Actively Recruiting
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)
Led by Sumitomo Pharma America, Inc. · Updated on 2026-03-24
606
Participants Needed
104
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating Enzomenib (DSP-5336), an oral drug, in patients with various types of acute leukemia, including relapsed or refractory acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and acute leukemia of ambiguous lineage. The study also includes patients with high-risk myelodysplastic syndromes (MDS) and relapsed multiple myeloma (MM) in selected sites. This phase 1/2 trial aims to assess the safety, pharmacokinetics, pharmacodynamics, and clinical activity of DSP-5336 alone or combined with standard AML treatments, particularly in patients with specific genetic mutations like MLL rearrangement or NPM1 mutation. The study involves dose escalation and dose expansion of DSP-5336 administered orally. Participants may receive DSP-5336 alone or combined with standard AML regimens such as venetoclax plus azacitidine or intensive chemotherapy with cytarabine and daunorubicin (7+3). Different study arms include patients with or without certain medications like CYP3A4 inhibitor azoles, and those with specific genetic profiles. The trial evaluates recommended phase 2 doses for various patient groups and combination treatments. Participants will undergo assessments including monitoring for adverse events within 30 days after the last dose and evaluation of clinical responses approximately six months after treatment begins. Researchers will collect blood and bone marrow samples for genomic analysis and track drug levels in the body. Safety labs, ECGs, physical exams, and patient questionnaires will be performed throughout the study. The trial includes long-term follow-up of overall survival up to two years after treatment ends, with visits and tests scheduled to monitor health and treatment effects.
CONDITIONS
Brief Title
A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 12 years or older (with specific weight criteria for some groups)
- Diagnosis of relapsed or refractory AML, ALL, or acute leukemia of ambiguous lineage per WHO 2022 criteria
- For selected sites, diagnosis of MDS with at least 5% bone marrow blasts or multiple myeloma meeting specified treatment history
- Specific genetic markers required for some patient groups (e.g., MLL rearrangement, NPM1 mutation, FLT3 mutations)
- Eastern Cooperative Oncology Group performance status of 0 to 2
- Adequate organ function and laboratory values as defined in the protocol
- Negative pregnancy test for females of childbearing potential and use of effective contraception for all participants of childbearing potential
- Willingness to attend all required study visits
- Bone marrow or suitable tissue available for genomic analysis
You will not qualify if you...
- Left ventricular ejection fraction below 50%
- Diagnosis of acute promyelocytic leukemia
- Recent use of systemic calcineurin inhibitors (within 2 to 4 weeks depending on the drug)
- Clinically significant abnormal ECG findings including prolonged QTc interval
- Active, uncontrolled infections requiring intravenous therapy
- Use of certain CYP3A4/5 inhibitors or inducers not permitted unless switched prior to study entry
- Major surgery within 28 days before study drug
- Active central nervous system leukemia
- Recent stem cell transplant, CAR-T, or modified T-cell therapy within specified timeframes
- Ongoing graft-versus-host disease requiring systemic treatment
- Recent use of antineoplastic or investigational treatments within 7 days or 5 half-lives
- Significant concurrent medical conditions including severe heart or lung disease
- Active infections with HIV, hepatitis B or C
- Conditions limiting oral drug intake or absorption
- Cognitive or psychological impairments limiting compliance
- Pregnancy, breastfeeding, or planning pregnancy
- History of interstitial lung disease or Torsades de Pointes
- Plasma cell leukemia for MM patients
- Known intolerance or hypersensitivity to study drugs
- Recent live vaccine (within 30 days) for specific groups in the UK study arm
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 4 months for dose escalation and expansion phases
Participants receive oral DSP-5336 (Enzomenib) alone or in combination with other drugs including venetoclax, azacitidine, gilteritinib, or intensive chemotherapy regimens depending on their specific trial arm and disease status.
Regular visits during treatment for drug administration and monitoring
Duration - Up to 2 years after the end of treatment
Participants are monitored for safety, clinical response, and survival outcomes after treatment completion.
Approximately 6 months to 2 years of follow-up visits
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
Research Team
M
Matt Hitron, MD
T
Tomoko Kuwabara
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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