Not recruitingRare disease

Study of Tamibarotene, Venetoclax, and Azacitidine for Adults with RARA-positive Acute Myeloid Leukemia Not Eligible for Standard Therapy

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 certain characteristic known as RARA-positive. The study is testing a combination of three treatments: tamibarotene, venetoclax, and azacitidine. Tamibarotene, also known by its code name SY-1425, is a medication that targets specific leukemia cells. Venetoclax is a drug that helps to kill cancer cells by blocking a protein that prevents cell death. Azacitidine is a medication that works by interfering with the growth of cancer cells.

The purpose of this study is to understand how safe and tolerable the combination of tamibarotene, venetoclax, and azacitidine is for patients with RARA-positive AML who have not received treatment before and are not eligible for standard intensive chemotherapy. The study will also compare the effectiveness of the three-drug combination to just venetoclax and azacitidine. Participants will take these medications in cycles, with each cycle lasting a few weeks. The study will monitor the participants' health and response to the treatment over time.

Throughout the study, participants will receive regular check-ups to assess their health, including blood tests and other evaluations to monitor the effects of the treatment. The study aims to gather information on how well the treatment works and any side effects that may occur. This information will help determine the best dose and schedule for using these medications together in treating RARA-positive AML.

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
Initiation of treatment
Upon joining the study, the patient begins treatment on Cycle 1 Day 1. The treatment involves a combination of medications: tamibarotene, venetoclax, and azacitidine.
The patient must have a confirmed RARA-positive status by Cycle 1 Day 8 to continue in the study.
Step 2
Medication administration
Tamibarotene is administered orally in tablet form. The specific dosage and frequency are determined by the study protocol.
Venetoclax is also administered orally in tablet form. The dosage and frequency are adjusted based on the patient's response and tolerability.
Azacitidine is administered either subcutaneously or intravenously. The dosage and frequency are specified in the study protocol.
Step 3
Monitoring and assessments
Throughout the trial, the patient undergoes regular monitoring to assess safety and tolerability. This includes clinical laboratory tests, ECGs, and vital sign measurements.
The patient's response to the treatment is evaluated through various assessments, including complete remission (CR) and complete remission with incomplete blood count recovery (CRi).
Step 4
Continuation and adjustments
Based on the patient's response and any side effects experienced, adjustments to the medication dosage and regimen may be made.
The study aims to determine the most effective and tolerable dose and regimen of the combination therapy.
Step 5
Completion of trial
The trial is estimated to conclude by April 30, 2028. The patient's participation may continue until the study's end or until the patient meets specific criteria for discontinuation.
Final assessments are conducted to evaluate the overall response and any long-term effects of the treatment.

Who can join the trial?

9 criteria

Patients must be at least 18 years old when they agree to participate in the study.
Patients need to have a blood test to check for a specific marker called **RARA** before starting treatment. The test must show they are **RARA-positive** by the eighth day of the first treatment cycle to join the study.
Patients should have a type of leukemia called **acute myeloid leukemia (AML)** that has not been treated before. They must have a certain number of leukemia cells in their blood or bone marrow and be unable to handle strong chemotherapy due to age, health status, or other health issues.
Patients must have a performance status score, which measures their ability to perform daily activities, between 0 to 3 if they are under 75 years old, or 0 to 2 if they are 75 or older.
Patients need to have a white blood cell count below 25,000 per microliter before starting the study drug. If needed, procedures or medications can be used to lower the count.
Patients must have certain levels of organ function, including liver and kidney function, as measured by specific tests.
Women who can become pregnant must have a negative pregnancy test before starting treatment.
Patients must agree to follow the study schedule, treatment plans, and tests. Women who can become pregnant and male patients must use two methods of birth control, including a barrier method.
Patients must be able to provide written consent to participate in the study.

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Verified sites

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

Assistance Publique Hopitaux De Paris
Verified
Paris, France
Centre Hospitalier De La Cote Basque
Verified
Bayonne, France
Centre Hospitalier De Versailles
Verified
Le Chesnay-Rocquencourt, France

Trial locations

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Investigated drugs

Tamibarotene
is a medication being studied for its potential to treat patients with a specific type of acute myeloid leukemia (AML) that is positive for RARA. It is being tested in combination with other drugs to see if it can improve treatment outcomes for patients who have not received prior treatment and are not eligible for standard induction therapy.
Venetoclax
is a medication used to help treat certain types of blood cancers by targeting and inhibiting a protein that helps cancer cells survive. In this trial, it is combined with other medications to evaluate its effectiveness in treating RARA-positive AML.
Azacitidine
is a medication that works by interfering with the growth of cancer cells. It is commonly used in the treatment of certain blood disorders and is being tested in combination with other drugs in this trial to assess its role in treating RARA-positive AML.

What is already known about the treatment

Tamibarotene
Tamibarotene is administered orally and is currently being studied in combination with other drugs for the treatment of RARA-positive acute myeloid leukemia (AML). It is not yet widely used in standard medical practice but is under investigation in clinical trials. The main therapeutic indication is for patients with RARA-positive AML who are not eligible for standard induction therapy. At the molecular level, tamibarotene works by binding to specific receptors in the body, which can influence the growth and survival of cancer cells. It is classified as a retinoic acid receptor agonist.
Venetoclax
Venetoclax is taken orally and is approved for use in certain types of leukemia, including chronic lymphocytic leukemia and AML. It is being studied in combination with other medications for RARA-positive AML. Venetoclax is a BCL-2 inhibitor, which means it helps to promote the death of cancer cells by blocking a protein that allows them to survive. This medication is part of a class of drugs known as apoptosis inducers.
Azacitidine
Azacitidine is administered via injection and is used in the treatment of certain blood disorders, including AML. It is being evaluated in combination with other drugs for RARA-positive AML. Azacitidine works by incorporating into the DNA of cancer cells, disrupting their growth and division. It is classified as a DNA methyltransferase inhibitor, which helps to restore normal function to genes that control cell growth.

Investigated diseases

RARA-positive acute myeloid leukemia – This is a type of acute myeloid leukemia characterized by the presence of a specific genetic marker known as RARA. It is a cancer of the blood and bone marrow that affects the production of blood cells. The disease progresses rapidly, leading to the accumulation of immature white blood cells, known as blasts, which interfere with normal blood cell production. Patients may experience symptoms such as fatigue, frequent infections, and easy bruising or bleeding due to the lack of healthy blood cells. The condition is considered rare and requires specialized medical attention to manage its progression.