A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of luspatercept in patients with lower-risk MDS and ringsideroblastic phenotype (MDS-RS)
Myelodysplastic syndromes (MDS) are conditions where the bone marrow struggles to make enough healthy blood cells. In a specific type called MDS with ring sideroblasts (MDS-RS), people often develop severe anaemia and need regular blood transfusions to stay well. This Phase III clinical trial tests an injection called luspatercept (brand name Reblozyl). The main goal is to find out if luspatercept can help patients go at least 24 weeks without needing a red blood cell transfusion. It also checks whether the treatment improves energy levels, daily mobility, and overall quality of life. Because this is an open-label study, every participant receives the real medicine rather than a dummy treatment (placebo). The trial is open to adults aged 18 and over with lower-risk MDS-RS.
At a glance
What is this study about?
Myelodysplastic syndromes (MDS) affect the bone marrow, the spongy centre of your bones where blood cells are produced. In people with lower-risk MDS with ring sideroblasts (MDS-RS), red blood cells do not mature properly. Because red blood cells carry oxygen around the body, having too few of them causes anaemia. This can leave you feeling constantly exhausted, breathless, weak, and dizzy. Many people need regular blood transfusions at hospital to manage these symptoms.
While blood transfusions are essential and life-saving, having them frequently can be difficult to fit into daily life. Over time, repeated transfusions can also cause iron to build up in your body, which can put strain on organs like your heart and liver. Finding treatments that encourage the bone marrow to produce its own healthy red blood cells is an important goal for doctors and researchers.
This study tests a medicine called luspatercept (Reblozyl). Luspatercept works by helping young red blood cells mature so they can enter the bloodstream normally. The researchers want to see how many people can become free from blood transfusions for 24 weeks or longer. They will also track whether people's haemoglobin levels rise, whether iron levels drop, and how the medicine affects everyday wellbeing.
This is an 'open-label' study, which means both you and your doctor will know you are receiving luspatercept; there is no dummy drug (placebo). Taking part helps researchers understand how well the medicine works in everyday practice and how safe it is over a longer period.
Key takeaways
- Tests luspatercept (Reblozyl) to see if it reduces the need for blood transfusions in lower-risk MDS-RS.
- Open to adults aged 18 and over with lower-risk MDS and ring sideroblasts.
- All participants receive the active medicine; there is no placebo.
- Given as an injection under the skin, usually once every three weeks for up to a year.
- Your health, blood counts, and quality of life will be closely tracked by specialists.
Who may be eligible?
This trial is designed for adults aged 18 and older who have been diagnosed with a specific type of blood condition called lower-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS). 'Ring sideroblasts' means that iron has built up in an unusual ring shape inside early red blood cells, which your specialist can identify from bone marrow tests.
It is mainly intended for people whose anaemia means they need regular blood transfusions, or who have not had enough benefit from other standard treatments. Both men and women can take part, provided they are well enough to attend hospital visits for monitoring and injections.
You may not be able to join if your MDS is considered higher-risk, if the condition shows signs of turning into acute leukaemia, or if you have other severe medical conditions (such as serious kidney or liver disease) that could make taking a new medicine unsafe. Your haematology team will carry out detailed blood and health checks to confirm 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.
- I am aged 18 or older.
- I have been diagnosed with lower-risk myelodysplastic syndrome (MDS).
- My MDS has ring sideroblasts (MDS-RS).
- I have anaemia and need regular red blood cell transfusions (or have low haemoglobin levels).
- I am able to attend regular hospital visits for injections and blood tests.
What does participation involve?
Taking part in this study lasts for at least one year (52 weeks), with possible follow-up visits afterwards. You will receive luspatercept as an injection given just under the skin (subcutaneously), usually once every three weeks at the hospital clinic. During your visits, the research team will take blood samples to check your blood counts and iron levels, check your blood pressure, and ask about any side effects. You will also be asked to fill out simple questionnaires about your daily wellbeing and energy, and you may complete a quick walking test (called the 'Timed Up and Go' test) to see how the treatment affects your mobility. If you need blood transfusions during the trial, these will still be given as required.
Potential risks and benefits
Locations (3)
- —UnverifiedSpain
- —UnverifiedAustria
- —UnverifiedGermany
Common questions
Will I receive a dummy drug (placebo) in this trial?
No. This is an open-label study, which means every person who joins will receive the active study medicine, luspatercept.
How is the medicine given?
Luspatercept is given as a small injection under the skin, similar to an insulin injection, usually once every three weeks at the hospital.
Will this treatment cure my MDS?
No, luspatercept is not a cure for MDS. It is designed to manage the condition by treating anaemia and helping you avoid regular blood transfusions.
Can I still have blood transfusions if I need them?
Yes. If your blood counts drop or you feel unwell, your medical team will provide blood transfusions just as they normally would.
Do I have to pay to take part?
No. Clinical trial treatments, tests, and study-related hospital visits are provided free of charge, and some travel expenses may be reimbursed.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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