Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Randomized, Double-blind, Placebo-controlled Phase 3 Study of Tamibarotene Plus Azacitidine Versus Placebo Plus Azacitidine in Newly Diagnosed Adult Patients Selected for RARA-positive Higher-risk Myelodysplastic Syndrome

This research study is for adults who have recently been diagnosed with a type of blood condition called higher-risk myelodysplastic syndrome (MDS) that tests positive for a specific marker called RARA. The study is comparing a new medication, tamibarotene, when given with a standard treatment called azacitidine, against receiving a dummy drug (placebo) with azacitidine. It's a 'Phase 3' study, which means it's one of the final steps before a new drug might become widely available. The main goal is to find out if adding tamibarotene increases the number of people who achieve a 'complete remission,' where signs of the disease significantly decrease or disappear. Researchers will also look at how long people live, if they need fewer blood transfusions, and how long any improvements last.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Syros Pharmaceuticals Inc.

Enrolment target

417

Start

06 Jun 2024

Newly Diagnosed RARA-positive Adult Patients with Higher-risk Myelodysplastic Syndrome

What is this study about?

This study is looking into a blood condition called myelodysplastic syndrome, or MDS. Specifically, it's for adults who have a higher-risk type of MDS that has been recently diagnosed and shows a particular genetic marker called 'RARA-positive.' MDS means your body isn't making enough healthy blood cells. In higher-risk MDS, the condition is more likely to worsen, so finding effective treatments is very important.

The study is testing a new drug called tamibarotene. Participants will be split into two groups by chance (like flipping a coin). One group will receive tamibarotene along with a standard treatment called azacitidine. The other group will receive a 'placebo' (a dummy drug that looks like tamibarotene but contains no active medicine) along with azacitidine. This is so researchers can fairly compare if tamibarotene makes a difference when added to the usual treatment. Neither you nor your doctor will know which treatment you are receiving until the end of the study, which helps ensure the results are unbiased.

The main aim of this study is to see if more people achieve a 'complete remission' with the new drug combination. Complete remission means that tests show the disease has significantly improved or is no longer detectable. The researchers will also be carefully watching other important things, like how long people live, if they need fewer blood transfusions, how long any improvements last, and how quickly people respond to the treatment. This helps us understand the full effects of the new treatment.

Key takeaways

This study is for adults with recently diagnosed, RARA-positive, higher-risk MDS.
It's testing a new drug (tamibarotene) combined with standard treatment (azacitidine) against a placebo with azacitidine.
The main goal is to see if the new combination increases complete remission rates.
You would receive close medical monitoring and regular assessments.
Participation is voluntary, and you can withdraw at any time.
Results from this Phase 3 study could help bring a new treatment option to patients.

Who may be eligible?

This study is for adult patients, meaning you must be at least 18 years old. There's no upper age limit.

You must have been recently diagnosed with a higher-risk form of myelodysplastic syndrome (MDS). Importantly, your specific type of MDS needs to be positive for a particular genetic marker called RARA. Your doctor will be able to tell you if you meet this specific requirement.

Since this is for newly diagnosed patients, you likely haven't received extensive prior treatment for your MDS, though your doctor will be able to confirm if your medical history aligns with the study's exact criteria.

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 18 years old or older?
Have you been recently diagnosed with myelodysplastic syndrome (MDS)?
Has your doctor told you that your MDS is a 'higher-risk' type?
Has your doctor confirmed that your MDS is 'RARA-positive'?
Have you not had extensive previous treatments for your MDS?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive either the new study drug (tamibarotene) or a placebo, both in addition to the standard azacitidine treatment. These medications would be given as injections. You would have regular appointments for check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. The study involves a 'double-blind' approach, meaning neither you nor your study doctor will know whether you are receiving the active new drug or the placebo, ensuring fairness in the study results. The exact number of visits and the total duration in the study will be explained in detail by the study team, and it would involve ongoing monitoring and follow-up.

Potential risks and benefits

Taking part in a clinical trial offers the chance to access a new potential treatment that isn't yet widely available, and you would receive very close medical monitoring. While the new drug might offer benefits, it's also possible it may not help your condition, or you might experience side effects, some of which could be serious. All potential risks and side effects will be fully explained to you before you decide to participate. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

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

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Unverified
Austria
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Unverified
Poland
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Unverified
Czechia
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Unverified
Belgium
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Unverified
Germany
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Unverified
Spain
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Unverified
France
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Unverified
Hungary
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Unverified
Italy

Common questions

What is myelodysplastic syndrome (MDS)?

MDS is a condition where your body doesn't produce enough healthy blood cells in the bone marrow. In 'higher-risk' MDS, there's a greater chance of it developing into a more serious blood cancer.

What does 'RARA-positive' mean?

RARA-positive refers to a specific genetic feature found in some types of MDS. This study is specifically looking for people with this marker because the new drug is designed to work with it.

What is azacitidine?

Azacitidine is a standard medication commonly used to treat MDS. It helps improve the way blood cells are made and can slow the progression of the disease.

What is a 'placebo'?

A placebo is an inactive substance or treatment that looks exactly like the real medication but contains no active drug. It's used to compare against the new drug to see if the drug itself is causing any effects.

What is 'complete remission'?

Complete remission means that after treatment, the signs and symptoms of your disease have either significantly reduced or are no longer detectable through medical tests.