Active not recruitingPHASE3INTERVENTIONAL

A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

This research study is for people in the UK diagnosed with primary myelofibrosis, or myelofibrosis that developed after polycythemia vera or essential thrombocythemia. It focuses on those who also have a very low platelet count (below 50,000/μL). The study is testing a drug called pacritinib against standard treatments chosen by your doctor. It aims to see if pacritinib is more effective at managing myelofibrosis symptoms. Patients will be randomly assigned to receive either pacritinib or one of several standard treatments, and researchers will carefully monitor how participants respond to their assigned medication.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Swedish Orphan Biovitrum

Enrolment target

407

Start

26 Jun 2017

Estimated completion

13 Oct 2028

Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis

What is this study about?

This study is looking into a new treatment for myelofibrosis, a rare bone marrow disorder where scar tissue replaces healthy bone marrow, affecting blood cell production. Specifically, it's for people with primary myelofibrosis, or those who developed myelofibrosis after having polycythemia vera or essential thrombocythemia. It focuses on individuals whose platelet counts are very low, below 50,000/μL.

The main goal of this study is to compare a drug called pacritinib with other standard treatments that your doctor might usually suggest. These other treatments could include medications like corticosteroids, hydroxyurea, danazol, or a low dose of ruxolitinib. These existing treatments are referred to as 'Physician's Choice' because your doctor would decide which one is most suitable for you, based on established medical practice.

Participants will be placed into one of two groups by chance (randomly) – two-thirds will receive pacritinib, and one-third will receive a Physician's Choice treatment. This setup helps researchers determine if pacritinib works better or has different effects compared to current care. This type of study, known as a Phase 3 study, is crucial for understanding how well new treatments work and whether they are safe before they can be made widely available. The study aims to recruit about 399 patients in total across different locations, including North America and other regions like the UK.

Key takeaways

The study is investigating a new drug, pacritinib, for myelofibrosis.
It focuses on patients with myelofibrosis and very low platelet counts.
Pacritinib is compared against existing standard treatments chosen by a doctor.
Participation involves taking medication, regular check-ups, and symptom monitoring.
You will be randomly assigned to one of two treatment groups.
The study aims to understand if pacritinib is a safe and effective treatment.

Who may be eligible?

This study is for adults aged 18 or over who have been diagnosed with primary myelofibrosis, or myelofibrosis that has developed from polycythemia vera or essential thrombocythemia.

You would need to have a very low platelet count (below 50,000/μL) and your spleen would need to be enlarged – specifically, at least 5cm below your ribs as felt by a doctor. You should also be experiencing certain myelofibrosis symptoms that add up to a specific score on a symptom questionnaire.

It's important that you have not previously been treated with more than one JAK2 inhibitor (a type of drug sometimes used for myelofibrosis), or have had no or very limited previous exposure to them. Your overall health and organ function (like heart, liver, and kidneys) would also need to be within acceptable limits as determined by medical tests.

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?
Do you have a diagnosis of primary myelofibrosis, or myelofibrosis that followed polycythemia vera or essential thrombocythemia?
Is your platelet count currently below 50,000/μL?
Do you have noticeable enlargement of your spleen (at least 5cm below your ribs)?
Are you able to attend regular appointments for assessments and scans?
Have you had no or very limited treatment with JAK2 inhibitor medications before?

Answer every question to see your result.

What does participation involve?

If you are eligible and choose to take part, you will be randomly assigned to one of two treatment groups: two out of three participants will receive pacritinib, and one out of three will receive a standard treatment chosen by your doctor from a pre-approved list. You will take the assigned medication twice a day. The treatment will continue as long as it's helping you, doesn't cause unbearable side effects, or until your condition gets worse. You won't be able to switch between the study treatments.

Throughout the study, you'll have regular visits for assessments, which will include physical examinations, blood tests, and imaging scans like MRIs or CT scans, particularly to check your spleen size. You will also complete questionnaires about your symptoms. Even if you stop taking the study medication early, you should continue with all scheduled check-ups for at least 24 weeks, unless you withdraw consent, your condition changes significantly (e.g., needing a splenectomy), or the study ends. The total duration of your participation will depend on your individual response to treatment.

Potential risks and benefits

Participating in this study might offer the potential benefit of receiving a new treatment, pacritinib, which could help manage your myelofibrosis symptoms, particularly if you have very low platelet counts. However, as with any medication, there are potential risks and side effects associated with both pacritinib and the standard treatments. These will be fully explained to you by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (207)

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

University of Alabama at Birmingham, (UAB) Hospital, Comprehensive Cancer Center
Verified postcode
Birmingham, United States
Mayo Clinic Hospital
Verified postcode
Phoenix, United States
City of Hope
Verified postcode
Duarte, United States
USC Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
UCLA David Geffen School of Medicine
Verified postcode
Los Angeles, United States
University of Colorado Cancer Center
Verified postcode
Aurora, United States
Rocky Mountain Cancer Centers (US Oncology/McKesson)
Verified postcode
Boulder, United States
Yale School of Medicine
Verified postcode
New Haven, United States
Georgetown University Hospital
Verified postcode
Washington D.C., United States
George Washington University-Medical Faculty Associates
Verified postcode
Washington D.C., United States
Cleveland Clinic Florida
Verified postcode
Weston, United States
Northwestern Memorial Hospital
Verified postcode
Chicago, United States

Common questions

What is myelofibrosis?

Myelofibrosis is a rare bone marrow disorder where scar tissue replaces healthy bone marrow, disrupting the normal production of blood cells.

What does 'randomized' mean?

Randomized means you'll be assigned by chance to either receive pacritinib or one of the doctor's choice treatments, like flipping a coin. This helps ensure fair comparison.

What are platelet counts?

Platelets are tiny blood cells that help your blood clot. Low platelet counts can increase the risk of bruising and bleeding.

Can I stop participating in the study at any time?

Yes, you have the right to withdraw from the study at any time, for any reason, without affecting your regular medical care.

What is a 'JAK2 inhibitor'?

JAK2 inhibitors are a type of medication sometimes used to treat myelofibrosis by targeting specific signals in the body that contribute to the disease.