RecruitingPHASE2INTERVENTIONAL

Pacritinib vs. Hydroxyurea in Advanced Proliferative Chronic Myelomonocytic Leukemia

This study is investigating a new medication, pacritinib, for adults with an advanced form of chronic myelomonocytic leukemia (CMML). CMML is a specific type of blood cancer. The main goal is to find out if pacritinib is more effective at controlling the disease and has fewer side effects compared to a common treatment called hydroxyurea. Participants will be randomly assigned to receive either pacritinib twice daily or hydroxyurea for up to 48 weeks. After this treatment period, researchers will continue to check on participants for about a year to understand the long-term effects. The study aims to provide clearer information about the best ways to treat this challenging condition.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Theradex

Enrolment target

Start

01 Jan 2026

Estimated completion

01 Dec 2028

Leukemia, Myelomonocytic, Chronic

What is this study about?

This clinical trial is designed to investigate a potential new treatment for a specific type of blood cancer called chronic myelomonocytic leukemia (CMML), specifically in its advanced form. CMML is a condition where too many abnormal white blood cells are made in the bone marrow. Currently, one common treatment for this condition is a medicine called hydroxyurea. This study aims to see if a newer medicine, pacritinib, could be a better option.

The main questions the researchers want to answer are: Does pacritinib control the cancer more effectively than hydroxyurea? And what are the side effects of each medicine? By comparing these two treatments, the study hopes to find out if pacritinib is not only better at fighting the disease but also easier for people to tolerate. Participants will take either pacritinib or hydroxyurea for about 48 weeks (nearly a year).

It's important to understand that this is a 'Phase 2' study. This means the medicine pacritinib has already been tested in earlier stages, and now researchers want to learn more about its effectiveness and safety in a larger group of people with this specific type of CMML. The results of this study will help doctors better understand how to treat this condition in the future.

Key takeaways

This study compares a new medicine (pacritinib) with a standard one (hydroxyurea) for advanced CMML.
It aims to see if pacritinib is better at controlling the cancer and has fewer side effects.
Participants will take one of the medicines for up to 48 weeks.
Your involvement will last for about two years, including follow-up checks.
You'll be closely monitored by a medical team throughout the study.

Who may be eligible?

To be able to take part in this study, you would need to have an advanced form of chronic myelomonocytic leukemia (CMML), specifically the CMML-1 type. Your doctor would check if your CMML shows certain signs, such as a high white blood cell count, an enlarged spleen, or a low platelet count. Your overall health and how well your organs are working will also be checked to make sure it's safe for you to participate.

There are also some reasons why you might not be able to join. For example, if you've had certain other cancers recently, or if you've had a bone marrow transplant in the last year. You also can't have taken certain other medications for your CMML recently, like another type of medicine called a JAK inhibitor, or if you are already expected to have a bone marrow transplant soon. Women who could become pregnant and men must agree to use effective birth control during and for a period after the study.

The study is open to adults aged 18 and over, of any gender. Your doctor will carefully review all the specific criteria to confirm if this study is suitable 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.

Are you an adult aged 18 or older?
Do you have advanced CMML-1, with a high white blood cell count, enlarged spleen, or low platelets?
Have you NOT had certain other cancers recently (within 2 years)?
Have you NOT had a bone marrow transplant within the last year, or expect to have one soon?
Have you NOT taken certain other CMML medicines recently (like a JAK inhibitor)?
Are you willing to use effective birth control if you are a woman who could become pregnant or a man?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll first have a screening period of about a month to check if the study is right for you. If you meet all the requirements, you will be randomly assigned to one of two groups: either you'll receive pacritinib twice a day, or you'll receive hydroxyurea. You won't get to choose which medicine you receive, as this helps make the study fair.

The treatment period will last for up to 48 weeks (about 11 months). During this time, you'll have regular visits to the clinic so the research team can monitor your health, check how you're responding to the treatment, and manage any side effects. After your treatment period finishes, you'll be followed up for an additional year to see how you're doing long-term. In total, your involvement in the study could last for about two years, including the initial checks, treatment, and follow-up.

Potential risks and benefits

Participating in a clinical trial offers the chance to receive a new medication that might be more effective than standard treatments, and you'll receive close medical monitoring. However, there are potential risks, as pacritinib is still being investigated, and it might have side effects that are not yet fully known or could be more severe than those of hydroxyurea. Hydroxyurea also has known side effects. It's important to remember that the new treatment might not work for everyone, and there's always a risk of experiencing uncomfortable or serious side effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (6)

Moffitt Cancer Center
Verified postcode
Tampa, United States· Recruiting
Winship Cancer Institute at Emory
Verified postcode
Atlanta, United States· Recruiting
Mayo Clinic Rochester
Verified postcode
Rochester, United States· Recruiting
MD Anderson Cancer Center
Verified postcode
Houston, United States· Recruiting
King's College Hospital, NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting
The Christie, NHS Foundation Trust
Verified postcode
Manchester, United Kingdom· Recruiting

Common questions

What is CMML?

CMML stands for Chronic Myelomonocytic Leukemia. It's a type of blood cancer where your bone marrow makes too many abnormal white blood cells.

What does 'randomly assigned' mean?

This means you will be put into a treatment group (either pacritinib or hydroxyurea) by chance, like flipping a coin. Neither you nor your doctor will choose which medicine you get.

How long will I take the study medicine?

You will take the study medicine for up to 48 weeks, which is almost a year.

Can I stop taking part in the study?

Yes, you can choose to stop participating in the study at any time, for any reason. Your decision will not affect your usual medical care.

Will I know which medicine I'm taking?

Yes, this is an 'open-label' study, which means both you and your study doctor will know whether you are receiving pacritinib or hydroxyurea.

How to find out more

Zach Albaugh

zalbaugh@theradex.com Official trial record

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