RecruitingPHASE3INTERVENTIONAL

A Phase 3 Study of INCA033989 Versus Best Available Therapy in Participants With Essential Thrombocythemia

This research study is looking at a new treatment called INCA033989 for a blood condition called essential thrombocythemia (ET). ET causes your body to make too many platelets, which are tiny blood cells that help stop bleeding. The study aims to compare INCA033989 with treatments that doctors currently use, known as 'best available therapy.' We want to find out if this new medicine is safe and effective for people with ET who have a specific gene change called a CALR mutation and have already received other treatments for their condition. The study is in Phase 3, meaning it's one of the final steps before a new medicine might be approved for wider use. It's an important step in finding better ways to manage ET.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Incyte Corporation

Enrolment target

426

Start

08 Sep 2026

Estimated completion

01 Nov 2030

Essential Thrombocythemia

What is this study about?

This study is about finding better ways to help people with a condition called essential thrombocythemia, often shortened to ET. ET is a long-term blood disorder where your body produces too many platelets. Platelets are important for clotting blood, but having too many can lead to problems like unusual bleeding or blood clots.

The main goal of this study is to compare a new medicine, called INCA033989, with the treatments that doctors currently consider the best options for ET. These current treatments are called 'best available therapy.' The study focuses on people who have a specific genetic change, known as a CALR mutation, which is often found in ET. Participants in this study will have already tried other medicines for their ET.

By comparing the new medicine with existing treatments, researchers hope to learn if INCA033989 is more effective at managing ET symptoms, reducing risks, and improving the overall health of patients. This is a 'Phase 3' study, which means it's a large and important step in determining if the new medicine could eventually become a standard treatment for ET.

Key takeaways

This study is testing a new medicine (INCA033989) for essential thrombocythemia (ET).
It's for people with a CALR gene change who've already tried other ET treatments.
The study compares the new medicine to standard 'best available' treatments.
It aims to see if INCA033989 is a safe and effective new option for ET patients.
Participation involves regular clinic visits and close health monitoring.

Who may be eligible?

To join this study, you would first need to have a confirmed diagnosis of essential thrombocythemia (ET) that doctors consider 'high-risk.' This means your ET has certain features that make it more likely to cause complications. You also need to have a specific genetic change called a CALR mutation. This mutation will be checked if you don't already know if you have it. Additionally, you must have already tried at least one other medicine to help lower your platelet count (this is called cytoreductive therapy).

There are also some reasons why you might not be able to join. For example, if you have any other type of blood cancer apart from ET, or if you've had major bleeding or a serious blood clot in the last three months. You also can't have had a stem cell transplant in the past.

Other reasons for not being able to join include if you still have serious side effects (Grade 2 or worse) from previous treatments, unless these are long-term, stable issues not expected to get better, like some nerve problems. If you've had certain other cancers in the past, you might not be eligible unless they were successfully treated more than two years ago and show no signs of returning. The study team will review your full medical history to make sure the study is right for you.

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.

Do I have a confirmed diagnosis of high-risk essential thrombocythemia (ET)?
Have I been told I have a CALR gene mutation?
Have I already taken at least one medicine to lower my platelet count (cytoreductive therapy)?
Have I had any other blood cancers or recent serious bleeding/clots?
Have I had a stem cell transplant before?
Are any side effects from my previous treatments still serious?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned to receive either the new medicine, INCA033989, or one of the 'best available therapies' that your doctor would normally prescribe for your condition. This will be decided by chance, like flipping a coin. You will have regular visits to the study clinic for check-ups, blood tests, and other assessments to monitor your health and how well the treatment is working. These visits will help doctors understand any changes in your condition and to watch for any side effects. You will be taking the study medication for a certain period, and there will be follow-up appointments even after you stop taking the medicine to continue monitoring your health. The total time you would be involved in the study, including follow-up, will be explained by the study team, but typically clinical trials can last for several months or even a few years.

Potential risks and benefits

Taking part in this study could offer some potential benefits, such as access to a new treatment (INCA033989) that is not yet widely available and might be more effective than current options. You would also receive close medical attention and monitoring throughout the study. However, like all medicines, INCA033989 may have side effects, some of which could be serious or unexpected. The 'best available therapies' also have known risks and side effects. There's also no guarantee that the new treatment will work for you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (180)

Banner Md Anderson Cancer Center
Verified postcode
Gilbert, United States· Not yet recruiting
The University of Arizona Cancer Center
Verified postcode
Tucson, United States· Not yet recruiting
City of Hope Medical Center
Verified postcode
Duarte, United States· Not yet recruiting
Marin Cancer Care
Verified postcode
Greenbrae, United States· Recruiting
University of California, Los Angeles Medical Center
Verified postcode
Los Angeles, United States· Not yet recruiting
Stanford Cancer Center
Verified postcode
Palo Alto, United States· Not yet recruiting
UCSF
Verified postcode
San Francisco, United States· Withdrawn
Rocky Mountain Cancer Centers-Lone Tree
Verified postcode
Lone Tree, United States· Not yet recruiting
Medstar Georgetown Medical Center, Inc
Verified postcode
Washington D.C., United States· Not yet recruiting
Florida Cancer Specialists & Research Institute
Verified postcode
Fort Myers, United States· Not yet recruiting
University of Miami Sylvester Comprehensive Cancer Center
Verified postcode
Miami, United States· Not yet recruiting
Cleveland Clinic
Verified postcode
Weston, United States· Not yet recruiting

Common questions

What is essential thrombocythemia (ET)?

ET is a blood condition where your body makes too many platelets, which are cells that help your blood clot. This can lead to problems with bleeding or blood clots.

What is a CALR mutation?

It's a specific change in your genes that is often found in people with ET. The study is specifically looking at patients who have this mutation.

What does 'cytoreductive therapy' mean?

This is a type of medicine or treatment that helps to reduce the number of blood cells, in this case, platelets, in your body.

What does 'Phase 3' mean for a study?

Phase 3 means this is one of the final and largest stages of testing a new medicine. It compares the new treatment with existing ones to see if it's safe and effective before it can be approved for wider use.

Will I know which treatment I'm receiving?

In some studies like this one, participants are randomly assigned to a treatment group, and often neither you nor your study doctor will know which treatment you are receiving (this is called 'blinding'). This helps ensure the results are fair.

How to find out more

Incyte Corporation Call Center (US)

medinfo@incyte.com 1.855.463.3463 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.