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Study Comparing Luspatercept and Epoetin Alfa for Treating Anemia in Patients with Low-Risk Myelodysplastic Syndrome Who Are Not Dependent on Blood 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 is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. The study aims to compare the effectiveness and safety of two treatments for anemia caused by MDS. The treatments being compared are Luspatercept, also known by its code name ACE-536, and Epoetin Alfa. Both of these treatments are designed to help increase the production of red blood cells in the body.

The purpose of the study is to see how well these treatments work in people with lower-risk MDS who are not dependent on blood transfusions. Participants in the study will receive either Luspatercept or Epoetin Alfa through injections under the skin. The study will last for a period of up to 96 weeks, during which participants will be monitored to see if they need fewer blood transfusions and if their hemoglobin levels, which measure the amount of red blood cells, improve.

Throughout the study, participants will be regularly assessed to ensure their safety and to track the effectiveness of the treatments. The study will help determine which treatment is more effective in managing anemia in patients with MDS who have not previously been treated with erythropoiesis-stimulating agents, which are medications that help the body produce more red blood cells. This research is important for improving treatment options for people living with MDS.

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, the participant is confirmed to meet the eligibility criteria, including age, diagnosis of myelodysplastic syndrome (MDS), and transfusion independence.

    The participant's baseline hemoglobin (Hb) concentration and serum erythropoietin level are assessed to ensure they meet the study requirements.

  2. Step 2

    Randomization

    The participant is randomly assigned to receive either luspatercept or epoetin alfa.

    This randomization determines the treatment group for the duration of the study.

  3. Step 3

    Treatment administration

    Participants in the luspatercept group receive the medication as a subcutaneous injection. The dosage is either 25 mg or 75 mg, depending on the specific requirements of the study.

    Participants in the epoetin alfa group receive the medication as a subcutaneous injection. The available dosages are 2,000 IU, 4,000 IU, 6,000 IU, 20,000 IU, 30,000 IU, or 40,000 IU, administered in pre-filled syringes.

    The frequency and duration of administration are determined by the study protocol and the participant's response to treatment.

  4. Step 4

    Monitoring and assessments

    Throughout the study, regular monitoring of hemoglobin levels and other health indicators is conducted to assess the participant's response to treatment.

    Participants are evaluated for any symptoms of anemia, such as fatigue, weakness, shortness of breath, or dizziness.

  5. Step 5

    End of treatment period

    The treatment period lasts up to 96 weeks, during which the primary endpoint is assessed: conversion to transfusion dependence, defined as receiving 3 or more units of red blood cells within any 16-week interval.

    Secondary endpoints include achieving an increase in mean hemoglobin values from baseline by at least 1.5 g/dL in any continuous 16-week interval without transfusion.

  6. Step 6

    Study completion

    Upon completion of the study, final assessments are conducted to evaluate the overall efficacy and safety of the treatment received.

    Participants receive information about their treatment outcomes and any necessary follow-up care.

Who can join the trial?

8 criteria

  • Participant must be at least 18 years old or the local age of consent.
  • Participant must have a confirmed diagnosis of **Myelodysplastic Syndrome (MDS)**, which is a type of blood disorder. The diagnosis should meet specific risk levels as determined by a test called **IPSS-R**. This includes very low, low, or intermediate risk, with certain conditions in the bone marrow and blood.
  • Participant must have a baseline level of a substance in the blood called **serum erythropoietin** that is 500 U/L or lower.
  • Participant must not need regular blood transfusions, according to specific criteria. However, 1 to 2 units of blood transfusion are allowed if they were given for an acute event like surgery, bleeding, or infection, and not just for low hemoglobin levels.
  • Participant must have a baseline **hemoglobin (Hb)** concentration of 9.5 g/dL or lower. Hemoglobin is a protein in red blood cells that carries oxygen. This level is calculated using the average of the two lowest measurements taken within 16 weeks before the study starts.
  • Participant must have symptoms of **anemia**, such as moderate or greater fatigue, weakness, shortness of breath, or dizziness, as recorded during the screening period.
  • Participant must have an **Eastern Cooperative Oncology Group (ECOG)** score of 0, 1, or 2. This score is used to assess how well a person can perform daily activities.
  • Participant must not have used **erythropoiesis-stimulating agents** before, which are drugs that help the body make more red blood cells. However, if the participant has received no more than 2 doses of certain medications like epoetin alfa, and the last dose was at least 8 weeks before the study, they may still be eligible.

Who cannot join the trial?

8 criteria

  • Patients with a different condition than Myelodysplastic Syndrome (MDS) cannot participate. MDS is a group of disorders caused by poorly formed or dysfunctional blood cells.
  • Patients who are not in the age range specified for the study cannot participate. The study includes certain age groups only.
  • Patients who do not meet the specific health requirements set by the study cannot participate.
  • Patients who are not able to follow the study procedures or attend the required visits cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients with certain medical conditions that could interfere with the study results cannot participate.
  • Patients who are taking medications that could affect the study outcomes may not be eligible.
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Investigated drugs

  • Luspatercept (ACE 536)

    is a medication being studied for its ability to treat anemia in patients with certain types of myelodysplastic syndrome (MDS). It works by helping the body produce more red blood cells, which can reduce the need for blood transfusions in patients who are not currently dependent on them.

  • Epoetin Alfa

    is a medication used to treat anemia by stimulating the bone marrow to produce more red blood cells. It is commonly used in patients with various types of anemia, including those with myelodysplastic syndrome (MDS), to help increase their red blood cell count and reduce the need for blood transfusions.

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 (MDS). It is primarily indicated for patients with low to intermediate risk MDS who are not dependent on blood transfusions. Luspatercept works by binding to specific proteins in the body, which helps to enhance red blood cell production. It is classified as an erythroid maturation agent.

  • Epoetin Alfa

    Epoetin Alfa is given through subcutaneous or intravenous injection and is widely used in medicine to treat anemia, particularly in patients with chronic kidney disease or undergoing chemotherapy. In the context of myelodysplastic syndrome (MDS), it is being compared to luspatercept in clinical trials for its ability to manage anemia in patients who have not previously used erythropoiesis-stimulating agents. Epoetin Alfa functions by stimulating the bone marrow to produce more red blood cells, and it is classified as an erythropoiesis-stimulating agent.

Investigated diseases

Myelodysplastic Syndrome (MDS) – Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the bone marrow, which is responsible for producing blood cells, does not function properly. This leads to a shortage of one or more types of blood cells, such as red blood cells, white blood cells, or platelets. Symptoms may include fatigue, shortness of breath, easy bruising, and frequent infections. Over time, MDS can progress to more severe forms of anemia or even acute myeloid leukemia. The progression and symptoms can vary widely among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500430-29-00Protocol codeCA056-025Estimated enrolment366 patientsSponsorCelgene Corp.

sourced from the EU Clinical Trials Register and site verification

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