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Study on Luspatercept for Patients with Lower-risk Myelodysplastic Syndrome Requiring Red Blood Cell Transfusions

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Myelodysplastic Syndrome (MDS), which affects the blood and bone marrow, leading to anemia and the need for red blood cell (RBC) transfusions. The study will use a treatment called Luspatercept, also known by its code name BMS-986346 / ACE-536. Luspatercept is a medication that is given as an injection under the skin and is designed to help reduce the need for RBC transfusions in people with certain types of MDS.

The purpose of this study is to evaluate how effective and safe Luspatercept is when started at the highest approved dose for treating anemia in patients with lower-risk MDS who require RBC transfusions. Participants in the study will receive Luspatercept injections and will be monitored to see if they can achieve independence from RBC transfusions, meaning they no longer need them, along with an increase in their hemoglobin levels, which is a measure of the amount of oxygen-carrying protein in the blood.

The study will take place over a period of time, with participants receiving treatment and being observed for changes in their need for transfusions and any side effects they may experience. The main goal is to see if participants can go without needing transfusions for at least eight weeks while also having an increase in hemoglobin levels. The study will also look at how many RBC units are needed over certain periods and the time it takes for participants to achieve transfusion independence. Safety will be closely monitored by tracking any adverse events, which are unwanted effects that may occur during the study.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including age, diagnosis of myelodysplastic syndrome, and the need for red blood cell transfusions.

    Participants are divided into two groups based on previous treatment with erythropoiesis-stimulating agents (ESAs).

  2. Step 2

    Treatment initiation

    The treatment involves the administration of luspatercept, a medication provided as a powder for solution for injection.

    The medication is administered subcutaneously, which means it is injected under the skin.

    The maximum approved dose is used to start the treatment.

  3. Step 3

    Treatment schedule

    The treatment is designed to evaluate the independence from red blood cell transfusions and an increase in hemoglobin levels.

    The primary goal is to achieve transfusion independence for at least 8 weeks with a concurrent increase in hemoglobin levels by at least 1 g/dL from Week 1 to Week 24.

  4. Step 4

    Monitoring and evaluation

    Participants are monitored for changes in the number of red blood cell units transfused over specific periods, from Week 9 to Week 24 and from Week 33 to Week 48.

    The time from the first dose to the first onset of transfusion independence is also evaluated.

  5. Step 5

    Safety assessment

    The safety of the treatment is assessed by monitoring the type, frequency, and severity of any adverse events from the start of the study to 6 weeks after the last dose and from Week 1 to Week 48.

  6. Step 6

    Study duration

    The study is expected to continue until May 2027, with recruitment starting in March 2024.

Who can join the trial?

8 criteria

  • The participant must be **18 years or older**.
  • The participant must have a documented diagnosis of **myelodysplastic syndrome (MDS)**, which is a group of disorders caused by poorly formed or dysfunctional blood cells.
  • The participant must have an **Eastern Cooperative Oncology Group (ECOG) score** of 0, 1, or 2. This score is used to assess how a disease affects a patient's daily living abilities, with 0 being fully active and 2 being capable of all self-care but unable to carry out any work activities.
  • For Cohort 1 only: The participant must have an **endogenous serum erythropoietin (EPO) level** of less than 500 U/L and must not have had any prior **erythropoiesis-stimulating agent (ESA) treatment**. EPO is a hormone that helps produce red blood cells, and ESA treatments are used to stimulate red blood cell production.
  • For Cohort 2 only: The participant must be **refractory** (meaning not responding) or **intolerant** (unable to tolerate) to prior ESA treatment. This means they either did not respond to the treatment or had to stop it due to side effects.
  • The participant must require **red blood cell (RBC) transfusions**, which means they need to receive blood from a donor to maintain their health.
  • The participant must have an average transfusion requirement of at least 1 unit of **packed red blood cells (pRBCs)** over a minimum of 8 weeks before starting the study treatment.
  • The participant's **hemoglobin (Hb) levels** must be 10.0 g/dL or lower at the time of or within 7 days before receiving an RBC transfusion for it to count towards meeting the eligibility criteria. Hemoglobin is a protein in red blood cells that carries oxygen throughout the body.

Who cannot join the trial?

5 criteria

  • Patients with a different medical condition than myelodysplastic syndrome cannot participate. This is a condition where the bone marrow does not produce enough healthy blood cells.
  • Patients who do not require red blood cell transfusions are excluded. This means if you don't need extra blood due to low red blood cells, you cannot join.
  • Patients who are not in the age range specified for the study cannot participate. The study is for certain age groups only.
  • Patients who are not classified as having very low, low, or intermediate risk according to a specific system used to assess the severity of their condition cannot join. This system helps determine how serious the condition is.
  • Patients who are not able to achieve a hemoglobin increase of at least 1 g/dL with the treatment cannot participate. Hemoglobin is a protein in red blood cells that carries oxygen.
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Investigated drugs

Luspatercept: This medication is being studied to see if it can help people with certain types of anemia who need regular red blood cell transfusions. It is used to try to increase the amount of hemoglobin in the blood, which can help reduce the need for these transfusions. The study is looking at how effective and safe it is when given at the highest approved dose.

What is already known about the treatment

Luspatercept – Luspatercept is administered via subcutaneous injection and is currently being studied in clinical trials for its effectiveness in treating anemia associated with myelodysplastic syndrome. It is approved for use in patients with very low, low, or intermediate-risk myelodysplastic syndromes who require red blood cell transfusions. The medication works by binding to certain proteins in the body, which helps to increase the production of red blood cells. Luspatercept is classified as an erythroid maturation agent, which means it aids in the maturation of red blood cells to improve anemia.

Investigated diseases

Myelodysplastic syndrome – This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when something goes wrong in the bone marrow, where blood cells are produced. People with this syndrome often have a shortage of one or more types of blood cells, leading to symptoms like fatigue, infections, or easy bruising. Over time, the condition can progress, causing more severe symptoms as the bone marrow becomes less effective at producing healthy blood cells. The disease can vary greatly in severity, with some individuals experiencing mild symptoms and others facing more significant health challenges. The progression of the disease can lead to a need for regular blood transfusions to manage symptoms.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-504541-31-00Protocol codeCA0561060Estimated enrolment100 patientsSponsorCelgene Corp.

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