Actively Recruiting

Phase 1
Phase 2
Age: 12Years +
All Genders
NCT04988555

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

Led by Sumitomo Pharma America, Inc. · Updated on 2026-03-24

606

Participants Needed

104

Research Sites

304 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

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

Who Can Participate

Age: 12Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with relapsed or refractory AML, ALL, or acute leukemia of ambiguous lineage per WHO 2022, or selected sites with high-risk relapsed/refractory MDS or relapsed MM
  • Patients with MDS must have bone marrow blasts ≥5% and relapsed or refractory disease after at least 2 cycles of hypomethylating agent treatment
  • MM patients must have progressed after at least 3 prior regimens including proteasome inhibitor, immunomodulatory drug, and anti-CD38 antibody
  • AML patients in venetoclax and azacitidine cohort must have MLL rearrangement or NPM1 mutation
  • AML patients in gilteritinib cohort must have MLL rearrangement or NPM1 mutation plus specific FLT3 mutations
  • Newly diagnosed AML patients must have MLL rearrangement or NPM1 mutation and no prior AML treatment except hydroxyurea
  • Patients aged ≥18 years; in some sites, patients ≥12 years and ≥40 kg may enroll for DSP-5336 monotherapy
  • ECOG performance status ≤2
  • White blood cell count below 30,000/μL for monotherapy or below 25,000/μL for combination arms at enrollment
  • Adequate kidney and liver function per protocol
  • Prior toxicities resolved to grade 1 or less except alopecia or neuropathy
  • Negative pregnancy test for females of childbearing potential and use of contraception by all men and women of childbearing potential
  • Bone marrow material available for genomic analysis
  • Willing to attend study visits and have an estimated life expectancy of at least 3 months
Not Eligible

You will not qualify if you...

  • Left ventricular ejection fraction less than 50%
  • Diagnosis of acute promyelocytic leukemia
  • Use of systemic calcineurin inhibitors within 2 weeks before first dose
  • Clinically significant abnormal ECGs including prolonged QT interval
  • Active uncontrolled infection requiring IV therapy
  • Use of strong CYP3A4/5 inhibitors or inducers like ketoconazole or itraconazole
  • Major surgery within 28 days before first dose
  • Active central nervous system leukemia
  • Hematopoietic stem cell transplant or CAR-T therapy within 60 days (6 months in UK)
  • Donor lymphocyte infusion within 28 days or active graft-versus-host disease needing treatment
  • Recent antineoplastic or investigational treatments within 7 days or 5 half-lives
  • Significant concurrent medical conditions including severe heart, lung, or metabolic diseases
  • Active HIV, hepatitis B or C infection
  • Severe swallowing or absorption problems
  • Cognitive or psychological conditions preventing study compliance
  • Pregnant or breastfeeding women or planning pregnancy
  • History of interstitial lung disease or significant drug-induced lung disease
  • History of Torsades de Pointes arrhythmia
  • Use of systemic calcineurin inhibitors within 4 weeks before first dose
  • Plasma cell leukemia for MM patients
  • Prior FLT3 inhibitor use in relapsed refractory setting for gilteritinib cohort
  • Known hypersensitivity to study drugs
  • Recent live vaccine within 30 days before first dose for DSP-5336 plus venetoclax/azacitidine arm in UK

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 104 locations

1

Hoag Family Cancer Center

Newport Beach, California, United States, 92663

Actively Recruiting

2

Stanford University

Palo Alto, California, United States, 94304

Not Yet Recruiting

3

Colorado Blood Cancer Institute

Denver, Colorado, United States, 80218

Actively Recruiting

4

Georgetown Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, United States, 20007

