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Study of EP0042 alone and in combination with venetoclax and azacitidine in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CMML)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial investigates a new treatment approach for patients with blood cancers including Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Chronic Myelomonocytic Leukemia. The study evaluates the safety and effectiveness of a new experimental drug called EP0042, which will be tested both alone and in combination with other cancer medications.

The treatment involves taking EP0042 in capsule form, either by itself or together with two other medications: venetoclax (taken as tablets) and azacitidine (given as an injection). The study aims to find the right dose of EP0042 that is both safe and potentially effective for treating these blood cancers.

During the study, patients will receive the medications and be monitored regularly to check how well they tolerate the treatment and how their disease responds. Doctors will track any side effects and measure how the medications work in the body. The study will also look at how long patients respond to the treatment and their overall survival.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment with EP0042 alone
You will receive EP0042 as tablets taken by mouth. This is the first phase of treatment for patients with advanced blood cancer.
Your doctor will monitor your health closely through regular examinations, blood tests, and heart tests (ECG).
Step 2
Combined treatment option A
If you have a specific type of blood cancer called FLT3 wildtype AML, you may receive a combination of two medications:
- EP0042 tablets taken by mouth
- Venetoclax (Venclyxto) tablets taken by mouth in different strengths (10 mg, 50 mg, or 100 mg)
Step 3
Combined treatment option B
For certain patients, treatment may include three medications:
- EP0042 tablets taken by mouth
- Venetoclax (Venclyxto) tablets taken by mouth
- Azacitidine (Vidaza) given as an injection under the skin
Regular health monitoring will continue throughout this phase.
Step 4
Ongoing monitoring
Throughout the study, your doctor will track:
- How your body responds to the medication
- Any side effects you may experience
- Changes in your blood test results
- Your overall health status
Step 5
Study completion
The study is expected to continue until July 31, 2025
Your participation length will depend on how your body responds to the treatment

Who can join the trial?

9 criteria

Must be at least 18 years old at the time of providing consent
Must have confirmed diagnosis of leukemia (blood cancer) through histological (examination of tissue samples) or cytological (examination of cells) testing
Must be able to understand and provide written informed consent before any study procedures begin
Must agree to allow access to previous tumor tissue samples
Must be able to swallow and retain oral medication (take pills by mouth)
Both men and women may participate in the study
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Must have one of these conditions:
- Acute Myeloid Leukemia (AML)
- Myelodysplastic Syndrome (MDS)
- Chronic Myelomonocytic Leukemia (CMML)

Who cannot join the trial?

16 criteria

Age below 18 years
Pregnancy or breastfeeding women
Participation in another clinical trial within the last 30 days
Active infections that require systemic treatment
Severe heart conditions or uncontrolled high blood pressure
Severe liver problems (liver function tests more than 3 times the normal limit)
Severe kidney problems (creatinine clearance less than 30 mL/min)
History of other cancers in the past 2 years (except for successfully treated non-melanoma skin cancer or cervical cancer in situ)
Known HIV infection (Human Immunodeficiency Virus)
Active hepatitis B or hepatitis C infection
Major surgery within 4 weeks before starting the study
Mental conditions that could interfere with following study procedures
Known allergic reactions to similar medications
Life expectancy less than 3 months
Unable to swallow oral medications
Significant bleeding disorders

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Amsterdam UMC
Amsterdam, The Netherlands
Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands

Trial locations

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Investigated drugs

EP0042
is an investigational anti-cancer medication being studied for treating advanced malignancies, particularly in patients with relapsed or refractory acute myeloid leukemia (AML) and related conditions.
Venetoclax
is a medication that works by blocking a protein called BCL-2, which helps cancer cells survive. It is being studied in combination with EP0042 for patients with specific types of leukemia.
Azacitidine
is a hypomethylating agent that helps fight cancer by affecting how genes are expressed in cancer cells. In this trial, it is being studied as part of a combination treatment with EP0042 and venetoclax.

What is already known about the treatment

EP0042
An investigational drug being studied in clinical trials for the treatment of various blood cancers, including Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Chronic Myelomonocytic Leukemia (CMML). The drug is being evaluated both as a standalone treatment and in combination with other anti-cancer medications, particularly venetoclax and azacitidine. The primary focus of current clinical trials is to establish its safety and tolerability profile in patients with relapsed or refractory blood cancers.
Venetoclax
An oral medication that works by blocking the BCL-2 protein, which helps cancer cells survive. This targeted therapy is being studied in combination with EP0042 for patients with AML, particularly those with FLT3 wild-type disease. The drug has shown promise in treating blood cancers by promoting cancer cell death and is currently being evaluated in combination therapy approaches.
Azacitidine
A hypomethylating agent administered as an injection or oral medication that helps treat blood disorders by preventing the growth of cancer cells. In this clinical trial, it is being studied as part of a triple combination therapy with EP0042 and venetoclax for patients with newly diagnosed or relapsed/refractory AML. The drug works by interfering with DNA production in cancer cells and promoting normal blood cell development.

Investigated diseases

Acute Myeloid Leukemia (AML)
A blood and bone marrow cancer that affects the production of normal blood cells. In AML, abnormal white blood cells rapidly accumulate in the bone marrow, interfering with the production of normal blood cells. The disease develops quickly, with immature blood cells (called blasts) building up in the bone marrow and blood. These abnormal cells fail to function properly and can interfere with the body's normal blood cell production.
Myelodysplastic Syndrome (MDS)
A group of disorders where blood-forming cells in the bone marrow become abnormal and fail to produce enough healthy blood cells. The condition causes immature blood cells to fail to develop normally, leading to low numbers of one or more types of blood cells. The bone marrow produces blood cells that are misshapen and underdeveloped. These abnormal cells often die earlier than healthy cells.
Chronic Myelomonocytic Leukemia (CMML)
A condition that affects blood-forming cells in the bone marrow, causing an increase in white blood cells called monocytes. The disease has features of both a myelodysplastic syndrome and a myeloproliferative disorder. CMML causes abnormal white blood cells to build up in the bone marrow and blood, while other blood cell types may decrease. The condition typically develops gradually over time.