr
Receives: Drug: Enzomenib
Phase 2 - Arm JExperimentalR/R acute leukemia with MLLr
Receives: Drug: Enzomenib; Drug: azoles
Phase 2 - Arm KExperimentalR/R AML with NPM1m
Receives: Drug: Enzomenib; Drug: azoles
Phase 1 - Arm LExperimentalNewly diagnosed AML KMT2Ar FIT or UNFIT
Receives: Drug: Enzomenib; Drug: azoles; Drug: Venetoclax; Drug: Azacitidine (AZA)
Phase 1 - Arm MExperimentalNewly diagnosed AML NPM1m UNFIT only
Receives: Drug: Enzomenib; Drug: azoles; Drug: Venetoclax; Drug: Azacitidine (AZA)
Phase 1 - Arm NExperimentalNewly diagnosed AML with KMT2Ar or NPM1m
Receives: Drug: Enzomenib; Drug: azoles; Drug: Intensive chemotherapy with 7 + 3

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of patients with adverse events and serious adverse events in Phase 1

    Assessment of safety of DSP-5336 administered in participants with advanced hematologic malignancies by reporting of adverse events and serious adverse events in Phase 1

    Measured: 30 days from last dose

  2. Determination of Recommended Phase 2 Dose (RP2D)

    The RP2D is based on adverse events, pharmacokinetics, and clinical response

    Measured: Within 4 months from first dose

  3. Determination of Recommended Phase 2 Dose (RP2D) for patients with relapse and refractory AML who are enrolled into the combination venetoclax and azacitidine arm

    The RP2D is based on adverse events, pharmacokinetics, and clinical response

    Measured: Within 4 months from first dose

  4. Determination of Recommended Phase 2 Dose (RP2D) for patients with relapse and refractory AML who are enrolled into the gilteritinib arm

    The RP2D is based on assessment of Dose Limiting Toxicities

    Measured: Within 4 months from first dose

  5. Optimal dose of DSP-5336 (RP2D) for patients newly diagnosed with AML enrolled into the combination venetoclax and azacitidine arm

    The RP2D is based on adverse events, pharmacokinetics and clinical response

    Measured: Within 4 months from the first dose

  6. Determination of Recommended Phase 2 Dose (RP2D) for patients enrolled into the 7 + 3 arm

    The RP2D is based on assessment of Dose Limiting Toxicities

    Measured: Within 4 months from first dose

  7. Number of patients achieving complete response (CR) and complete response with partial hematologic recovery (CRh) in Phase 2

    Disease response defined by the FDA guidance and ELN2017

    Measured: Approximately 6 months after first dose

Secondary outcomes (13)

  1. The maximum observed concentration (Cmax) of of DSP-5336, venetoclax and gilteritinib

    Cmax of DSP-5336, venetoclax and gilteritinib are calculated from the individual concentration time curve

    Measured: Approximately 3 months after first dose

  2. Area under the plasma concentration vs. time curve (AUClast) of DSP-5336, venetoclax and gilteritinib

    The determination of AUClast using the linear/log trapezoidal rule

    Measured: Approximately 3 months after first dose

  3. T(1/2) of DSP-5336, venetoclax and gilteritinib

    Elimination half-life (t[1/2]) of DSP-5336, venetoclax and gilteritinib

    Measured: Approximately 3 months after first dose

  4. The maximum observed concentration (Cmax) of of DSP-5336 in the presence of high-fat meal

    Cmax of DSP-5336 is calculated from the individual concentration time curve

    Measured: Approximately 3 months after first dose

  5. Area under the plasma concentration vs. time curve (AUClast) of DSP-5336 in the presence of high-fat meal

    The determination of AUClast using the linear/log trapezoidal rule

    Measured: Approximately 3 months after first dose

  6. t(1/2) of of DSP-5336 in the presence of high fat meal

    Elimination half-life (t[1/2]) of DSP-5336

    Measured: Approximately 3 months after first dose

  7. Number of patients achieving complete response (CR) in the 7+3 arm in Phase 1

    Disease response defined by the ELN2017

    Measured: Approximately 6 months after first dose

  8. Number of patients achieving CRc, which means complete response (CR), complete response with partial hematologic recovery (CRh) or complete response with incomplete count recovery (CRi) in the 7+3 arm in Phase 1

    Disease response defined by the FDA guidance and ELN2017

    Measured: Approximately 6 months after first dose

  9. Number of patients with adverse events and serious adverse events in Phase 2

    Assessment of safety of DSP-5336 administered in patients with advanced hematologic malignancies by reporting of adverse events and serious adverse events in Phase 2

    Measured: 30 days from the last dose

  10. Number of patients achieving CRc, which means complete response (CR), complete response with partial hematologic recovery (CRh) or complete response with Incomplete count recovery (CRi) in Phase 2

    Disease response defined by the FDA guidance and ELN2027

    Measured: Approximately 6 months after first dose

  11. Number of patients achieving ORR, which means complete response (CR), complete response with Incomplete count recovery (CRi) or MFLS (Morphologic leukemia-free state) in Phase 2

    Disease response defined by ELN2027

    Measured: Approximately 6 months after first dose

  12. Event-Free Survival in Phase 2

    The time period from treatment initiation to hematologic relapse, progressive disease or death

    Measured: Approximately 6 months after first dose

  13. Overall Survival in Phase 2

    The time period from treatment initiation to death

    Measured: Two years after the end of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Aug 2021
  2. Study start28 Feb 2022
  3. Primary completion30 Jun 2027 (estimated)
  4. Study completion31 Dec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
Bayesian Regression Model for Phase 1 dose escalation
Masking
None (open label)
Enrolment
606 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04988555
Sponsor's protocol ID
DSP-5336-101
Status last verified
Mar 2026
First submitted
26 Jul 2021
Last updated
24 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Enzomenib, azoles and Venetoclax in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Sumitomo Pharma America, Inc. at 104 sites in 12 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 36 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 104 sites in United States, France, Japan, Spain, United Kingdom and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Matt Hitron, MD (508-481-6700, matthew.hitron@us.sumitomo-pharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients with adverse events and serious adverse events in Phase 1 (time frame: 30 days from last dose). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04988555.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2026.

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