Not Yet Recruiting

5

University of Miami

Miami, Florida, United States, 33136

Actively Recruiting

6

Miami Cancer Institute

Miami, Florida, United States, 33176

Not Yet Recruiting

7

Moffitt Cancer Center

Tampa, Florida, United States, 33612

Not Yet Recruiting

8

Northwestern

Chicago, Illinois, United States, 60611

Actively Recruiting

9

Sibley Memorial Hospital

Baltimore, Maryland, United States, 20016

Actively Recruiting

10

University of Maryland

Baltimore, Maryland, United States, 21201

Actively Recruiting

11

Johns Hopkins Main Center

Baltimore, Maryland, United States, 21287

Actively Recruiting

12

Tufts University

Boston, Massachusetts, United States, 02111

Withdrawn

13

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Actively Recruiting

14

Atlantic Health

Morristown, New Jersey, United States, 07960

Actively Recruiting

15

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, United States, 08901

Actively Recruiting

16

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States, 14203

Actively Recruiting

17

Mount Sinai Hospital

New York, New York, United States, 10029

Completed

18

Columbia University

New York, New York, United States, 10032

Completed

19

UNC Hospital

Chapel Hill, North Carolina, United States, 27514

Actively Recruiting

20

Duke University

Durham, North Carolina, United States, 27705

Actively Recruiting

21

Atrium Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

22

The Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States, 43210

Actively Recruiting

23

Oncology Associates of Oregon

Eugene, Oregon, United States, 97401

Actively Recruiting

24

Sidney Kimmel Comprehensive Cancer Center

Philadelphia, Pennsylvania, United States, 19107

Actively Recruiting

25

Allegheny Health Network

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

26

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Actively Recruiting

27

TriStar Centennial Medical Center

Nashville, Tennessee, United States, 37203

Actively Recruiting

28

MDACC

Houston, Texas, United States, 77030

Actively Recruiting

29

Huntsman Cancer Institute

Salt Lake City, Utah, United States, 84112

Actively Recruiting

30

Intermountain Healthcare

Salt Lake City, Utah, United States, 84143

Actively Recruiting

31

University of Virginia

Charlottesville, Virginia, United States, 22908

Actively Recruiting

32

Virginia Cancer Specialists

Fairfax, Virginia, United States, 22031

Actively Recruiting

33

Virginia Oncology Associates

Norfolk, Virginia, United States, 23502

Actively Recruiting

34

ZNA Cadix

Antwerp, Belgium

Actively Recruiting

35

UZ Ghent

Ghent, Belgium, 9000

Actively Recruiting

36

University Hospitals Leuven

Leuven, Belgium

Actively Recruiting

37

AZ Delta

Roeselare, Belgium

Actively Recruiting

38

Tom Baker Cancer Center

Calgary, Alberta, Canada, T2N 5G2

Actively Recruiting

39

University of Alberta

Edmonton, Canada, T6G 2R3

Actively Recruiting

40

Centre Hospitalier Universitaire d'Angers

Angers, France

Actively Recruiting

41

Hopital Avicenne

Bobigny, France, 93000

Not Yet Recruiting

42

Centre Hospitalier Le Mans

Le Mans, France

Actively Recruiting

43

Hopital Claude Huriez

Lille, France, 59037

Not Yet Recruiting

44

Centre Hospitalier Universitaire de Limoges

Limoges, France

Actively Recruiting

45

Hospices Civils de Lyon

Lyon, France

Actively Recruiting

46

Institut Paoli-Calmettes

Marseille, France

Actively Recruiting

47

CHU de Nantes

Nantes, France, 44093

Not Yet Recruiting

48

CHU de Nice - Hôpital l'Archet 1

Nice, France

Actively Recruiting

49

Hopital Saint-Louis

Paris, France

Actively Recruiting

50

CHU Bordeaux

Talence, France, 33000

Not Yet Recruiting

51

Institut Gustave Roussy

Villejuif, France, 94800

Not Yet Recruiting

52

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

Bologna, Italy

Actively Recruiting

53

Ospedale di Busto Arsizio

Busto Arsizio, Italy, 21052

Not Yet Recruiting

54

Ospedale Policlinico San Martino, IRCCS

Genoa, Italy

Actively Recruiting

55

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

Meldola, Italy

Actively Recruiting

56

Istituto Oncologico Veneto (IOV), IRCCS

Padua, Italy

Not Yet Recruiting

57

Università degli Studi di Perugia

Perugia, Italy

Actively Recruiting

