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Study on Luspatercept for Treating Anemia in Adults and Adolescents with Alpha-Thalassemia

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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 the treatment of alpha-thalassemia, a blood disorder that affects the production of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. The study will use a medication called luspatercept, also known by its code name BMS-986346/ACE-536. Luspatercept is a type of protein designed to help improve the production of red blood cells in people with this condition. The trial will also involve a comparison with a placebo to evaluate the effectiveness of the treatment.

The purpose of the study is to determine how well luspatercept works in treating anemia, a condition characterized by a lack of healthy red blood cells, in adults with alpha-thalassemia. Additionally, the study aims to assess the safety and appropriate dosage of luspatercept in adolescents with the same condition. Participants in the study will receive either luspatercept or a placebo, and their response to the treatment will be monitored over a period of time. The study will look at how the treatment affects the need for blood transfusions and the levels of hemoglobin in the blood.

Throughout the study, participants will receive regular injections of the medication or placebo and will be closely monitored by healthcare professionals. The study will last for several months, during which time the safety and effectiveness of luspatercept will be evaluated. The goal is to find a safe and effective treatment option for people with alpha-thalassemia, potentially reducing their need for blood transfusions and improving their overall quality of life.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current health status.

    Participants must have a documented diagnosis of alpha-thalassemia HbH disease and meet specific criteria regarding previous blood transfusions and hemoglobin levels.

  2. Step 2

    Treatment initiation

    The treatment phase begins with the administration of luspatercept, a medication given as a subcutaneous injection. This means it is injected under the skin.

    The dosage of luspatercept is determined based on the participant's specific needs and is administered at regular intervals as specified by the study protocol.

  3. Step 3

    Monitoring and follow-up

    Participants are monitored regularly to assess the effectiveness and safety of the treatment. This includes blood tests to measure hemoglobin levels and check for any side effects.

    Regular follow-up visits are scheduled to ensure the participant's well-being and to make any necessary adjustments to the treatment plan.

  4. Step 4

    Evaluation of treatment response

    The primary goal for adult participants is to achieve a significant reduction in the need for blood transfusions or an increase in hemoglobin levels, depending on their transfusion status.

    For adolescent participants, the focus is on confirming a safe and tolerable dose of luspatercept and monitoring for any dose-limiting side effects.

  5. Step 5

    Completion of study participation

    At the end of the study period, a final assessment is conducted to evaluate the overall response to the treatment and any long-term effects.

    Participants may receive guidance on future treatment options based on the outcomes of the study.

Who can join the trial?

8 criteria

  • Must be an adult aged 18 years or older, or an adolescent aged 12 to less than 18 years.
  • Must have a documented diagnosis of alpha-thalassemia HbH disease.
  • For adults, if you are transfusion dependent (TD), you must have received at least 6 red blood cell (RBC) units in the 24 weeks before joining the study, with no transfusion-free period longer than 56 days during that time.
  • For adults, if you are not transfusion dependent (NTD), you must have received less than 6 RBC units in the 24 weeks before joining the study, have been transfusion-free for at least 8 weeks before joining, and have a mean hemoglobin (Hb) level of 10 g/dL or less, based on at least 2 measurements taken at least 1 week apart within 4 weeks before joining. Hemoglobin values within 21 days after a transfusion will not be counted.
  • For adolescents, if you are transfusion dependent (TD), you must have had at least 4 RBC transfusion events in the 24 weeks before joining the study, with no transfusion-free period longer than 56 days during that time, and a history of regular transfusions for at least 2 years.
  • For adolescents, if you are not transfusion dependent (NTD), you must have had less than 4 RBC transfusion events in the 24 weeks before joining the study, have been transfusion-free for at least 8 weeks before joining, and have a mean hemoglobin (Hb) level of 10 g/dL or less, based on at least 2 measurements taken at least 1 week apart within 4 weeks before joining. Hemoglobin values within 21 days after a transfusion will not be counted.
  • Must have a performance status score of 50 or higher, which measures your ability to perform daily activities. For those aged 16 and older, the Karnofsky score is used, and for those under 16, the Lansky score is used.
  • Both male and female participants are eligible.

Who cannot join the trial?

10 criteria

  • Participants with any other serious health condition that could interfere with the study.
  • Individuals who have had a recent major surgery or are planning to have one during the study period.
  • People who are currently participating in another clinical trial.
  • Participants who have a history of allergic reactions to the study medication or similar drugs.
  • Individuals with uncontrolled high blood pressure.
  • People with severe liver or kidney disease.
  • Participants who are pregnant or breastfeeding.
  • Individuals with a history of drug or alcohol abuse.
  • People who have received a blood transfusion within a certain period before the study.
  • Participants with any other condition that the study doctors believe would make it unsafe for them to participate.
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Investigated drugs

Luspatercept is a medication being studied for its potential to help people with a type of anemia called alpha-thalassemia. Anemia is a condition where you don't have enough healthy red blood cells to carry oxygen throughout your body. Luspatercept works by helping the body produce more red blood cells, which can improve the symptoms of anemia. In this clinical trial, researchers are looking at how well luspatercept works in adults with a specific form of alpha-thalassemia known as HbH disease. They are also studying its safety and how it behaves in the body in adolescents with the same condition. The goal is to see if luspatercept can be a safe and effective treatment option for people with this type of anemia.

What is already known about the treatment

Luspatercept – Luspatercept is administered through an injection under the skin, typically in a healthcare setting. It is currently being studied in clinical trials for its effectiveness and safety in treating anemia associated with alpha-thalassemia, a blood disorder. The main therapeutic use of Luspatercept is to improve red blood cell production in patients with this condition. At the molecular level, Luspatercept works by binding to certain proteins that regulate red blood cell production, thereby enhancing the body's ability to produce these cells. It is classified as an erythroid maturation agent, which means it helps mature red blood cells.

Investigated diseases

Alpha-thalassemia – Alpha-thalassemia is a blood disorder that reduces the production of hemoglobin, the protein in red blood cells that carries oxygen. It is caused by mutations in the genes responsible for hemoglobin production, leading to a shortage of alpha-globin chains. This results in the formation of abnormal hemoglobin molecules, which can cause red blood cells to be destroyed prematurely. The disease can range from mild to severe, depending on the number of gene mutations. In its more severe form, known as Hemoglobin H disease, it can lead to significant anemia and related symptoms. Over time, individuals may experience fatigue, weakness, and other complications due to the reduced oxygen-carrying capacity of their blood.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-502328-35-00Protocol codeCA056-015Estimated enrolment177 patientsSponsorCelgene Corp.

sourced from the EU Clinical Trials Register and site verification

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