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Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

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 the long-term safety of a treatment called luspatercept, also known by its code name ACE-536 or BMS-986346. The study involves participants who have previously taken part in other trials using this treatment. The diseases being studied include myelodysplastic syndrome (MDS), beta-thalassemia, and myelofibrosis. These are conditions that affect the blood and bone marrow, leading to issues like anemia and other blood-related problems.

The purpose of this study is to evaluate the long-term safety of luspatercept, including monitoring for any progression to more serious conditions such as acute myeloid leukemia (AML) or the development of other types of cancer. Participants in this study will receive luspatercept as a solution for injection, which is administered under the skin. The study is designed as an open-label, single-arm, rollover study, meaning all participants will receive the treatment without a comparison group receiving a placebo.

Throughout the study, participants will be monitored for any adverse events or changes in their condition. The study will also track overall survival and any new growths that may develop. The maximum treatment period for participants is up to 360 days, during which they will continue to receive the treatment and be regularly assessed by healthcare professionals. This study aims to provide valuable information on the long-term effects and safety of luspatercept for individuals with these blood disorders.

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

    Upon joining the study, eligibility is confirmed. This includes being 18 years or older and having participated in a previous luspatercept trial.

    An informed consent document is signed, indicating understanding and agreement to the study's requirements.

  2. Step 2

    Initial Assessment

    Initial assessments are conducted to establish a baseline for health and safety monitoring.

    For females of childbearing potential, two negative pregnancy tests are required before starting the study therapy.

  3. Step 3

    Treatment Phase

    The treatment involves receiving luspatercept through subcutaneous injections.

    The medication is available in two forms: Reblozyl 25 mg and Reblozyl 75 mg powder for solution for injection.

    The frequency and dosage are determined by the study protocol and the investigator's assessment of clinical benefit.

  4. Step 4

    Monitoring and Follow-up

    Regular monitoring is conducted to evaluate long-term safety, including any adverse events or progression to other conditions.

    Participants are required to adhere to the study visit schedule for ongoing assessments.

  5. Step 5

    End of Study

    The study is estimated to conclude by February 23, 2029.

    Upon completion, participants may undergo final assessments to evaluate overall health and any long-term effects of the treatment.

Who can join the trial?

11 criteria

  • The person must be at least 18 years old when they sign the consent form.
  • The person must be willing and able to follow the schedule of study visits and other study requirements.
  • The person must already be part of a study involving a treatment called luspatercept and continue to meet the requirements of that study. They must be in one of the following situations:
    • Currently receiving luspatercept and benefiting from it, according to the study doctor.
    • Previously received a placebo (a harmless pill with no effect) and should now start luspatercept treatment.
    • In the follow-up phase of the previous study, having been treated with either luspatercept or placebo, and should continue in the long-term follow-up phase.
    • The person must understand and willingly sign a consent form before any study-related tests or procedures are done.
    • The person must have followed the rules of the previous study, as checked by the study doctor.
    • For women who can have children and are currently receiving treatment:
      • They must have two negative pregnancy tests before starting the study treatment.
      • They must agree to regular pregnancy tests during and after the study.
      • They must either commit to not having sexual contact or agree to use effective birth control methods before, during, and after the study.
      • For men currently receiving treatment:
        • They must agree to use a condom during sexual contact with a pregnant woman or a woman who can have children while in the study and for a period after stopping the study treatment, even if they have had a vasectomy (a procedure to prevent pregnancy).

Who cannot join the trial?

7 criteria

  • Patients who have not participated in other clinical trials involving the medication called luspatercept.
  • Patients who have a history of progressing to a condition called acute myeloid leukemia (AML), which is a type of blood cancer.
  • Patients who have developed other types of cancers or pre-cancerous conditions.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial groups.
  • Patients who are not male or female, as the study includes both genders.
  • Patients who are considered part of a vulnerable population, which means they might need special protection or care.
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Investigated drugs

Luspatercept is a medication being studied for its long-term safety in patients who have previously participated in other clinical trials involving this drug. It is used to treat certain types of anemia by helping the body produce more red blood cells. This study aims to monitor any potential progression to acute myeloid leukemia or other related conditions in patients using luspatercept over an extended period.

What is already known about the treatment

Luspatercept – Luspatercept is administered via subcutaneous injection and is currently being studied in clinical trials for its long-term safety. It is primarily used for treating conditions like myelodysplastic syndrome, beta-thalassemia, and myelofibrosis. The medication works by binding to certain proteins in the body, which helps to regulate the production of red blood cells. Luspatercept is classified as an erythroid maturation agent, which means it aids in the development of red blood cells.

Investigated diseases

  • Myelodysplastic Syndrome (MDS)

    This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the blood-forming cells in the bone marrow are damaged, leading to a shortage of one or more types of blood cells. Over time, MDS can progress to more severe forms, affecting the production of red blood cells, white blood cells, and platelets. Patients may experience symptoms like fatigue, infections, or easy bruising due to low blood counts. The condition can vary in severity, with some cases remaining stable for years while others progress more rapidly.

  • Beta-Thalassemia (THAL)

    This 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 HBB gene, leading to anemia and fatigue. The severity of beta-thalassemia can range from mild to severe, depending on the specific genetic mutations. In severe cases, individuals may require regular blood transfusions to manage symptoms. Over time, the condition can lead to complications such as bone deformities and growth delays.

  • Myelofibrosis (MF)

    This is a rare type of bone marrow cancer that disrupts the body's normal production of blood cells. It leads to extensive scarring in the bone marrow, resulting in severe anemia, weakness, and fatigue. As the disease progresses, it can cause an enlarged spleen and liver, as well as other complications. Patients may experience symptoms such as night sweats, fever, and bone pain. The progression of myelofibrosis can vary, with some individuals experiencing a slow course while others may have a more rapid decline.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502498-40-00Protocol codeACE-536-LTFU-001Estimated enrolment293 patientsSponsorCelgene Corp.

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