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)

2 1 1 1

What is this study about?

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.

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).

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)

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.

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

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 Study?

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
Must have one of these conditions:
- Acute Myeloid Leukemia (AML)
- Myelodysplastic Syndrome (MDS)
- Chronic Myelomonocytic Leukemia (CMML)

Who Cannot Join the Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country	Status
Aneraysog Ube	Amsterdam	The Netherlands
Exauxos Udhgmbruwfog Mxggwqx Chpgtdc Rxabvdonw (bzxojui Mzq	Rotterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not yet recruiting	16.05.2021

Trial locations

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.

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.

Trial ID:

2024-514588-24-00

Protocol code:

EP0042-101

NCT ID:

NCT04581512

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

Other Trials to Consider

#1 Clinical Trials Search in Europe

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)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?