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AuthorisedTherapeutic exploratory (Phase II)Interventional

A Phase 2 Open-Label Study to Evaluate Momelotinib in combination with Luspatercept in Participants with Transfusion Dependent Primary or Secondary Myelofibrosis

This clinical study, called a Phase 2 trial, is for people in the UK who have myelofibrosis, a condition that affects the bone marrow, and rely on regular blood transfusions. Researchers are testing a combination of two medicines, Momelotinib and Luspatercept (also known as Reblozyl). The main aim is to find out how many people can stop needing blood transfusions for at least 12 weeks after taking these medicines. The study is also looking at the safety of the combination, checking for any side effects and how the body handles the drugs. Participants must be 18 or older and can be male or female. This is an early-stage study to explore the potential benefits of this new treatment approach.

At a glance

Status
Authorised
Phase
Therapeutic exploratory (Phase II)
Sponsor
Glaxosmithkline Research & Development Limited
Enrolment target
51
Start
25 Mar 2025

What is this study about?

This study is for people in the UK who have a type of bone marrow disorder called myelofibrosis. Myelofibrosis can cause problems with how your body makes blood cells, often leading to anaemia, which means you don't have enough healthy red blood cells. To deal with this, many people with myelofibrosis need regular blood transfusions to top up their red blood cells. These transfusions can be time-consuming and impact daily life.

Researchers are interested in a new approach combining two medicines: Momelotinib and Luspatercept (also known as Reblozyl). Luspatercept is already approved for some other blood disorders, and Momelotinib is a newer medicine being investigated. The hope is that by using them together, they can help improve the body's ability to make its own red blood cells, reducing or even stopping the need for transfusions.

The main thing the researchers want to find out is how many people can stop needing blood transfusions for at least 12 weeks. They will also be closely watching for any side effects, how well the body tolerates the medicines, and any changes in general health or blood tests. This is an exploratory study, meaning it's an early step to understand if this combination could be a good treatment option in the future.

Key takeaways

  • This study is for adults with myelofibrosis who need regular blood transfusions.
  • It tests a combination of two drugs, Momelotinib and Luspatercept (Reblozyl).
  • The main goal is to see if the treatment can help people stop needing transfusions.
  • The study also looks at how safe the drug combination is and any side effects.
  • Participation involves regular clinic visits for blood tests and health checks.
  • You can stop taking part in the study whenever you want to.

Who may be eligible?

To join this study, you would need to be an adult, 18 years old or older. The study is open to both men and women.

The most important thing is that you must have a diagnosis of myelofibrosis, either primary (meaning it started on its own) or secondary (meaning it developed after another bone marrow condition). Additionally, you need to be dependent on blood transfusions, meaning you regularly require them to manage your condition.

There will be other specific medical checks to make sure the study is safe and suitable for you, but these are the main starting points. Your doctor will be able to tell you if you meet all the criteria.

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.

  1. Are you 18 years old or older?
  2. Do you have a diagnosis of myelofibrosis?
  3. Do you currently require regular blood transfusions for your myelofibrosis?
  4. Are you able to attend regular clinic appointments and follow study instructions?
Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the new drug combination of Momelotinib and Luspatercept (Reblozyl). This would involve taking the medicines as instructed by the study team. You would have regular visits to the clinic for check-ups, which might include blood tests, measuring your vital signs (like blood pressure and heart rate), and physical examinations. The study will particularly focus on whether you continue to need blood transfusions.

The main part of the study will last for 24 weeks, during which they will closely monitor your health and how the treatment is working. There will likely be follow-up appointments after this main period to keep an eye on your progress. The total duration of your participation would be explained in full detail by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that the combination of Momelotinib and Luspatercept might help reduce or even stop your need for blood transfusions, improving your quality of life. However, like all medicines, these drugs can have side effects, which could range from mild to more serious. The study team will carefully monitor you for any side effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    France
  • Unverified
    Italy
  • Unverified
    Germany
  • Unverified
    Spain

Common questions

What is myelofibrosis?

Myelofibrosis is a rare chronic condition where your bone marrow, which usually makes blood cells, becomes scarred and doesn't work properly.

What does 'transfusion dependent' mean?

It means you regularly need blood transfusions to help your body get enough red blood cells because your own bone marrow isn't producing them adequately.

Are the medicines in this study new?

Yes, Momelotinib is a newer medicine being tested, and Luspatercept (Reblozyl), while used for other conditions, is new in combination for myelofibrosis in this study.

How long will I be in the study?

The main part of the study where your treatment and progress are closely monitored will last for 24 weeks. There may be follow-up after this.

Can I stop participating if I want to?

Yes, you are completely free to withdraw from the study at any time, for any reason, without it affecting your medical care.

How to find out more

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

Discussion

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