Actively Recruiting
Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS
Led by Clinical Hub for Interventional Research (CHOIR) · Updated on 2026-03-18
12
Participants Needed
5
Research Sites
263 weeks
Total Duration
On this page
Sponsors
C
Clinical Hub for Interventional Research (CHOIR)
Lead Sponsor
T
The University of New South Wales
Collaborating Sponsor
AI-Summary
What this Trial Is About
The goal of this project is to see if two new potential treatments (defactinib and the combination tablet of decitabine/cedazuridine) can safely be combined to improve outcomes in people with high-risk myelodysplastic syndrome (MDS), certain forms of Acute Myeloid Leukaemia (AML), and Chronic Myelomonocytic Leukaemia (CMML). Decitabine/cedazuridine is approved for use by the Australian Therapeutics Goods Administration (TGA) as treatment for MDS. Defactinib is an experimental treatment. This means it is not an approved treatment for MDS in Australia. So far it has been given to over 625 patients in studies across the world. All study participants will receive active treatment, there is no placebo. Participants will take the decitabine/cedazuridine treatment once a day for 5 days in a row (day 1 to day 5) on its own for the first month (cycle). From month 2 participants will take the decitabine/cedazuridine treatment and will also take the defactinib treatment, both for 5 days in a row on days 1 to day 5 each month (cycle). Defactinib is taken twice a day.
CONDITIONS
Official Title
Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosed with intermediate-2 or high-risk myelodysplastic syndrome (MDS), or
- Diagnosed with acute myeloid leukemia (AML) with 20-30% marrow blasts and multilineage dysplasia, or
- Diagnosed with chronic myelomonocytic leukemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Not suitable for allogeneic stem cell transplant
- Female participants of childbearing potential must agree to use two effective contraceptive methods during the study and for 6 months after, and have a negative pregnancy test at screening
- Male participants with female partners of childbearing potential must agree to use two effective contraceptive methods during the study and for 6 months after, and avoid sperm donation
- Provide signed informed consent before any study procedures
You will not qualify if you...
- Acute myeloid leukemia (AML) with 30% or more blasts in bone marrow
- Prior allogeneic or autologous stem cell transplant
- Prior treatment with more than one cycle of hypomethylating agents
- Evidence of central nervous system or pulmonary leukostasis, disseminated intravascular coagulation, or CNS leukemia
- Use of thrombopoiesis-stimulating agents, erythropoiesis stimulating agents, or other hematopoietic growth factors within 28 days before cycle 1
- Use of any other investigational drug from another trial within 28 days before cycle 1
- Use of medications or supplements that interact with defactinib during the study, including strong CYP3A4 or CYP2C9 inhibitors or inducers, or P-glycoprotein inhibitors or inducers
- Current treatment with warfarin unless switched to an alternative anticoagulant
- Use of hydrea for more than 7 days before cycle 1
- Use of corticosteroids above 10mg prednisolone daily unless for conditions other than MDS
- Active inflammatory bowel disease or other gastrointestinal disorders affecting drug absorption or increasing risk of toxicity
- History of other malignancies within the past 12 months, except certain skin or cervical cancers and incidental prostate cancer
- Significant active cardiac disease in the past 6 months including severe heart failure, unstable angina, or recent heart attack
- Baseline QT interval above 440 ms (males) or 450 ms (females)
- Active systemic infections not improving with treatment, active hepatitis B infection, or uncontrolled HIV or hepatitis C infection
- Certain abnormal liver or kidney lab tests beyond specified limits
- Known or suspected allergy to study drugs or their components
- Pregnant or breastfeeding
- Any other condition judged by the investigator to increase risk or interfere with study participation or data interpretation
AI-Screening
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Trial Site Locations
Total: 5 locations
1
Prince of Wales Hospital
Sydney, New South Wales, Australia, 2031
Actively Recruiting
2
Royal Prince Alfred Hospital
Sydney, New South Wales, Australia, 2050
Actively Recruiting
3
Concord Repatriation and General Hospital
Sydney, New South Wales, Australia, 2139
Actively Recruiting
4
Nepean Hospital
Sydney, New South Wales, Australia, 2747
Actively Recruiting
5
Westmead Hospital
Westmead, New South Wales, Australia, 2145
Actively Recruiting
Research Team
J
John Pimanda, Professor
CONTACT
M
Mark Polizzotto, Professor
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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