Assessing the safety and tolerability of oral ruxolitinib in combination with 5-azacitidine in patients with advanced phase myeloproliferative neoplasms (MPN), including myelodysplastic syndromes (MDS) or acute myeloid leukaemia (AML) arising from MPN.
Myeloproliferative neoplasms (MPNs) are rare blood conditions where the bone marrow makes too many blood cells. Over time, these conditions can become more serious and develop into advanced disorders or leukaemia. When this happens, doctors need better and safer treatment options. This Phase 1 study tests the combination of two drugs: ruxolitinib (taken as a tablet) and azacitidine (given by injection). Both medicines are already used to treat certain blood problems, but researchers want to know if combining them is safe and manageable for patients with advanced MPNs. Patients aged 16 and over with advanced MPN may be eligible. Some people will receive the drug combination, while others who cannot or prefer not to take the trial drugs can join an observational group to help doctors monitor their health and learn more about the condition.
At a glance
What is this study about?
Myeloproliferative neoplasms (MPNs) are a group of chronic conditions affecting the bone marrow, the spongy centre inside your bones where blood cells are made. In MPNs, such as polycythaemia vera, essential thrombocythaemia, or myelofibrosis, the body produces too many blood cells. For some people, the condition can slowly become more active and develop into an advanced phase, known as myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML). During this shift, the bone marrow starts producing higher numbers of immature cells called 'blasts'.
When MPNs enter this advanced phase, standard treatments often become less effective. Doctors are keen to explore newer drug combinations that might help control the disease more successfully and relieve difficult symptoms. This trial investigates whether combining two existing medications—ruxolitinib and azacitidine—is safe and well tolerated when taken together.
Ruxolitinib works by blocking certain signals inside blood cells that tell them to divide and grow out of control. Azacitidine works differently, by helping to switch on genes that stop abnormal cells from growing, encouraging them to mature or die off. While both drugs are already used separately for blood disorders, combining them may offer a stronger way to tackle advanced disease.
Because this is an early-stage (Phase 1) trial, the primary focus is finding out what side effects happen, what dose is safe, and how patients feel during treatment. The study also includes an observational group, which tracks people who are receiving other standard treatments, helping researchers compare outcomes and understand how advanced MPN affects patients over time.
Key takeaways
- Tests the safety of combining ruxolitinib and azacitidine for advanced MPNs.
- Open to patients aged 16 and over with ET, PV, or myelofibrosis that has progressed.
- A Phase 1 study focused on safety, side effects, and finding the right dose.
- Requires hospital visits, blood tests, and bone marrow checks.
- Offers an observational option for people who cannot or choose not to take the trial drugs.
- Taking part is entirely voluntary and you can stop at any time.
Who may be eligible?
You may be able to take part if you are 16 or older and have previously been diagnosed with an MPN, such as essential thrombocythaemia, polycythaemia vera, or myelofibrosis. To join the treatment group, tests must show that your condition has progressed to an advanced phase, meaning that at least 10% of the cells in your blood or bone marrow are immature blast cells.
You will also need to be reasonably active and able to look after yourself, with acceptable kidney and liver function on standard blood tests. Because the study team needs to check how the treatment affects your bone marrow and daily life, you must be willing to have routine bone marrow tests and fill out simple questionnaires about your symptoms.
If you do not meet all the health requirements to receive the study medicines, or if you prefer not to take them, you might still be able to join the observational group. This allows your medical team to follow your care and learn from your health journey without changing your chosen treatment plan.
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 16 years of age or older?
- Have you been diagnosed with an MPN (such as ET, PV, or myelofibrosis)?
- Has your specialist told you that your blood disorder has reached an advanced stage?
- Are your liver and kidneys working reasonably well?
- Are you able and willing to attend hospital appointments and undergo bone marrow tests?
What does participation involve?
If you take part in the treatment group, you will take ruxolitinib tablets by mouth at home and receive azacitidine as an injection under the skin or into a vein at the hospital on scheduled days. You will need to attend regular hospital appointments for check-ups, blood tests, and health reviews so the doctors can monitor how you are doing. The team will perform bone marrow biopsies at set points during the study to check how the disease is responding, and you will be asked to fill in regular questionnaires about your symptoms and quality of life. The treatment continues as long as it helps you and any side effects remain manageable, followed by a period where the team keeps track of your health.
Potential risks and benefits
Locations (15)
- Beatson West of Scotland Cancer CentreCity onlyGlasgow, Scotland
- Belfast City HospitalApproximateBelfast, Northern Ireland
- The ChristieApproximateManchester, England
- Churchill HospitalApproximateOxford, England
- Hammersmith Hospital LaboratoryCity onlyLondon, England
- Nottingham City HospitalUnverifiedNottingham, England
- University Hospital BirminghamUnverifiedBirmingham, England
- Southampton General Hospital LaboratoryUnverifiedSouthampton, England
- St James's University Hosp LaboratoryUnverifiedLeeds, England
- University Hospital of WalesUnverifiedCardiff, Wales
- St. Bartholomews HospitalUnverifiedLondon, England
- Guy's and St Thomas' NHS Foundation TrustUnverifiedLondon, England
Common questions
What is the purpose of a Phase 1 clinical trial?
A Phase 1 trial is an early-stage study designed mainly to check if a new medicine or drug combination is safe, to see what side effects it causes, and to find the best dose to use.
Will taking part cure my blood condition?
This study is not expected to cure the condition. The aim is to find out if these medicines can safely control the disease, improve symptoms, and slow down its progression.
How are the medicines given?
Ruxolitinib is a tablet you swallow at home. Azacitidine is given as an injection, either just under the skin or through a drip into a vein, which is done during your hospital visits.
What is the observational part of the study?
The observational group is for people who cannot or choose not to take the trial medicines. Instead of receiving the study drugs, you continue your standard care while researchers collect health information to better understand advanced MPNs.
Can I leave the study if I feel it is not right for me?
Yes. You have the right to withdraw from the trial at any time, for any reason. Doing so will not affect your relationship with your medical team or the quality of your regular NHS care.
How to find out more
Sonia Fox
Always speak to your GP or specialist before deciding to take part in a study.
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