Active not recruitingPHASE2INTERVENTIONAL

Ruxolitinib vs Allogeneic SCT for Patients With Myelofibrosis According to Donor Availability

This research study is for people in the UK aged 18 to 70 with a bone marrow condition called myelofibrosis. Myelofibrosis causes scarring in your bone marrow, affecting how your blood cells are made. The study compares two ways of treating this condition. Some patients who have a matching stem cell donor will receive a stem cell transplant after a three-month course of a drug called ruxolitinib. Other patients, who don't have a suitable donor, will continue taking ruxolitinib. Doctors want to see which approach works best, looking at how symptoms improve, how the spleen changes, and overall quality of life. This is a Phase 2 study, meaning it's still in the earlier stages of testing new treatments.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Universitätsklinikum Hamburg-Eppendorf

Enrolment target

Start

21 Dec 2016

Estimated completion

07 Oct 2025

Bone Marrow Fibrosis

What is this study about?

This study is looking at the best way to treat a serious blood condition called myelofibrosis. Myelofibrosis affects your bone marrow, which is the spongy material inside your bones where blood cells are made. In myelofibrosis, scar tissue builds up in the bone marrow, making it harder for your body to produce healthy blood cells. This can lead to symptoms like tiredness, a swollen spleen, and other problems.

The study aims to compare two different treatment approaches. One approach involves a stem cell transplant (also known as an allogeneic SCT). This is a procedure where healthy blood-forming stem cells are given to you after your own bone marrow has been prepared. This can be a powerful treatment, but it requires a suitable donor. The other approach involves continuing treatment with a medication called ruxolitinib. Ruxolitinib is a drug that helps manage the symptoms of myelofibrosis and can reduce spleen size.

For patients who have a suitable donor, the study involves taking ruxolitinib for three months, followed by a potential stem cell transplant. For patients who don't have a suitable donor, the study involves continuing to take ruxolitinib. The doctors will then compare how well both groups do over time. They will look at many things, including how much the spleen shrinks, how much symptoms improve (like tiredness or itching), your overall quality of life, and the occurrence of any side effects. They will also check if the disease gets better or worse, and how long people live overall.

Key takeaways

Compares two treatments for myelofibrosis: ruxolitinib vs. ruxolitinib plus stem cell transplant.
For adults aged 18-70 with specific types of myelofibrosis.
Aims to find the most effective treatment for improving symptoms and quality of life.
Involves regular hospital visits and health monitoring.
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To be considered for this study, you need to be an adult between 18 and 70 years old with a diagnosis of myelofibrosis. This includes primary myelofibrosis or myelofibrosis that developed after other blood conditions like polycythaemia vera or essential thrombocythemia. Your condition needs to be considered 'intermediate 2' or 'high-risk' by specific medical guidelines, or 'intermediate 1-risk' if you have certain genetic changes or need regular blood transfusions. Importantly, your platelet count (a type of blood cell) must be at least 50. Also, you must not have received ruxolitinib treatment before, and you should be generally well enough to participate, as assessed by your doctor.

There are several reasons why you might not be able to join the study. These include having severe problems with your kidneys, liver, lungs, or heart. For example, if your liver or kidney function is too low, or if your heart is not pumping properly. You also cannot participate if you have HIV, or if you are pregnant or breastfeeding. If you have active serious infections, severe mental health conditions, or have recently taken part in another study with certain experimental drugs, you would also not be eligible. Finally, if your myelofibrosis has changed into a more aggressive type of leukaemia, you would not be able to participate.

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 between 18 and 70 years old?
Do you have a diagnosis of myelofibrosis (primary, post-polycythemia vera, or post-essential thrombocythemia) that is considered intermediate-2, high-risk, or intermediate-1 with specific genetic changes or needing transfusions?
Have you *not* taken ruxolitinib before?
Are your kidney, liver, lung, and heart functions generally healthy?
Are you not pregnant or breastfeeding?

Answer every question to see your result.

What does participation involve?

If you join this study, your participation will involve several visits to the hospital for assessments. You will either receive the medication ruxolitinib for a set period, or you will take ruxolitinib followed by a stem cell transplant if you have a suitable donor. The treatment and transplant process itself can take many months, and there will be regular follow-up appointments afterwards, which could continue for several years, to monitor your health and the effects of the treatment. You will have blood tests, physical examinations, and answer questionnaires about your symptoms and quality of life at various points throughout the study.

Potential risks and benefits

Taking part in this study may offer potential benefits, such as receiving close medical attention and access to a treatment approach that could be very effective for your myelofibrosis. However, all medical treatments carry potential risks. Ruxolitinib can have side effects, and a stem cell transplant is a major procedure with significant risks, including infections and reactions from the donor cells. The research team will explain all known side effects and risks to you in detail. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (14)

Universitätsklinkum Aachen
Verified postcode
Aachen, Germany
HELIOS Klinikum Berlin-Buch
Verified postcode
Berlin, Germany
Universitätsklinikum Bonn
Verified postcode
Bonn, Germany
Universitätsklinikum Düsseldorf
Verified postcode
Düsseldorf, Germany
Universitätsklinkum Halle
Verified postcode
Halle, Germany
University Medical Center Hamburg-Eppendorf
Verified postcode
Hamburg, Germany
Universitätsklinikum Jena
Verified postcode
Jena, Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Verified postcode
Mainz, Germany
Johannes Wesling Klinikum Minden
Verified postcode
Minden, Germany
Universitätsklinikum Münster
Verified postcode
Münster, Germany
Klinikum Nürnberg
Verified postcode
Nuremberg, Germany
Robert-Bosch-Krankenhaus Stuttgart
Verified postcode
Stuttgart, Germany

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where scar tissue builds up in your bone marrow, making it difficult for your body to make healthy blood cells.

What is a stem cell transplant?

A stem cell transplant involves replacing your blood-forming cells with healthy new ones, usually from a matching donor.

What is ruxolitinib?

Ruxolitinib is a medicine used to treat myelofibrosis symptoms and help reduce the size of an enlarged spleen.

Will I definitely get a stem cell transplant if I join?

Only if you have a suitable matching donor and your doctors decide it's the right path after your initial ruxolitinib treatment.

How long will the study last?

Participation duration varies, but generally involves a 3-month initial treatment, followed by ongoing treatment or transplant, and long-term follow-up.