CompletedPhase IIInterventional

A study on the safety and effectiveness of fedratinib with ropeginterferon alfa-2b in patients with myelofibrosis

Myelofibrosis is an uncommon bone marrow cancer that causes scar tissue to build up in the bone marrow, making it difficult for the body to produce healthy blood cells. Many people with this condition have an altered gene known as JAK2. The FEDORA study tests two medicines taken together: fedratinib, a daily tablet that blocks abnormal cancer cell growth, and ropeginterferon alfa-2b, an injection given under the skin that helps the immune system recognise and fight the disease. Doctors want to find out if combining these two drugs is safe, manageable, and effective at shrinking an enlarged spleen and improving symptoms. Participants will take fedratinib on its own for the first month, before slowly adding the injection. The study lasts for up to two years, during which doctors will closely monitor participants' health, blood counts, and quality of life.

At a glance

Status

Completed

Phase

Phase II

Sponsor

University of Birmingham

Enrolment target

Start

12 Sep 2022

Estimated completion

31 Mar 2024

Myelofibrosis

What is this study about?

Myelofibrosis is a rare type of blood cancer that affects the bone marrow, the spongy centre inside your bones where blood cells are made. In myelofibrosis, scar tissue slowly replaces normal marrow, making it difficult to produce enough healthy red blood cells, white blood cells, and platelets. This often leads to severe tiredness, weakness, frequent infections, and an enlarged spleen, which can cause discomfort in your tummy.

Over half of people with myelofibrosis have a fault in a gene called JAK2. This faulty gene causes the body to make too many abnormal blood cells. Current treatments called JAK2 inhibitors can help ease symptoms and shrink the spleen, but they do not cure the disease. Interferon is another type of treatment that supports your immune system and may make cancer cells more sensitive to other medicines.

The FEDORA trial is testing whether combining a JAK2 inhibitor (fedratinib tablets) with a gentle, long-acting interferon (ropeginterferon alfa-2b injections) works better than using one medicine alone. Researchers hope this two-pronged approach will help control the cancer more effectively and improve everyday quality of life for people living with myelofibrosis.

Key takeaways

Testing a new drug combination (a daily tablet and regular injection) for myelofibrosis.
Aimed at adults with myelofibrosis who carry the JAK2 gene mutation.
People who have already received JAK2 inhibitors or interferon cannot take part.
The treatment and monitoring period lasts for up to two years.
Patients are monitored very closely for changes in blood cell counts and signs of infection.

Who may be eligible?

You may be able to join this study if you are 18 or older and have been diagnosed with primary or secondary myelofibrosis that requires treatment. You must also have the specific JAK2 gene change confirmed by a test, have sufficient blood counts (such as platelets and white blood cells), and have an active disease that meets certain risk levels.

You will not be able to participate if you have previously taken a JAK2 inhibitor or an interferon medicine. The study is also not suitable for people with very low levels of vitamin B1 (thiamine), active heart problems, uncontrolled thyroid or autoimmune disorders, or a history of severe mental health conditions like major depression. Anyone who is pregnant, breastfeeding, or planning a pregnancy cannot take part.

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.

I am aged 18 or over.
I have been diagnosed with myelofibrosis and have tested positive for the JAK2 gene change.
My doctor has advised that my myelofibrosis now needs medical treatment.
I have never been treated with a JAK2 inhibitor (such as ruxolitinib) or an interferon medicine.
I do not have a history of severe depression, serious heart problems, or untreated autoimmune disease.

Answer every question to see your result.

What does participation involve?

Taking part in the study lasts for up to two years (24 monthly cycles). During the first month, you will take fedratinib tablets once a day by mouth on their own. This helps your doctor check how well your body handles the medicine. In the second month, you will begin adding ropeginterferon alfa-2b, which is an injection you give yourself under the skin. The dose of the injection will start low and be gently adjusted depending on your blood tests and side effects.

You will need to attend regular hospital appointments for check-ups. These visits will include physical examinations, regular blood tests, checks of your vitamin B1 (thiamine) levels, and quality of life questionnaires. You will also have ultrasound scans of your tummy every six months to measure your spleen, as well as routine bone marrow checks to see how your disease is responding.

Potential risks and benefits

There is no guarantee that taking part will improve your condition, but the information gathered may help improve treatments for people with myelofibrosis in the future. You will also receive very close medical care and monitoring throughout the trial. However, both medicines can cause side effects. The main risk is a further drop in your blood counts, which can cause anaemia, bruising, or an increased risk of serious infections. If you develop a fever during the study, you must seek medical help immediately. Taking part is completely voluntary, and you are free to leave the study at any time without it affecting your standard NHS care.

Locations (12)

Belfast Health and Social Care Trust
City only
Belfast, Northern Ireland
NHS Greater Glasgow and Clyde
City only
Glasgow, Scotland
John Radcliffe Hopsital
City only
Oxford, England
Queen's Medical Centre
Approximate
Nottingham, England
Queen Elizabeth Medical Centre
City only
Birmingham, England
Northern General Hospital
Unverified
Sheffield, England
Southampton General Hospital
Unverified
Southampton, England
St James's University Hospital
Unverified
Leeds, England
The Christie NHS Foundation Trust
Unverified
Manchester, England
University College London Hospitals NHS Foundation Trust
Unverified
London, England
Cardiff & Vale University Lhb
Unverified
Cardiff, Wales
Guy's and St Thomas' NHS Foundation Trust
Unverified
London, England

Common questions

How are the medicines taken?

Fedratinib is taken by mouth as a daily tablet. Ropeginterferon alfa-2b is given as an injection under the skin, which you or a carer can be trained to give at home.

Will this study cure my myelofibrosis?

No, this trial is not expected to cure the disease. The goal is to see if the combination can safely shrink your spleen, improve symptoms, and slow down the cancer.

What should I do if I get a fever during the study?

A high temperature can be a sign of a serious infection due to low white blood cells. You will be given clear instructions to contact the medical team and seek urgent hospital treatment right away.

Do I have to pay for the study treatments?

No. The trial medicines and all study-related tests are provided free of charge through the research team.

Can I leave the trial if I change my mind?

Yes, you can leave the study at any point without giving a reason. Your regular doctors will support you and continue your usual medical care.

How to find out more

Catherine Thomas

FEDORA@trials.bham.ac.uk +44 121 371 7863 Official trial record

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