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Study on the Effectiveness and Safety of Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions

Verified siteRegistered drugNo placebo
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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), specifically a type called lower-risk MDS with ring sideroblasts (MDS-RS). MDS is a group of disorders caused by poorly formed or dysfunctional blood cells. The study is investigating the effectiveness and safety of a treatment called luspatercept, which is also known by its code name ACE-536. Luspatercept is a medication that is given as an injection under the skin and is designed to help improve anemia, a condition where the body does not have enough healthy red blood cells.

The purpose of this study is to see if luspatercept can help patients with lower-risk MDS-RS who need regular red blood cell transfusions. These patients have not responded well to other treatments that stimulate the production of red blood cells. The study will follow participants over a period of 24 weeks to see if they can achieve independence from red blood cell transfusions. This means that the patients would not need transfusions to maintain healthy red blood cell levels during this time.

Participants in the study will receive luspatercept injections and will be monitored for any changes in their need for red blood cell transfusions, as well as any side effects they may experience. The study will also look at other outcomes, such as changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, and overall survival. The goal is to determine if luspatercept can provide a safe and effective treatment option for patients with lower-risk MDS-RS.

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 patient will be required to sign an informed consent form. This form confirms the patient's understanding of the study and willingness to participate.

    The patient must be at least 18 years old and able to follow the study schedule and requirements.

  2. Step 2

    Initial assessment

    The patient will undergo an initial assessment to confirm eligibility. This includes verifying a diagnosis of myelodysplastic syndrome (MDS) with specific characteristics, such as the presence of ring sideroblasts in the bone marrow.

    The patient must have a history of anemia requiring red blood cell (RBC) transfusions and must have been unresponsive, intolerant, or ineligible for previous treatments with erythropoiesis-stimulating agents (ESA).

  3. Step 3

    Treatment initiation

    The patient will begin treatment with luspatercept, administered as a subcutaneous injection. The specific dosage will be determined by the healthcare provider based on the patient's needs.

    The treatment aims to reduce the need for RBC transfusions and improve anemia symptoms.

  4. Step 4

    Treatment schedule

    The patient will receive luspatercept injections at regular intervals, as determined by the healthcare provider. The frequency and duration of the treatment will be specified in the study protocol.

    The patient will be monitored for any side effects or adverse reactions to the treatment.

  5. Step 5

    Ongoing assessments

    Throughout the study, the patient will undergo regular assessments to evaluate the effectiveness of the treatment. This includes monitoring the patient's hemoglobin levels and the need for RBC transfusions.

    The patient will also be assessed for any changes in symptoms and overall health status.

  6. Step 6

    End of treatment

    At the end of the treatment period, the patient will have a final assessment to determine the overall impact of the treatment on their condition.

    The patient will be informed about the results and any further steps or follow-up care that may be necessary.

Who can join the trial?

10 criteria

  • Must be 18 years or older.
  • Must be willing and able to follow the study schedule and requirements.
  • Must understand and sign the consent form before any study procedures.
  • Must have a confirmed diagnosis of a specific type of blood disorder called MDS with certain characteristics in the bone marrow and blood.
  • Must have tried certain treatments for anemia and either not responded, stopped responding, or been unable to tolerate them.
  • If previously treated with specific medications, they must be stopped at least 4 weeks before starting the study treatment.
  • Must need regular blood transfusions, with specific requirements for the amount and timing of these transfusions.
  • Must have an ECOG score of 0, 1, or 2, which is a measure of general health and ability to perform daily activities.
  • If a woman can become pregnant, she must have two negative pregnancy tests before starting the study and agree to use effective birth control during and after the study.
  • Men must agree to use a condom during sexual contact with a pregnant woman or a woman who can become pregnant while in the study and for a period after the study ends.

Who cannot join the trial?

3 criteria

  • Patients who do not have anemia due to very low-, low-, or intermediate-risk MDS with ring sideroblasts (RS) as defined by the International Prognostic Scoring System-Revised (IPSS-R).
  • Patients who do not require red blood cell (RBC) transfusions.
  • Patients who are not refractory, intolerant, or ineligible to prior erythropoiesis-stimulating agents (ESA) treatment. Refractory means that the treatment no longer works for the patient. Intolerant means that the patient cannot tolerate the side effects of the treatment. Ineligible means that the patient cannot receive the treatment for other reasons.
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Investigated drugs

Luspatercept is a medication used in this clinical trial to help patients with a specific type of blood disorder called myelodysplastic syndromes (MDS) that have a ring sideroblastic phenotype. This condition often leads to anemia, which means the body doesn't have enough healthy red blood cells. Luspatercept works by helping the body produce more red blood cells, which can reduce or eliminate the need for blood transfusions. The goal of using luspatercept in this study is to see if it can help patients become independent of red blood cell transfusions, improving their quality of life.

What is already known about the treatment

Luspatercept – Luspatercept is administered as an injection under the skin, typically in the upper arm, thigh, or abdomen. It is currently being studied in clinical trials for its effectiveness and safety in treating anemia in patients with a specific type of blood disorder called myelodysplastic syndromes with ring sideroblasts (MDS-RS). The main therapeutic use of luspatercept is to help patients become less dependent on red blood cell transfusions. At the molecular level, luspatercept works by binding to certain proteins in the body to help increase the production of red blood cells. It is classified as an erythroid maturation agent, which means it helps mature red blood cells.

Investigated diseases

Myelodysplastic Syndromes with Ring Sideroblasts (MDS-RS) – Myelodysplastic Syndromes with Ring Sideroblasts are a group of disorders caused by poorly formed or dysfunctional blood cells. In MDS-RS, the bone marrow produces ring sideroblasts, which are abnormal red blood cell precursors with iron-loaded mitochondria. This condition leads to anemia, as the body does not produce enough healthy red blood cells. Over time, patients may require regular red blood cell transfusions due to the persistent anemia. The disease can progress slowly, with symptoms like fatigue and weakness becoming more pronounced as the anemia worsens. The risk of progression to more severe forms of blood disorders varies among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515069-33-00Protocol codeLUSPLUSEstimated enrolment58 patientsSponsorGWT-Tud GmbH

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