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Study on the Safety and Effects of Luspatercept for Children with Beta 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 a condition called Beta Thalassemia, which is a blood disorder that reduces the production of hemoglobin. Hemoglobin is an important part of red blood cells that carries oxygen throughout the body. The study is testing a treatment called Luspatercept, 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 increase the production of healthy red blood cells in people with Beta Thalassemia.

The purpose of this study is to find a safe and tolerable dose of Luspatercept for children with Beta Thalassemia who either need regular blood transfusions or do not. The study will also look at how the body processes the medication. Participants in the study will receive either Luspatercept or a placebo. The study will last for a period of time, during which participants will receive regular injections and attend follow-up visits to monitor their health and the effects of the treatment.

Throughout the study, researchers will collect information on the safety of Luspatercept, including any side effects that may occur. They will also measure changes in the need for blood transfusions and levels of hemoglobin in the participants. This information will help determine if Luspatercept is a beneficial treatment option for children with Beta Thalassemia. The study aims to provide valuable insights into managing this condition and improving the quality of life for those affected.

The research process

The trial runs in 7 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 will be required to sign an informed consent form. This form confirms understanding and agreement to participate in the study.

    The participant must be between 6 and 18 years old and have a documented diagnosis of beta thalassemia or HbE/beta thalassemia.

  2. Step 2

    Initial assessments

    Initial assessments will be conducted to confirm eligibility. These assessments may include medical history review, physical examination, and laboratory tests.

    Participants will be categorized as either transfusion-dependent (TD) or non-transfusion-dependent (NTD) based on their transfusion history.

  3. Step 3

    Treatment administration

    Participants will receive luspatercept as a solution for injection. The medication is administered subcutaneously, which means it is injected under the skin.

    The dosage and frequency of administration will be determined based on the participant's specific needs and the study protocol.

  4. Step 4

    Regular follow-up visits

    Participants will attend regular follow-up visits to monitor their health and response to the treatment. These visits will include physical examinations and laboratory tests.

    The frequency of these visits will be outlined in the study schedule provided to the participant.

  5. Step 5

    Safety and effectiveness evaluations

    Throughout the study, evaluations will be conducted to assess the safety and effectiveness of luspatercept. This includes monitoring for any side effects and changes in hemoglobin levels.

    Participants will be asked to report any adverse effects or changes in their health to the study team.

  6. Step 6

    End of treatment

    At the end of the treatment period, participants will undergo a final assessment to evaluate the overall impact of the treatment.

    Participants will be informed about the results and any further steps if necessary.

  7. Step 7

    Safety follow-up

    A safety follow-up visit will be scheduled approximately 9 weeks after the end of treatment to ensure the participant's well-being.

    This visit will include a final check-up and any necessary laboratory tests.

Who can join the trial?

10 criteria

  • Participant must be between 6 and 17 years old at the time of signing the consent form.
  • Participant must have a documented diagnosis of β-thalassemia or HbE/β-thalassemia.
  • For participants who need regular blood transfusions (transfusion-dependent): Must have had at least 4 blood transfusion events in the 24 weeks before joining the study, with no gap of 42 days or more without a transfusion.
  • For participants who do not need regular blood transfusions (non-transfusion-dependent): Must have had fewer than 4 blood transfusion events in the 24 weeks before joining the study and must not have had a blood transfusion for at least 8 weeks before joining. Their average hemoglobin level must be 10 g/dL or lower, based on at least 2 measurements taken at least 1 week apart within 4 weeks before joining.
  • Participant must have a performance status score of 50 or higher, which measures their ability to perform daily activities.
  • Male participants must agree to use a condom during sexual contact with a pregnant female or a female who can become pregnant while in the study and for at least 12 weeks after stopping the study treatment.
  • Female participants who can become pregnant must have 2 negative pregnancy tests before starting the study treatment and agree to ongoing pregnancy testing during the study.
  • Female participants who can become pregnant must agree to use effective birth control or commit to not having heterosexual contact during the study and for 12 weeks after stopping the study treatment.
  • Participant (and when applicable, their parent or legal representative) must understand and voluntarily sign the consent form before any study-related procedures.
  • Participant (and when applicable, their parent or legal representative) must be willing and able to follow the study visit schedule and other study requirements.

Who cannot join the trial?

10 criteria

  • Patients who have any other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent major surgery or are planning to have surgery during the study period.
  • Patients who have a known allergy to the study medication or any of its ingredients.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have an active infection that requires treatment.
  • Patients who have received a blood transfusion within a certain period before the study starts.
  • Patients who have a history of certain types of cancer.
  • Patients who have a condition that affects their ability to understand or follow study instructions.
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Investigated drugs

Luspatercept is a medication being studied to help people with a blood condition called beta thalassemia. This condition affects how the body makes red blood cells, which are important for carrying oxygen throughout the body. Luspatercept works by helping the body produce more healthy red blood cells. In this clinical trial, researchers are trying to find out the best dose of Luspatercept that is safe and can be tolerated by children with beta thalassemia. They are also studying how the medication moves through the body and how it is processed. This information will help determine if Luspatercept can be a useful treatment for children with this condition, whether they need regular blood transfusions or not.

What is already known about the treatment

Luspatercept – Luspatercept is administered as an injection under the skin, typically in a healthcare setting. It is currently being studied in clinical trials, particularly for its safety and how it behaves in the body in children with beta thalassemia, a blood disorder. The main use of Luspatercept is to help manage anemia in patients with beta thalassemia, whether they need regular blood transfusions or not. It works by helping the body produce more red blood cells, which can reduce the need for transfusions. Luspatercept is classified as an erythroid maturation agent, which means it helps red blood cells mature properly.

Investigated diseases

Beta Thalassemia – Beta Thalassemia is a blood disorder that reduces the production of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. This condition is caused by mutations in the HBB gene, leading to a deficiency in beta-globin chains. As a result, individuals with Beta Thalassemia experience anemia, which can cause fatigue, weakness, and pale skin. Over time, the body attempts to compensate for the lack of hemoglobin by producing more red blood cells, which can lead to bone deformities and an enlarged spleen. The severity of symptoms can vary, with some individuals requiring regular blood transfusions to manage their condition. In some cases, iron overload can occur due to frequent blood transfusions, necessitating additional management strategies.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2022-502499-22-00Protocol codeACE-536-B-THAL-004Estimated enrolment76 patientsSponsorCelgene Corp.

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).