Active not recruitingPHASE3INTERVENTIONAL

ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions

This study, called ELEMENT-MDS, focuses on adults who have a specific blood condition called Myelodysplastic Syndrome (MDS). These individuals often suffer from anaemia (low red blood cells) and feel tired, weak, or breathless, but they haven't yet reached a point where they need regular blood transfusions. The main goal of this research is to compare a new drug, Luspatercept, with a commonly used medicine, Epoetin Alfa. Researchers want to find out if Luspatercept is more effective and safer at improving anaemia in people with lower-risk MDS who haven't used similar medicines before. By doing this, they hope to find better ways to help patients manage their condition and improve their quality of life.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Bristol-Myers Squibb

Enrolment target

360

Start

24 Oct 2023

Estimated completion

11 Mar 2030

Myelodysplastic Syndromes

What is this study about?

Myelodysplastic Syndromes (MDS) are a group of conditions where the bone marrow, which is the spongy part inside your bones, doesn't produce enough healthy blood cells. This often leads to anaemia, a common problem where you don't have enough red blood cells to carry oxygen around your body. Symptoms of anaemia can include feeling very tired, weak, short of breath, or dizzy. For many people with MDS, their anaemia gets worse over time, and they might eventually need blood transfusions.

This study is looking for better ways to treat anaemia in people with MDS, specifically those with lower-risk forms of the disease who haven't yet needed blood transfusions and haven't used certain other anaemia medicines before. The study wants to see if a new medicine called Luspatercept works better than a standard treatment, Epoetin Alfa, in helping people produce more red blood cells and feel better.

The main aim is to compare how well these two medicines work and how safe they are. By finding effective treatments that can help people avoid or delay blood transfusions, researchers hope to improve the quality of life for those living with MDS.

Key takeaways

Targets adults with specific lower-risk Myelodysplastic Syndrome (MDS).
Focuses on anaemia treatment, especially for those not needing blood transfusions.
Compares a new medicine, Luspatercept, with a standard one, Epoetin Alfa.
Aims to improve anaemia symptoms and potentially delay blood transfusions.
Participation involves regular clinic visits for monitoring and treatment.
Your health and safety are carefully monitored throughout the study.

Who may be eligible?

To join this study, you need to be an adult (18 or older) with a confirmed diagnosis of a specific type of Myelodysplastic Syndrome (MDS). This type of MDS should be classified as very low, low, or intermediate-risk by doctors. You also need to have anaemia symptoms like fatigue or shortness of breath, but importantly, you should not be currently needing regular blood transfusions.

Another key requirement is that you haven't received certain anaemia-stimulating medicines before, or if you have, it was only a couple of doses and more than 8 weeks ago. Your blood tests will also need to show a certain level of a hormone related to red blood cell production, and your haemoglobin (red blood cell count) needs to be at a specific low level to qualify.

You cannot join if your MDS was caused by previous treatments like chemotherapy or radiation for other conditions, or if you have a history of another blood cancer called AML. You're also excluded if you've had a stroke or a mini-stroke (TIA).

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 an adult aged 18 or over?
Do you have a diagnosis of lower-risk Myelodysplastic Syndrome (MDS)?
Do you have symptoms of anaemia (like fatigue or shortness of breath)?
Are you currently NOT receiving regular blood transfusions?
Have you NOT had certain other anaemia medicines recently?
Do you NOT have a history of AML or strokes?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be assigned by chance to receive either the study medicine, Luspatercept, or the standard medicine, Epoetin Alfa. You won't get to choose which one you receive, similar to flipping a coin. You'll have regular visits to the clinic for check-ups, blood tests, and to receive your medication. The doctors will closely monitor how your anaemia is responding to the treatment and keep an eye on any side effects you might experience. They'll also ask you about your symptoms and how you're feeling. We don't have details on the exact number of visits, their frequency, or the total duration of the study in this summary, but these would be fully explained to you before you agree to participate.

Potential risks and benefits

Taking part in this study might offer potential benefits, such as receiving a new treatment that could improve your anaemia symptoms and potentially reduce your need for blood transfusions. However, as with any medicine, there are potential risks and side effects, which will be fully explained to you. It's also possible that the study medicine may not work for you, or you might experience side effects that outweigh any benefits. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (144)

Local Institution - 0070
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Clovis, United States
Local Institution - 0179
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Fountain Valley, United States
Local Institution - 0211
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Los Alamitos, United States
Local Institution - 0258
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Los Angeles, United States
Local Institution - 0247
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Orange, United States
Local Institution - 0209
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Oxnard, United States
Local Institution - 0183
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Walnut Creek, United States
Local Institution - 0227
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Hartford, United States
Local Institution - 0173
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New Haven, United States
Halifax Health Medical Center
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Daytona Beach, United States
Local Institution - 0202
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Fort Myers, United States
Local Institution - 0180
Verified postcode
Jacksonville, United States

Common questions

What is Myelodysplastic Syndrome (MDS)?

MDS is a condition where your bone marrow doesn't make enough healthy blood cells, often leading to anaemia.

What is anaemia?

Anaemia means you have too few red blood cells, which can make you feel tired, weak, and short of breath.

What is the purpose of this study?

The study aims to see if a new medicine, Luspatercept, is better and safer at treating anaemia in people with MDS compared to a standard medicine, Epoetin Alfa.

Will I get to choose which medicine I receive?

No, you will be randomly assigned to either Luspatercept or Epoetin Alfa, like drawing lots.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your usual medical care.