CompletedPhase IIInterventional

A comparison of response to treatment in patients with Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) Overlap Syndromes taking ASTX727 versus best supportive care

MDS/MPN overlap syndromes are rare blood cancers where the bone marrow cannot make healthy blood cells properly. This can cause severe tiredness, frequent infections, and easy bruising. Right now, there are very few treatments that directly target these diseases. This trial compares standard supportive care with a newer tablet called ASTX727. Similar existing medicines usually have to be given as regular injections in hospital, but ASTX727 can be taken by mouth at home. Researchers want to see if ASTX727 can help people live longer, improve their blood counts, and offer a better quality of life. Half of the people taking part will receive standard NHS supportive care (such as blood transfusions and symptom relief), while the other half will take the ASTX727 tablets. Both groups will have regular hospital check-ups so doctors can closely monitor their health.

At a glance

Status

Completed

Phase

Phase II

Sponsor

University of Birmingham

Enrolment target

Start

31 May 2022

Estimated completion

22 May 2024

Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) Overlap Syndromes

What is this study about?

MDS/MPN overlap syndromes are rare types of blood cancer that affect the bone marrow—the spongy centre inside your bones where all blood cells are made. In these conditions, the bone marrow does not work properly: it struggles to make normal red cells and platelets, while often producing far too many abnormal white blood cells. This imbalance can leave people feeling exhausted, prone to catching infections, and at risk of unusual bleeding.

At present, effective treatment options for these syndromes are very limited. Most patients receive standard supportive care, which focuses on managing symptoms using blood transfusions or gentle tablets to lower high cell counts. While an injectable drug called azacitidine can sometimes help, it requires frequent hospital trips for injections under the skin and is not widely available for all types of overlap syndromes.

This study is testing a newer drug called ASTX727. It works by switching on genes that help blood cells develop properly, but it comes as a simple tablet taken at home. Researchers want to learn whether ASTX727 can slow down the disease, improve daily symptoms, and help people live longer compared with standard care alone.

Key takeaways

The trial compares a new oral tablet (ASTX727) against standard supportive care for MDS/MPN overlap syndromes.
ASTX727 is taken at home for 5 days out of every 28-day cycle.
Participants are randomly allocated by a computer to one of the two treatment groups.
Regular hospital appointments, blood tests, and bone marrow checks are required to monitor safety.
Joining the study is completely optional, and you can withdraw at any point.

Who may be eligible?

This trial is open to adults aged 18 and over who have been diagnosed with an MDS/MPN overlap syndrome that needs active medical treatment. This typically includes people experiencing troublesome symptoms such as severe anaemia, low platelets, high white blood cell counts, an enlarged spleen, or higher numbers of abnormal cells in their bone marrow.

To join, you must be well enough to carry out daily activities and travel to scheduled hospital appointments. You will also need blood tests showing that your liver and kidneys are working well enough to process the study medication safely.

You cannot take part if you have already received intensive chemotherapy or similar gene-targeting drugs in the past. Your hospital specialist will review your full medical history and perform standard blood tests to confirm whether this study is safe and appropriate 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.

I am aged 18 or older.
I have a confirmed diagnosis of an MDS/MPN overlap syndrome.
My condition causes symptoms or low blood counts that require medical management.
I have not previously had intensive chemotherapy or similar hypomethylating drugs.
I am well enough to attend regular monthly hospital visits.

Answer every question to see your result.

What does participation involve?

If you join the study, a computer will place you at random into one of two groups. One group receives best supportive care, which is the usual NHS treatment tailored to your symptoms (such as blood transfusions, antibiotics, or gentle medicines). The other group takes ASTX727 tablets once a day for the first 5 days of each 28-day cycle, for up to 6 cycles (around 6 months). If the tablet is helping you, you may be able to continue taking it for longer.

Taking part involves visiting the hospital on day 1 of each 4-week cycle (plus one extra visit in the first month) for health checks and blood tests. You will also have a bone marrow test at months 3 and 6 to see how the disease is responding. After treatment finishes, the research team will check on your health once a year until the study ends.

Potential risks and benefits

There is no guarantee that taking ASTX727 will work better than standard care, but it may help improve your blood counts and control your disease with fewer hospital visits. Like all cancer treatments, ASTX727 can cause side effects such as nausea, tiredness, or a temporary drop in blood counts that could increase your risk of infections or bruising. Your medical team will monitor you very closely and can adjust your treatment if you feel unwell. Taking part is entirely voluntary, and you are free to leave the trial at any time without your normal NHS care being affected.

Locations (13)

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

The Christie NHS Foundation Trust
City only
Manchester, England
Oxford University Hospitals NHS Foundation Trust
City only
Oxford, England
University College London Hospitals NHS Foundation Trust
City only
London, England
University Hospitals Birmingham NHS Foundation Trust
City only
Birmingham, England
NHS Greater Glasgow and Clyde
City only
Glasgow, Scotland
Belfast Health and Social Care Trust
Unverified
Belfast, Northern Ireland
Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Unverified
Nottingham, England
Sheffield Teaching Hospitals NHS Foundation Trust
Unverified
Sheffield, England
University Hospital Southampton NHS Foundation Trust
Unverified
Southampton, England
Leeds Teaching Hospitals NHS Trust
Unverified
Leeds, England
Cardiff & Vale University Lhb
Unverified
Cardiff, Wales
NHS Grampian
Unverified
Aberdeen, Scotland

Common questions

Will I definitely receive the new tablet?

Not necessarily. A computer randomly assigns you to either receive ASTX727 or standard supportive care, giving everyone a fair and equal chance.

Can I take ASTX727 at home?

Yes. ASTX727 is an oral tablet taken once a day for the first five days of every 28-day cycle, meaning you do not need injections in hospital.

Do I have to pay for the study medicine?

No. The trial medication is provided free of charge through the study, which is funded by Blood Cancer UK.

What happens if my condition gets worse during the trial?

Your safety comes first. If the treatment is not helping or your disease progresses, your doctor will stop the trial treatment and discuss other care options with you.

Can I change my mind after starting?

Yes. You have the right to withdraw from the study at any time, for any reason, and your medical team will continue to support you with the best available standard care.

How to find out more

Louise Hopkins

AMMO@trials.bham.ac.uk +44 121 371 7861 Official trial record

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