A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment
This study is for people in the UK with a rare type of bone marrow cancer called myelofibrosis, specifically those with more serious forms of the condition (intermediate-2 or high-risk) who haven't seen enough improvement from a common type of treatment called a JAK-inhibitor. The main goal is to find out if a new medication, called imetelstat, helps people live longer compared to standard care options. Participants will either receive imetelstat or the best available therapy, and researchers will carefully monitor their health outcomes. This research aims to offer a new treatment option for those whose current treatments are no longer effective.
At a glance
What is this study about?
This study is looking into a new medication called imetelstat for a rare blood cancer called myelofibrosis. Myelofibrosis affects your bone marrow, where blood cells are made, causing scar tissue to build up. This can lead to various symptoms like tiredness, a swollen tummy from an enlarged spleen, and different blood problems. For some people, a common treatment called a JAK-inhibitor doesn't work well enough or stops working over time. This study is specifically for these individuals, to see if imetelstat could be a better option.
The main aim of this research is to compare imetelstat with other existing treatments to see if it helps people with myelofibrosis live longer. It’s important to find new and effective treatments, especially for those who haven't benefited from previous therapies. By taking part in this study, you would be helping doctors understand if imetelstat can make a real difference in the lives of people living with myelofibrosis.
Participants will be placed into one of two groups: one receiving imetelstat, and the other receiving what doctors consider to be the 'best available therapy' for their condition. Researchers will then carefully track how well these treatments work, focusing on how long people live. This helps gather important information to improve future care for myelofibrosis. Throughout the study, your health will be closely monitored by a medical team.
Key takeaways
- Tests a new drug (imetelstat) for myelofibrosis.
- For adults who haven't responded to JAK-inhibitor treatments.
- Compares imetelstat to current standard care.
- Aims to see if imetelstat improves how long people live.
- Participation involves screening, treatment, and follow-up.
- You can stop participating at any time.
Who may be eligible?
This study is looking for adults aged 18 or older who have been diagnosed with myelofibrosis. This includes people who developed myelofibrosis initially, or after having other blood conditions like essential thrombocythemia or polycythemia vera.
Crucially, you must have a more serious form of myelofibrosis, rated as 'intermediate-2' or 'high-risk'. You also need to have tried a JAK-inhibitor treatment for at least three to six months but haven't seen enough improvement, or your condition has worsened while on it. This means your spleen might not have shrunk enough, your symptoms haven't improved significantly, or your condition has progressed.
It's important that you are not suitable for a stem cell transplant, as this is a different treatment option. The study aims to help those who have run out of other effective treatment options.
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 old or over?
- Have you been diagnosed with myelofibrosis (primary, post-ET or post-PV)?
- Is your myelofibrosis considered 'intermediate-2' or 'high-risk'?
- Have you previously tried a JAK-inhibitor treatment for at least 3-6 months but it's not working well enough or your condition got worse?
- Are you not currently eligible for an allogeneic stem cell transplant?
What does participation involve?
If you decide to join this study, your journey will happen in a few stages. First, there's a 'screening phase' of up to 28 days. During this time, your medical team will check if you meet all the study requirements. You'll also need to pause any previous treatments, including JAK-inhibitors, for 14 days before starting the study.
Once eligible, you'll be randomly assigned to one of two groups. Two out of three people will receive the new medication, imetelstat, while one out of three will receive the 'best available therapy' – which is the standard treatment your doctor recommends. You'll receive your assigned treatment until it's no longer helping or you decide to stop.
After you stop taking the study treatment, there's a 'follow-up' phase. This means the study team will continue to check in on your health regularly, which could involve appointments, until the study ends, or your situation changes. If you are in the 'best available therapy' group and your condition worsens, there might be an option to switch to imetelstat, if approved by the study sponsor. The total duration of your participation will depend on how you respond to treatment and the overall length of the study.
Potential risks and benefits
Locations (174)
- University of California-San Diego/Moores UCSD Cancer CenterVerified postcodeLa Jolla, United States
- UCLA David Geffen School of MedicineVerified postcodeLos Angeles, United States
- Smilow Cancer Center at YNHHVerified postcodeNew Haven, United States
- BRCR Medical Center IncVerified postcodePlantation, United States
- University of South FloridaVerified postcodeTampa, United States
- Norton Cancer InstituteVerified postcodeLouisville, United States
- Ochsner Clinic FoundationVerified postcodeNew Orleans, United States
- Maryland Oncology HematologyVerified postcodeRockville, United States
- Icahn School of Medicine at Mount SinaiVerified postcodeNew York, United States
- Duke University Medical CenterVerified postcodeDurham, United States
- Gabrail Cancer CenterVerified postcodeCanton, United States
- Prairie Lakes Health Care System, Inc.Verified postcodeWatertown, United States
Common questions
What is myelofibrosis?
Myelofibrosis is a rare type of chronic blood cancer where scar tissue forms in the bone marrow, affecting the production of healthy blood cells.
What is a JAK-inhibitor?
JAK-inhibitors are a type of medicine commonly used for myelofibrosis to help manage symptoms and reduce spleen size. This study is for people who haven't responded well to them.
What does 'randomised' mean?
Being 'randomised' means you'll be put into a treatment group by chance, like flipping a coin, to make sure the study results are fair and unbiased. You won't get to choose your group.
What is 'best available therapy'?
This refers to the standard treatments that doctors would typically recommend for your myelofibrosis condition outside of a clinical trial.
Can I stop participating in the study if I want to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual 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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