Preclinical Development of Tuspetinib for the Treatment of Acute Myeloid Leukemia.
Himangshu Sonowal, William G Rice, Raphael Bejar...
https://pubmed.ncbi.nlm.nih.gov/39665627Actively Recruiting
Phase 1
Phase 2
Age: 18Years +
All Genders
ID03850574
Phase 1/2 Study of Tuspetinib (HM43239) Safety, Tolerability, and Pharmacokinetics in Adults with Relapsed or Refractory Acute Myeloid Leukemia
Led by Aptose Biosciences Inc. · Updated on 2025-08-26
240
Participants Needed
34
Research Sites
21 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety, tolerability, and pharmacokinetics of tuspetinib (HM43239) in adults with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that has relapsed or is refractory after at least one prior treatment, as well as in newly diagnosed AML patients who cannot receive intensive chemotherapy. This phase 1/2 open-label study aims to find a safe and potentially effective dose of tuspetinib for future research. Tuspetinib will be given either alone or combined with other drugs such as venetoclax or venetoclax plus azacitidine, depending on the study part. The study includes several parts: Part A dose escalation with up to 6 dose levels of tuspetinib alone, Part B dose exploration at up to 4 dose levels, Part C dose expansion testing tuspetinib alone or with venetoclax at specified doses, and Part D dose exploration combining tuspetinib with venetoclax and azacitidine for newly diagnosed AML patients ineligible for intensive chemotherapy. Participants will be closely monitored through blood tests, physical exams, and assessments of drug levels in the blood during at least one 28-day treatment cycle and up to four years for long-term safety and response. Researchers will measure adverse events, determine the maximum tolerated dose, and evaluate treatment responses such as remission rates and survival outcomes. The study involves multiple centers and aims to provide detailed information on how tuspetinib works and is tolerated alone and in combination treatments.
CONDITIONS
Brief Title
Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Diagnosed with acute myeloid leukemia (AML), myelodysplastic syndrome with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) according to WHO 2016 criteria
- Relapsed or refractory disease after at least one prior therapy, or newly diagnosed AML ineligible for intensive chemotherapy (Part D)
- Eastern Cooperative Oncology Group (ECOG) performance status of 2 or less
- Specific laboratory test criteria met for liver and kidney function as detailed in study
- Suitable for oral study drug administration with life expectancy of at least 3 months
- Female participants must be non-childbearing or use effective contraception; male participants and partners must use effective contraception
- Agreement not to participate in another interventional study during treatment
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia (APL) or BCR-ABL-positive leukemia
- Active malignancy other than AML, MDS-IB2, or CMML
- Persistent non-hematological toxicities grade 2 or higher from prior treatments
- Recent hematopoietic stem cell transplant (HSCT) within 2 months or related complications
- Meningeal or central nervous system involvement with leukemia
- Disseminated intravascular coagulation abnormality
- Major surgery or radiation therapy within 4 weeks prior to first study dose
- Congestive heart failure NYHA class 3 or 4 or significant cardiac abnormalities
- Active infections including COVID-19, HIV, hepatitis B or C
- Other medical conditions or history that make participation unsuitable as judged by investigator
- White blood cell count above specified threshold unless controlled
- Prior treatments disallowed in Part D such as certain chemotherapies, CAR-T, or experimental therapies
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive tuspetinib as a single agent or in combination with venetoclax and azacitidine to assess safety, tolerability, and pharmacokinetics.
Weekly visits for up to 4 years
Follow-up
Duration - Up to 4 years
Participants are monitored for long-term safety and treatment outcomes after completing or discontinuing treatment.
Periodic visits depending on participant status
Trial Site Locations
Total: 34 locations
1
The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010
Active, Not Recruiting
3
University of California Irvine
Irvine, California, United States, 92697
Actively Recruiting
4
UCSD Moores Cancer Center
La Jolla, California, United States, 92093
Active, Not Recruiting
5
USC/Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
Actively Recruiting
6
Stanford Cancer Center
Palo Alto, California, United States, 94304
Actively Recruiting
7
University of California, Davis
Sacramento, California, United States, 95817
Actively Recruiting
8
Yale University
New Haven, Connecticut, United States, 06520
Actively Recruiting
9
University of Miami - Miller School of Medicine
Miami, Florida, United States, 33136
Actively Recruiting
10
Emory University
Atlanta, Georgia, United States, 30322
Active, Not Recruiting
11
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Active, Not Recruiting
12
Duke University Medical Center
Durham, North Carolina, United States, 27705
Actively Recruiting
13
Cleveland Clinic - Taussig Cancer Center
Cleveland, Ohio, United States, 44106
Active, Not Recruiting
14
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States, 43210
Actively Recruiting
15
MD Anderson Cancer Center
Huston, Texas, United States, 77030
Actively Recruiting
16
Border Medical Oncology
Albury, New South Wales, Australia, 2640
Active, Not Recruiting
17
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia, 4006
Active, Not Recruiting
18
Townsville University Hospital
Townsville, Queensland, Australia, 4812
Active, Not Recruiting
19
St Vincent's Hospital Melbourne
Fitzroy, Victoria, Australia, 3065
Active, Not Recruiting
20
Sir Charles Gairdner Hospital
Nedlands, Western Australia, Australia, 6009
Active, Not Recruiting
21
Universitätsklinikum Leipzig
Leipzig, Saxony, Germany, 04103
Active, Not Recruiting
22
Charité Universitätsmedizin Berlin
Berlin, State of Berlin, Germany, 13353
Active, Not Recruiting
23
Auckland City Hospital
Grafton, Auckland, New Zealand, 1023
Active, Not Recruiting
24
Seoul National University Hospital
Seoul, Seoul, South Korea, 03080
Completed
25
Asan Medical Center
Seoul, Seoul, South Korea, 05505
Active, Not Recruiting
26
Samsung Medical Center
Seoul, Seoul, South Korea, 06351
Active, Not Recruiting
27
Kyungpook National University Hospital
Daegu, South Korea, 41944
Active, Not Recruiting
28
Pusan National University Hospital
Pusan, South Korea, 49241
Active, Not Recruiting
29
Seoul National University Bundang Hospital
Seongnam, South Korea, 13620
Active, Not Recruiting
30
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona, Spain, 08035
Active, Not Recruiting
31
Hospital Quirón Madrid
Pozuelo de Alarcón, Madrid, Spain, 28223
Active, Not Recruiting
32
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, Spain, 33011
Active, Not Recruiting
33
Hospital Clinico Universitario de Valencia
Valencia, Valencia, Spain, 46010
Active, Not Recruiting
34
Hospital Universitari i Politècnic La Fe
Valencia, Valencia, Spain, 46026
Active, Not Recruiting
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Research Team
R
Rafael Bejar, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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