RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Roginolisib in Combination With Ruxolitinib in Patients With Myelofibrosis (MF) Who Are Unresponsive to JAK Inhibitors

This research study is for adults living with myelofibrosis, a type of blood cancer, who are already taking ruxolitinib but haven't seen enough improvement. Researchers want to see if adding an experimental drug called roginolisib to ruxolitinib is safe and if people can tolerate it well. This is an early-stage study, meaning it's one of the first times this drug combination is being tested in a larger group of patients. The main goal is to understand any side effects and how the body handles the new medication. This information is crucial for deciding if this treatment should be studied further.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

iOnctura

Enrolment target

Start

17 Nov 2025

Estimated completion

01 Jul 2028

Myelofibrosis (MF)

What is this study about?

This study is looking into a new treatment for a condition called myelofibrosis. Myelofibrosis is a rare type of blood cancer where the bone marrow, which makes blood cells, becomes scarred. This can lead to problems like a swollen spleen, tiredness, and other symptoms.

Many people with myelofibrosis are already treated with a medicine called ruxolitinib. However, for some individuals, ruxolitinib doesn't work as well as hoped, or its effects might wear off over time. This study is designed for those patients. Researchers want to see if adding an experimental drug called roginolisib to ruxolitinib can help when ruxolitinib alone isn't enough.

The main aim of this study is to check the safety of combining roginolisib with ruxolitinib. It's like testing a new recipe – you want to make sure all the ingredients work well together and don't cause any unexpected problems. The study will also look at how well patients manage the new medicine and if they experience any side effects. This early research is important for finding better ways to help people with myelofibrosis.

Key takeaways

This study is for adults with myelofibrosis.
It tests a new drug, roginolisib, combined with your usual ruxolitinib.
It's for people whose myelofibrosis symptoms are not well controlled by ruxolitinib alone.
The main goals are to check the new drug combination's safety and how well people tolerate it.
Participation involves regular clinic visits and health checks.
You can stop participating at any time.

Who may be eligible?

To join this study, you must be at least 18 years old and have been diagnosed with myelofibrosis. This includes specific types like post-Polycythaemia Vera MF or post-Essential Thrombocythemia MF.

You must already be taking ruxolitinib for at least three months, with your dose stable for the past eight weeks. Importantly, your spleen should still be noticeably enlarged (at least 10 cm below your ribs) and not have shrunk much since starting ruxolitinib.

Additionally, you should be experiencing active symptoms of myelofibrosis, scoring 10 or more on a symptom questionnaire. Your general health should be good enough to take part in the study, and you can't have received most other myelofibrosis treatments in the last three months (other than ruxolitinib). Women who could become pregnant will need to use effective birth control throughout the study.

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 or older?
Do you have a diagnosis of myelofibrosis (MF, PPV-MF, or PET-MF)?
Have you been taking ruxolitinib for at least 3 months, with a stable dose for the past 8 weeks?
Is your spleen still enlarged and not shrunk much since starting ruxolitinib?
Are you currently having active myelofibrosis symptoms?
Are you able to visit the clinic regularly for check-ups and tests?

Answer every question to see your result.

What does participation involve?

This study will involve around 26 patients. It's split into two parts, with 13 patients in each part. In the first part, doctors will cautiously check the benefits and risks of the new drug combination. If that goes well, another 13 patients will join the second part to gather more information.

Throughout the study, you will take the experimental medicine, roginolisib, along with your usual ruxolitinib. You will have regular visits to the clinic for check-ups, blood tests, and other assessments to monitor your health and see how you are reacting to the treatment. The exact number of visits and tests will be explained by the study team, along with how long you will be taking the study medication. There will also be a follow-up period after you finish taking the study drugs.

Potential risks and benefits

Taking part in a study like this might offer some potential benefits, such as access to a new experimental treatment for your myelofibrosis that isn't yet widely available. However, there's no guarantee that the new drug will work for everyone, and it might not improve your condition. There are also potential risks involved, as new medicines can have side effects that are not yet fully known. The study team will carefully monitor you for any problems and explain all known risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (11)

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

Azienda Ospedaliero Universitaria Careggi Firenze, Struttura Complessa di Ematologia
Verified postcode
Florence, Italy· Recruiting
IRCCS Clinical Institute Humanitas
Verified postcode
Rozzano, Italy· Recruiting
Istituto di Ematolgia e Oncologia Medica
Verified postcode
Bologna, Italy· Recruiting
Azienda Sanitario Universitaria Friuli Centrale
Verified postcode
Udine, Italy· Recruiting
Vall d'Hebron University Hospital
Verified postcode
Barcelona, Spain· Recruiting
Avigunda Gran via de l'Hospitalet 199-203, 08908 L'Hospitalet de Llobregat
Verified postcode
Barcelona, Spain· Recruiting
START Madrid CIOCC
Verified postcode
Madrid, Spain· Not yet recruiting
Hospital Universitario de Salamanca
Verified postcode
Salamanca, Spain· Recruiting
United Lincolnshire Teach Hospitals NHS Trust, Pilgrim Hospital Boston
Verified postcode
Boston, United Kingdom· Not yet recruiting
Belfast City Hospital
Verified postcode
Belfast, United Kingdom· Not yet recruiting
Guy´s and St. Thomas NHS Foundation Trust
Verified postcode
London, United Kingdom· Not yet recruiting

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where scar tissue builds up in your bone marrow, affecting how your blood cells are made.

What is roginolisib?

Roginolisib is a new, experimental drug being tested to see if it can help people with myelofibrosis.

Why is this study being done?

This study aims to discover if adding roginolisib to ruxolitinib is safe for people whose myelofibrosis isn't fully controlled by ruxolitinib alone.

Will I stop taking my current medication?

No, you will continue to take ruxolitinib, and the new drug, roginolisib, will be added to it.

How long will the study last for me?

The full duration of your participation, including treatment and follow-up, will be explained by the study team if you are eligible.

How to find out more

Tracey Hammett, RN

t.hammett@ionctura.com +44 7776498978 Official trial record

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