Not recruitingRare disease

Study of Pembrolizumab and Azacitidine for Patients with Acute Myeloid Leukemia Facing Imminent Relapse

Verified siteRegistered drugNo placebo

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What is this trial about?

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

This clinical trial is focused on studying a type of blood cancer called Acute Myeloid Leukemia (AML), specifically in patients who have a genetic change known as NPM1 mutation. The study is for patients who are at risk of their cancer returning, which is known as a relapse. The treatment being tested involves two medications: Pembrolizumab, also known by its code name MK-3475, and Azacitidine. Pembrolizumab is given as an infusion, which means it is delivered directly into the bloodstream through a vein, while Azacitidine is given as an injection under the skin.

The purpose of the study is to evaluate how safe and effective the combination of Pembrolizumab and Azacitidine is for patients with AML who are experiencing a molecular relapse, which means there are signs of the disease returning at a microscopic level. The study will last for about 24 weeks, during which participants will receive up to 6 cycles of Azacitidine and up to 8 infusions of Pembrolizumab. The researchers will monitor the participants to see how many remain free of events such as a return of the cancer, the need for other treatments, or any cause of death during this period.

Throughout the study, the researchers will also look at overall survival rates, the number of patients who remain event-free after 12 weeks, and any deaths related to the treatment. They will also measure the levels of a marker called MRD (Minimal Residual Disease) to see how it changes over time. This study aims to provide valuable information on whether this combination of treatments can help manage AML in patients with the NPM1 mutation who are at risk of relapse.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Beginning of treatment
Upon joining the clinical trial, the patient will begin treatment with two medications: pembrolizumab and azacitidine.
Pembrolizumab is administered through an intravenous infusion, which means it is given directly into a vein. This will occur once every three weeks.
Azacitidine is administered subcutaneously, meaning it is injected under the skin. This will be done for seven consecutive days every four weeks.
Step 2
Treatment cycles
The treatment is organized into cycles. Each cycle lasts four weeks.
The patient will receive up to six cycles of azacitidine and up to eight infusions of pembrolizumab over the course of the trial.
Step 3
Monitoring and assessments
Throughout the trial, the patient's health and response to the treatment will be closely monitored.
Regular assessments will be conducted to evaluate the effectiveness and safety of the treatment.
Step 4
End of treatment evaluation
After 24 weeks of treatment, an evaluation will be conducted to determine the proportion of patients who remain event-free.
Events are defined as the first hematologic relapse, death from any cause, or the need for additional treatments beyond pembrolizumab and azacitidine.

Who can join the trial?

12 criteria

Signed informed consent.
Negative pregnancy test for women who can have children. This test must be done within 3 days before starting the study treatment. If the urine test is positive or unclear, a blood test will be needed.
Women who can have children must agree to use a reliable method of birth control during the study and for 120 days after the last dose of study medication. Note: Not having sex is acceptable if it is the usual lifestyle and preferred method of birth control for the participant.
Men who can father children must agree to use a reliable method of birth control starting with the first dose of study therapy and for 120 days after the last dose. Note: Not having sex is acceptable if it is the usual lifestyle and preferred method of birth control for the participant.
Must be 18 years of age or older.
Patients with a specific type of leukemia called NPM1mut AML who are in complete remission after standard chemotherapy.
Presence of MRD (minimal residual disease) indicating a high risk of the leukemia returning, confirmed by a central lab.
Patients who cannot have an immediate alloSCT (allogeneic stem cell transplant).
Patients who are not eligible for other intensive treatments.
Plan to receive AZA (azacitidine) therapy for molecular relapse.
ECOG performance status of 0 or 1, which means the patient is fully active or has some symptoms but does not need to stay in bed.
Must have adequate organ function as defined by specific medical tests done during the screening period.

Who cannot join the trial?

10 criteria

Patients with any other active cancer cannot participate.
Patients who have had a heart attack or stroke in the last 6 months are not eligible.
Patients with uncontrolled high blood pressure cannot join the study.
Patients with severe liver disease are excluded.
Patients with severe kidney disease cannot participate.
Patients who are pregnant or breastfeeding are not eligible.
Patients with a known allergy to the study drugs cannot join.
Patients with an active infection that requires treatment are excluded.
Patients who have received another investigational drug within the last 4 weeks cannot participate.
Patients with a history of autoimmune disease, which is when the body's immune system attacks its own cells, are not eligible.

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Verified sites

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

Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Verified
Munich, Germany
Technische Universitaet Dresden
Verified
Dresden, Germany
Universitaetsklinikum Heidelberg AöR
Verified
Heidelberg, Germany

Trial locations

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Investigated drugs

Pembrolizumab
is a type of medication known as an immunotherapy. It works by helping your immune system fight cancer cells. In this trial, pembrolizumab is used to help your body recognize and attack cancer cells more effectively, especially in patients who have a specific type of leukemia called NPM1mut AML and are experiencing a molecular relapse.
Azacitidine
is a medication that is used to treat certain types of blood cancers. It works by interfering with the growth of cancer cells, which can help to slow down or stop the progression of the disease. In this trial, azacitidine is used in combination with pembrolizumab to enhance the treatment's effectiveness in patients with NPM1mut AML who are at risk of a hematological relapse.

What is already known about the treatment

Pembrolizumab
Pembrolizumab is administered as an intravenous infusion, meaning it is given directly into a vein through a needle. It is currently being studied in combination with azacitidine for treating patients with acute myeloid leukemia (AML) who are at risk of relapse. The main therapeutic use of pembrolizumab is to help the immune system recognize and attack cancer cells. At the molecular level, it works by blocking a protein called PD-1, which can prevent the immune system from attacking cancer cells. Pembrolizumab is classified as an immune checkpoint inhibitor, a type of drug that helps the immune system fight cancer.
Azacitidine
Azacitidine is administered as an injection under the skin or as an intravenous infusion. It is being used in combination with pembrolizumab to treat patients with acute myeloid leukemia (AML) who are experiencing a molecular relapse. The primary therapeutic indication for azacitidine is to help control the growth of cancer cells in the blood and bone marrow. At the molecular level, azacitidine works by incorporating into the DNA and RNA of cancer cells, disrupting their growth and leading to cell death. It is classified as a hypomethylating agent, which is a type of drug that can alter the expression of genes in cancer cells.

Investigated diseases

Acute myeloid leukemia – Acute myeloid leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow. It progresses rapidly, leading to the accumulation of immature white blood cells called myeloblasts. These cells crowd out normal blood cells, causing symptoms like fatigue, frequent infections, and easy bruising or bleeding. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. The rapid progression of the disease requires prompt medical attention to manage its effects.