Study on Decitabine and Drug Combination for Elderly Patients with Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia

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What is this study about?

This clinical trial is focused on studying the effectiveness of different treatments for certain blood-related diseases, specifically Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Chronic Myelomonocytic Leukemia (CMML). These are conditions where the bone marrow, which is responsible for producing blood cells, does not function properly. The study aims to compare a new approach called precision therapy with the standard treatment currently used for these diseases in elderly patients.

The trial involves several medications, including Dacogen (decitabine), Nexavar (sorafenib), Vepesid (etoposide), Venclyxto (venetoclax, also known as ABT-199 or GDC-0199), Tafinlar (dabrafenib, also known as GSK2118436), Mekinist (trametinib, also known as GSK1120212B), Atorvastatin Accord (atorvastatin), Vidaza (azacitidine), and Hydroxyurea medac (hydroxycarbamide). Some patients will receive these medications, while others may receive a placebo, which is a substance with no active drug.

The purpose of the study is to evaluate how cost-effective the precision therapy strategy is compared to the standard treatment. Participants will be randomly assigned to receive either the precision therapy or the standard treatment. The study will monitor the health outcomes and costs associated with each treatment approach over a period of time. This will help determine which treatment strategy provides better value in terms of improving quality of life and managing the diseases effectively.

1 joining the study

Upon joining the study, you will be randomly assigned to either the precision therapy group or the standard therapy group. This process is called randomization and ensures that each participant has an equal chance of receiving either treatment.

2 initial assessment

You will undergo an initial assessment to evaluate your health status. This may include blood tests, imaging studies, and other necessary evaluations to confirm your eligibility and establish a baseline for your treatment.

3 treatment phase

If you are in the precision therapy group, you may receive medications such as decitabine (Dacogen 50 mg) administered intravenously, sorafenib (Nexavar 200 mg) taken orally, or other specified drugs. The specific medication, dosage, and frequency will depend on your individual treatment plan.

If you are in the standard therapy group, you may receive medications like azacitidine (Vidaza 25 mg/ml) administered as a subcutaneous injection or other standard treatments. The specific medication, dosage, and frequency will be determined by your healthcare provider.

4 monitoring and follow-up

Throughout the trial, your health will be closely monitored. This includes regular visits to the clinic for check-ups, blood tests, and other necessary evaluations to assess your response to the treatment and manage any side effects.

You will be required to report any side effects or changes in your health to the study team promptly.

5 end of treatment

At the end of the treatment phase, you will undergo a final assessment to evaluate the outcomes of the therapy. This may include similar tests and evaluations as the initial assessment.

The study team will discuss the results with you and provide guidance on the next steps for your care.

6 post-trial follow-up

After completing the trial, you may be asked to participate in follow-up visits to monitor your long-term health and gather additional data on the effectiveness of the treatment.

These follow-up visits are important for understanding the long-term benefits and any potential risks associated with the treatment.

Who Can Join the Study?

Patients with one of the following conditions:
- A newly diagnosed or returning case of Acute Myeloid Leukemia (AML) and related conditions, except for a specific type called acute promyelocytic leukemia.
- Acute leukemias of unclear origin.
- A newly diagnosed or returning case of Myelodysplastic Syndrome (MDS) with a specific risk score greater than 4.5.
- A newly diagnosed or returning case of Chronic Myelomonocytic Leukemia (CMML-2) with 10-19% immature blood cells in the bone marrow.
Patients must be 60 years or older.
Patients must not be eligible for intensive chemotherapy or a stem cell transplant from a donor.
White blood cell count must be 30 x 10⁹/L or less. Temporary treatment with hydroxyurea is allowed for up to 14 days but must be stopped before starting the study treatment.
Patients must have adequate kidney and liver function, unless the disease affects these organs. This is measured by:
- Serum creatinine (a kidney function marker) must be 2.5 mg/dL or less, unless related to AML.
- Serum bilirubin (a liver function marker) must be 2.5 times the upper limit of normal or less, unless related to AML or a condition called Gilbert’s syndrome.
- Alanine transaminase (ALT, a liver enzyme) must be 2.5 times the upper limit of normal or less, unless related to AML.
Patients aged 75 or older must have a WHO performance status of 0, 1, or 2. Patients aged 60 to 74 must have a status of 0 to 3. This status measures the patient’s ability to perform daily activities.
Patients must sign an informed consent form, agreeing to participate in the study.
Male patients only: Male patients who are sexually active must agree to use condoms for contraception from the first day of the study until at least 90 days after the last dose of the study drug. They must also agree not to donate sperm during this period. If the study drug requires longer contraception, the specific guidelines should be followed.

Who Cannot Join the Study?

Patients with Chronic myelomonocyte leukemia cannot participate.
Patients with Myelodysplastic syndrome cannot participate.
Patients with Acute myeloid leukemia cannot participate.

Where you can join this trial?

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Site Name	City	Country	Status
Aubhqfbc Uapuwwnlld Hzmxrdfr	Lorenskog	Norway

Trial status

Country	Status	Recruitment Start
Norway	Not recruiting	02.07.2021

Trial locations

Investigated drugs:

Precision Therapy is a treatment approach that involves using specific medications tailored to the genetic makeup of a patient’s cancer. In this trial, precision therapy aims to target the unique characteristics of cancer cells in patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). This approach is designed to be more effective and potentially have fewer side effects than standard treatments by focusing on the specific mutations or abnormalities present in the cancer cells.

Standard Therapy refers to the conventional treatment methods commonly used for patients with AML and MDS. This typically includes a combination of chemotherapy drugs that work to kill rapidly dividing cancer cells. Standard therapy is based on established protocols and has been used for many years to treat these types of cancers. The goal of standard therapy is to reduce the number of cancer cells and achieve remission, although it may not be as specifically targeted as precision therapy.

Investigated diseases:

Myelodysplastic syndrome

Chronic myelomonocytic leukemia – This disease is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. It is characterized by an increase in monocytes, a type of white blood cell, in the blood. Over time, the disease can lead to a decrease in other types of blood cells, causing symptoms like fatigue and easy bruising. The progression can vary, with some patients experiencing a slow course while others may see a more rapid development of symptoms. The disease can also lead to an enlarged spleen and liver. It is considered a type of myelodysplastic/myeloproliferative disease.

Myelodysplastic syndrome – This condition involves a group of disorders caused by poorly formed or dysfunctional blood cells. It originates in the bone marrow, where blood cells are produced, leading to a shortage of healthy blood cells in the bloodstream. Patients may experience symptoms such as fatigue, shortness of breath, and increased risk of infections due to low blood cell counts. The disease can progress slowly or more rapidly, depending on the specific type and severity. Over time, it may lead to more severe forms of blood disorders. It is often associated with abnormalities in the bone marrow and blood cell production.

Acute myeloid leukemia – This is a fast-growing cancer of the blood and bone marrow, characterized by the rapid proliferation of abnormal white blood cells. These cells accumulate in the bone marrow, interfering with the production of normal blood cells. Symptoms can include fatigue, fever, frequent infections, and easy bruising or bleeding. The disease progresses quickly, requiring prompt medical attention. It can lead to a significant reduction in red blood cells, white blood cells, and platelets. The rapid accumulation of abnormal cells can also cause swelling in the liver and spleen.

Trial ID:

2023-509092-16-00

Protocol code:

PALM

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Decitabine and Drug Combination for Elderly Patients with Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?