Bexmarilimab + Azacitidine Versus Placebo + Azacitidine in Participants With Treatment-naïve Higher-risk Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) are conditions where the bone marrow struggles to produce healthy blood cells. In higher-risk MDS, there is a greater chance of infections, severe tiredness, or the condition turning into leukaemia. The usual first treatment is a medicine called azacitidine. This trial investigates whether adding a new antibody drug, bexmarilimab, helps azacitidine work better. Bexmarilimab is designed to help the immune system clear abnormal cells. Participants are randomly placed into groups. One group receives standard azacitidine plus a dummy treatment (placebo). The other groups receive azacitidine plus bexmarilimab at either a lower or higher dose. Doctors will check blood counts, assess how well the bone marrow responds, and look closely at safety. This study aims to help researchers find the best dose and understand if this combination improves treatment for people newly diagnosed with higher-risk MDS.
At a glance
What is this study about?
Inside our bones is a spongy tissue called bone marrow, which acts like a factory producing red blood cells, white blood cells, and platelets. In myelodysplastic syndromes (MDS), this factory produces abnormal, immature cells rather than healthy, working blood cells. When MDS is graded as 'higher-risk', it means the condition is more aggressive, carries a greater chance of causing severe blood shortages, and has a higher risk of developing into acute myeloid leukaemia.
For many people with higher-risk MDS who cannot have an immediate stem cell transplant, the standard medical care is a medicine called azacitidine. While azacitidine can improve blood counts and delay disease progression, doctors are searching for newer treatments that can provide deeper and longer-lasting control.
This trial looks at an experimental drug called bexmarilimab. It is an antibody designed to target a specific protein on immune cells, potentially waking up the body's natural defences so they can target and clear abnormal blood cells. In this trial, researchers are testing two different doses of bexmarilimab given together with azacitidine, comparing them against azacitidine given with a placebo (a dummy drug).
By comparing these groups, doctors hope to determine the safest and most effective dose of bexmarilimab. The overarching goal is to see if adding this new drug can help patients live longer, improve their blood counts, and reduce the need for blood transfusions.
Key takeaways
- Tests a new antibody drug, bexmarilimab, combined with standard azacitidine.
- Aimed at adults newly diagnosed with higher-risk MDS who have not yet had treatment.
- Compares two different doses of bexmarilimab against a dummy treatment (placebo).
- All participants receive the standard active drug, azacitidine.
- Participation involves regular hospital visits, blood tests, and bone marrow checks.
- Joining is completely voluntary, and you can withdraw at any point.
Who may be eligible?
This trial is for adults aged 18 and over who have recently been diagnosed with higher-risk MDS and have not yet received treatment for it. To take part, you must be well enough to carry out everyday activities, have reasonable kidney and liver health, and be suitable to receive standard azacitidine treatment.
You cannot take part if your condition has already developed into acute myeloid leukaemia (AML) or certain other rare, overlapping blood disorders. People who are ready to have an immediate stem cell transplant will also not be eligible, as this study focuses on medical drug therapy rather than transplants.
Because of the potential effects of the medications on an unborn baby, women who could become pregnant must have a negative pregnancy test before starting. Both men and women taking part must agree to use reliable contraception throughout the study and for at least six months after finishing treatment.
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 newly diagnosed with higher-risk MDS.
- I have not yet received treatment for my MDS.
- I am not scheduled to have an immediate stem cell transplant.
- My kidney and liver tests show reasonably healthy function.
- I am able to attend regular hospital appointments for check-ups and treatment.
What does participation involve?
Taking part involves regular visits to the hospital clinic. Azacitidine is usually given as an injection under the skin or into a vein over several days at the start of each treatment cycle. Bexmarilimab (or the placebo) is given as a drip into a vein. During your visits, the medical team will carry out physical checks, run regular blood tests, and periodically perform bone marrow biopsies to see how your condition is responding. You will also be asked about any side effects or changes in how you feel. Treatment continues for as long as it helps manage the condition and remains safe for you, followed by regular check-ups to track your long-term health.
Potential risks and benefits
Locations (35)
- City of Hope Medical CenterVerified postcodeDuarte, United States· Not yet recruiting
- University of Colorado Cancer CenterVerified postcodeAurora, United States· Not yet recruiting
- Yale Cancer CenterVerified postcodeNew Haven, United States· Not yet recruiting
- Moffitt Malignant Hematology & Cellular Therapy at Memorial Healthcare SystemVerified postcodePembroke Pines, United States· Not yet recruiting
- Mass General Brigham Cancer InstituteVerified postcodeBoston, United States· Not yet recruiting
- University of North Carolina Lineberger Comprehensive Cancer CenterVerified postcodeChapel Hill, United States· Not yet recruiting
- Vanderbilt University Medical CenterVerified postcodeNashville, United States· Not yet recruiting
- The University of Texas MD Anderson Cancer CenterVerified postcodeHouston, United States· Not yet recruiting
- Medical College of Wisconsin - Clinical Cancer Center - Froedtert HospitalVerified postcodeMilwaukee, United States· Not yet recruiting
- Fakultní nemocnice BrnoVerified postcodeBrno, Czechia· Not yet recruiting
- Fakultní nemocnice Hradec KrálovéVerified postcodeHradec Králové, Czechia· Not yet recruiting
- Fakultni nemocnice OstravaApproximateOstrava-Poruba, Czechia· Not yet recruiting
Common questions
What is a placebo?
A placebo looks identical to the real medicine but contains no active ingredients. It helps researchers fairly check whether the new drug is truly responsible for any improvements or side effects.
Will I definitely receive bexmarilimab?
No. A computer randomly decides whether you receive bexmarilimab (at one of two doses) or the placebo. Everyone in the trial receives the active standard medicine, azacitidine.
How is the study medicine given?
Azacitidine is given either as injections under the skin or into a vein. Bexmarilimab (or the placebo) is given as an infusion drip directly into a vein at the clinic.
Can I leave the study if I change my mind?
Yes. You have the right to withdraw from the trial at any time. Doing so will not affect your relationship with your healthcare team or your access to standard NHS care.
Does taking part cost anything?
No. The study medications and trial-related tests are provided free of charge, and some trials offer support for travel expenses to hospital visits.
How to find out more
Joab Williamson, PhD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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