Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

Tamibarotene in Combination with Venetoclax and Azacitidine in Previously Untreated Adult Patients Selected for RARA-positive AML Who are Ineligible for Standard Induction Therapy

A new research study is investigating a combination of medicines for adults who have a specific type of blood cancer called RARA-positive acute myeloid leukaemia (AML) and are not able to have the usual strong initial treatments. The main goal of the study is to check the safety of giving a new drug called tamibarotene alongside two existing drugs, venetoclax and azacitidine. Researchers will carefully watch for any side effects and changes in blood tests, heart readings, and other body signs. Another key aim is to see how many patients achieve a complete or partial remission of their cancer. This is a Phase II study, meaning it's exploring whether this treatment combination is effective and safe enough to be tested on a larger scale in the future.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Syros Pharmaceuticals Inc.

Enrolment target

Start

05 Apr 2024

RARA-positive acute myeloid leukemia

What is this study about?

This study is designed for adults with a type of blood cancer called acute myeloid leukaemia (AML). Specifically, it's for those whose AML cells have a particular genetic marker known as RARA-positive. This is important because the new drug, tamibarotene, works by targeting this specific marker. The study is particularly for patients who, for various health reasons, are not strong enough to receive the usual intense initial treatments for AML.

The main purpose of this research is to see if combining tamibarotene with two other drugs, venetoclax and azacitidine, is a safe and helpful treatment option for these patients. Researchers will be looking very closely at any side effects people might experience and checking various health markers, like blood tests, heart activity (ECGs), and vital signs (like blood pressure and pulse). They also want to find out how many patients achieve remission, which means the cancer cells are reduced or cleared from the blood and bone marrow.

By carefully studying the effects of this drug combination, researchers hope to find new and more effective ways to treat RARA-positive AML, especially for patients who currently have limited treatment options. This is a "Phase II" study, which means it’s exploring whether the treatment works and is safe enough to be considered for further, larger studies in the future.

Key takeaways

This study evaluates a new drug combination for a specific type of AML.
It's designed for adults with RARA-positive AML who can't have standard treatments.
The main goals are to check the combination's safety and effectiveness in achieving remission.
You will receive tamibarotene, venetoclax, and azacitidine if you join.
Regular health checks, including blood tests and ECGs, will be part of the process.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, patients must be adults, generally 18 years old or older. Both men and women can take part.

Importantly, participants need to have a specific type of acute myeloid leukaemia (AML) where their cancer cells show a particular feature called "RARA-positive." Your doctor would test for this. Also, the study prioritises patients who cannot receive the standard, strong starting treatments for AML, often because of other health conditions or being less fit.

There may be other detailed health requirements, such as specific organ function tests, that your study doctor will discuss with you to ensure it's safe for you to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 years or older)?
Have you been diagnosed with RARA-positive acute myeloid leukaemia (AML)?
Has your doctor said that you are not suitable for standard intense induction chemotherapy for AML?
Are you able to attend regular hospital visits for treatment and check-ups?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive a combination of medicines: tamibarotene, venetoclax, and azacitidine. The treatments are given in cycles, and you will have different assessments before and during each cycle. These will include regular blood tests, checks of your heart's activity (ECGs), and measurements of your vital signs like blood pressure and pulse to monitor your health and any side effects. Researchers will also regularly check for changes in your cancer to see if the treatment is working, which might involve bone marrow biopsies. The overall duration of your participation will depend on how you respond to the treatment and your health, but you will be monitored closely throughout.

Potential risks and benefits

Participating in this study might offer a potential benefit: the chance to receive a new combination of treatments for your RARA-positive AML, especially if standard options aren't suitable for you. This treatment might help put your cancer into remission. However, like all medicines, these drugs can have side effects. Researchers will be carefully monitoring you for any unwanted effects, such as changes in your blood, heart, or general well-being. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is RARA-positive AML?

It's a specific type of blood cancer (acute myeloid leukaemia) where the cancer cells have a particular genetic feature called RARA that the new drug targets.

Why is this study for people who can't have standard treatment?

Some people are too unwell or have other health issues that make the usual strong AML treatments too risky for them. This study aims to find a safer option for them.

What does 'remission' mean in this study?

Remission means the cancer signs and symptoms have reduced or disappeared. Researchers will check if the treatment clears the cancer cells from your body.

Are the drugs in this study new?

Tamibarotene is a new drug being tested, while venetoclax and azacitidine are existing drugs already used to treat some blood cancers. This study explores them together.

What is a 'Phase II' study?

Phase II means researchers are looking to see if the treatment works for the condition and is safe enough to be tested in larger groups of people later on.