58

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

Ravenna, Italy, 48121

Not Yet Recruiting

59

PU A. Gemelli, Università Cattolica del Sacro Cuore

Rome, Italy

Actively Recruiting

60

Universita' Degli Studi Di Torino

Turin, Italy

Actively Recruiting

61

National Cancer Center Hospital East

Kashiwa-shi, Chiba, Japan, 277-8577

Actively Recruiting

62

University of Fukui Hospital

Yoshida-gun, Fukui, Japan, 910-1193

Actively Recruiting

63

Fukushima Medical University Hospital

Fukushima, Fukushima, Japan, 960-1295

Actively Recruiting

64

Tokai University Hospital

Isehara-shi, Kanagawa, Japan, 259-1193

Actively Recruiting

65

Nagasaki University Hospital

Nagasaki, Nagasaki, Japan, 852-8501

Actively Recruiting

66

Nippon Medical School Hospital

Bunkyo-ku, Tokyo, Japan, 113-8603

Actively Recruiting

67

Tokyo Metropolitan Komagome Hospital

Bunkyō City, Japan, 113-8677

Not Yet Recruiting

68

Kyushu University Hospital

Fukuoka, Japan, 812-8582

Actively Recruiting

69

Hokkaido University Hospital

Hokkaido, Japan, 060-8648

Not Yet Recruiting

70

Tohoku University Hospital

Miyagi, Japan, 980-8574

Actively Recruiting

71

Okayama University Hospital

Okayama, Japan, 700-8558

Actively Recruiting

72

Osaka University Hospital

Osaka, Japan, 565-0871

Actively Recruiting

73

National University Cancer Institute

Singapore, Singapore, 119074

Actively Recruiting

74

Chonnam National University Hwasun Hospital

Hwasun, South Korea

Actively Recruiting

75

Samsung Medical Center

Seoul, South Korea

Actively Recruiting

76

Seoul National University Hospital

Seoul, South Korea

Actively Recruiting

77

The Catholic University of Korea

Seoul, South Korea

Actively Recruiting

78

Hospital General Universitario De Albacete

Albacete, Spain

Actively Recruiting

79

Hospital Universitari Vall D'Hebron

Barcelona, Spain

Actively Recruiting

80

Institut Catala d'Oncologia

Barcelona, Spain

Actively Recruiting

81

Hospital San Pedro de Alcantara

Cáceres, Spain

Actively Recruiting

82

Hospital Universitario De Gran Canaria Dr. Negrin

Las Palmas, Spain

Actively Recruiting

83

MD Anderson Cancer Center

Madrid, Spain

Actively Recruiting

84

Hospital Universitario Central De Asturias

Oviedo, Spain

Actively Recruiting

85

Hospital Universitario de Salamanca

Salamanca, Spain

Actively Recruiting

86

Fundacion Instituto de Investigacion Marques de Valdecilla

Santander, Spain

Actively Recruiting

87

Complexo Hospitalario Universitario De Santiago

Santiago de Compostela, Spain

Actively Recruiting

88

Hospital Universitario y Politecnico La Fe

Valencia, Spain

Actively Recruiting

89

University Hospital Basel

Basel, Switzerland, 4031

Not Yet Recruiting

90

University Hospital Bern Inselspital

Bern, Switzerland, 3010

Not Yet Recruiting

91

Universitaetsspital Zuerich - Haematology

Zurich, Switzerland, 8091

Not Yet Recruiting

92

Taichung Veterans General Hospital

Taichung, Taiwan

Actively Recruiting

93

National Cheng Kung University Hospital

Tainan, Taiwan

Actively Recruiting

94

National Taiwan University Hospital

Taipei, Taiwan

Actively Recruiting

95

University Hospitals of Birmingham Centre for Clinical Hematology

Birmingham, United Kingdom

Actively Recruiting

96

Bristol Hematology & Oncology Centre

Bristol, United Kingdom

Actively Recruiting

97

NHS Lothian Western General

Edinburgh, United Kingdom, EH4 2XU

Not Yet Recruiting

98

King's College Hospital

London, United Kingdom

Actively Recruiting

99

Sarah Cannon Research Institute

London, United Kingdom

Not Yet Recruiting

100

University College London Hospitals NHS Foundation Trust

London, United Kingdom

Actively Recruiting

101

Christie Hospital NHS Foundation Trust

Manchester, United Kingdom

Actively Recruiting

102

Churchill Hospital Oxford

Oxford, United Kingdom

Not Yet Recruiting

103

University Hospitals of North Midlands NHS Foundation Trust

Stoke-on-Trent, United Kingdom

Not Yet Recruiting

104

The Royal Marsden NHS Foundation Trust

Sutton, United Kingdom

Actively Recruiting

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Research Team

M

Matt Hitron, MD

CONTACT

T

Tomoko Kuwabara

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

14

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