A Phase 2 Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination with Ruxolitinib in Subjects with Myelofibrosis (REFINE)
This research study is for people who have a blood condition called myelofibrosis. In this condition, the bone marrow, which makes blood cells, doesn't work properly, and the spleen can become very large. The study is testing a new medicine called Navitoclax. Researchers want to find out how safe Navitoclax is and how well it works to help shrink an enlarged spleen and make people feel better. They are trying Navitoclax on its own for some people, and for others, they are giving it along with another medicine called Ruxolitinib. Doctors will check the size of the spleen using scans like MRI or CT, and they will also monitor how people's symptoms change over time.
At a glance
What is this study about?
Myelofibrosis is a condition where the spongy tissue inside your bones, called bone marrow, becomes scarred. This scarring makes it hard for your body to make enough healthy blood cells. When this happens, other organs, especially your spleen, often have to work harder, which can make it grow much larger than normal. This can lead to various uncomfortable symptoms like tiredness, bone pain, and feeling full quickly.
This study, called REFINE, is looking into a new treatment called Navitoclax. We want to see if Navitoclax can help shrink an enlarged spleen in people with myelofibrosis. We are also interested in finding out if it can make people feel better by reducing their symptoms. Some people in the study will receive Navitoclax on its own, while others will take it alongside another medicine already used for myelofibrosis, called Ruxolitinib. By comparing these approaches, doctors hope to understand the best way to use Navitoclax.
The main goal is to see if the spleen shrinks by at least 35% after 24 weeks of treatment. This will be checked using scans like MRI or CT. We will also be carefully monitoring other important things, like whether people's overall symptoms improve significantly, if their red blood cell counts get better (which helps with tiredness), and if there are any changes in the scarring in their bone marrow. The information from this study will help doctors learn more about how to treat myelofibrosis effectively.
Key takeaways
- Exploring a new medicine, Navitoclax, for myelofibrosis.
- Aims to shrink an enlarged spleen and reduce symptoms.
- Tests Navitoclax alone or with current treatment (Ruxolitinib).
- Participation involves regular clinic visits, tests, and scans.
- Potential benefits include access to new treatment, but risks of side effects exist.
- Your decision to join is completely voluntary and can be changed.
Who may be eligible?
To be able to join this study, participants need to be at least 18 years old. There is no upper age limit, meaning older adults can also take part. The study is open to both men and women.
Beyond age and sex, there are specific medical details that researchers consider. You would need to have been diagnosed with myelofibrosis. The study is particularly looking for people whose spleen is enlarged, as reducing its size is a key aim of the treatment being tested.
Your doctor would carefully review your full medical history, including any other health conditions you might have or other medicines you are taking, to make sure this study is suitable and safe for you. It's important that your health is stable enough to participate in a clinical trial.
- Are you 18 years old or older?
- Have you been diagnosed with myelofibrosis?
- Do you have an enlarged spleen?
- Are you willing and able to attend regular clinic appointments and have medical tests?
- Are you generally in stable health otherwise?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would first have a full health check-up to make sure it's right for you. If you qualify, you would regularly take the study medicine, either Navitoclax alone or with Ruxolitinib, as instructed by the study team. You would have regular visits to the clinic, which might include blood tests, health checks, and discussions about how you're feeling and any side effects you might be having.
About 24 weeks (roughly 6 months) into the study, you would have an MRI or CT scan to measure your spleen size. You would also complete questionnaires about your symptoms to see if they have improved. These check-ups and assessments will continue throughout the study to monitor your health and how the treatment is working. The exact number of visits and the total length of your participation would be explained in detail before you start.
Potential risks and benefits
Locations (4)
- —Spain
- —Italy
- —Greece
- —Croatia
Common questions
What is myelofibrosis?
Myelofibrosis is a condition where your bone marrow, which makes blood cells, becomes scarred, leading to problems with blood cell production and an enlarged spleen.
What is Navitoclax?
Navitoclax is a new medicine being studied to see if it can help shrink an enlarged spleen and improve symptoms in people with myelofibrosis.
What is Ruxolitinib?
Ruxolitinib is a medicine already used to treat myelofibrosis, and in this study, it's being tested alongside Navitoclax for some participants.
How will progress be measured?
Doctors will use scans (MRI/CT) to measure changes in spleen size and use symptom questionnaires to see if your feelings of discomfort improve.
Can I stop participating in the study?
Yes, you can choose to leave the study at any time, and this will not affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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