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

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What is this study about?

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.

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.

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.

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).

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.

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 Study?

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.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Institut Gustave Roussy	Villejuif	France
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France

Other Sites

Site Name	City	Country
Centre Hospitalier De Versailles	Le Chesnay-Rocquencourt	France
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hôpital Avicenne	Bobigny	France
Centre Hospitalier De La Cote Basque	Bayonne	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Nice	Nice	France
Centre Hospitalier Universitaire De Caen Normandie	Caen	France
Cmnwvu Hdjhouexlyh Rlvjkjtx Dhvulcibropkaw	Angers	France
Cwziqm Hweuxalvljm Ez Unnqcqicnltfy Da Lpobfcd	Limoges	France
Auxszfflax Pofvrtlu Hcjyohwn Db Pxxvv	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.08.2021

Trial locations

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.

Investigated diseases:

Acute myeloid leukaemia

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.

Trial ID:

2024-510939-21-00

Protocol code:

SY-1425-202

NCT ID:

NCT04905407

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?