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Study on Luspatercept for Treating Anemia in Adults with Myelofibrosis on JAK2 Inhibitor Therapy Requiring Red Blood Cell 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 called anemia that is associated with a type of blood disorder known as myeloproliferative neoplasm-associated myelofibrosis. This condition often requires patients to receive regular red blood cell transfusions to manage their symptoms. The study is testing a treatment called luspatercept, also known by its code name ACE-536 (BMS-986346), to see if it can help reduce the need for these transfusions. Luspatercept is given as a solution for injection under the skin.

The purpose of the study is to compare the effectiveness and safety of luspatercept with a placebo in adults who are already receiving a type of medication called a JAK2 inhibitor as part of their standard treatment. Participants in the study will receive either luspatercept or a placebo, and their progress will be monitored over a period of time to see if they can become free from needing red blood cell transfusions for at least 12 weeks.

Throughout the study, participants will continue their usual treatment with the JAK2 inhibitor while receiving the study medication. The trial aims to provide valuable information on whether luspatercept can offer a significant benefit in managing anemia in patients with myelofibrosis, potentially improving their quality of life by reducing the frequency of blood transfusions needed.

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

    Joining the study

    Upon joining the study, the participant will be randomly assigned to receive either the study medication, luspatercept, or a placebo. This process is double-blind, meaning neither the participant nor the study team will know which treatment is being administered.

  2. Step 2

    Treatment administration

    The study involves the administration of luspatercept or placebo through subcutaneous injections. The specific products used are Reblozyl 75 mg and Reblozyl 25 mg, both in powder form for solution for injection.

    Injections are given at regular intervals as determined by the study protocol. The exact dosage and frequency will be communicated by the study team.

  3. Step 3

    Monitoring and assessments

    Throughout the study, regular monitoring and assessments will be conducted to evaluate the participant's response to the treatment. This includes tracking the need for red blood cell transfusions and any changes in anemia symptoms.

    Participants will undergo various tests and assessments to ensure their safety and to measure the effectiveness of the treatment.

  4. Step 4

    Blinded core treatment period

    The blinded core treatment period lasts up to 24 weeks. During this time, the primary goal is to determine if the participant becomes free from the need for red blood cell transfusions over any consecutive 12-week period.

    A secondary goal is to assess if the participant remains transfusion-free over any consecutive 16-week period starting during this treatment phase.

  5. Step 5

    End of study participation

    At the end of the study, participants will undergo final assessments to evaluate the overall impact of the treatment.

    Participants will be informed about the results of the study and their specific treatment allocation (whether they received luspatercept or placebo).

Who can join the trial?

7 criteria

  • The person must be at least 18 years old when they sign the consent form.
  • The person must have a diagnosis of primary myelofibrosis (PMF) or myelofibrosis that developed after other conditions, confirmed by a recent pathology report. Myelofibrosis is a condition where scar tissue forms in the bone marrow, affecting blood cell production.
  • The person must need regular red blood cell (RBC) transfusions. This means they need 4 to 12 units of RBCs over 12 weeks, with no gap longer than 6 weeks without at least one transfusion. Transfusions are needed for anemia, which is a low level of red blood cells, causing symptoms like tiredness or shortness of breath.
  • The person must be on continuous JAK2 inhibitor therapy, a type of medication for myelofibrosis, for at least 32 weeks, with a stable dose for at least 16 weeks before joining the study. They should also be expected to continue this stable dose for at least 24 weeks after joining the study.
  • The person must have an Eastern Cooperative Oncology Group (ECOG) performance score of 2 or less. This score measures how well a person can perform daily activities, with lower scores indicating better ability.
  • Females who can have children must have two negative pregnancy tests before starting the study and agree to ongoing pregnancy testing. They must either practice true abstinence from heterosexual contact or use effective birth control before and during the study, and for 12 weeks after stopping the study treatment.
  • Male participants must practice true abstinence or use a condom during sexual contact with a pregnant female or a female who can have children while in the study, during breaks in treatment, and for at least 12 weeks after stopping the study treatment, even if they have had a vasectomy.

Who cannot join the trial?

3 criteria

  • Patients who do not have anemia related to a condition called myeloproliferative neoplasm-associated myelofibrosis cannot participate. This is a type of blood disorder.
  • Patients who are not currently receiving treatment with a JAK2 inhibitor cannot participate. JAK2 inhibitors are medications used to treat certain blood disorders.
  • Patients who do not need regular red blood cell transfusions cannot participate. A red blood cell transfusion is a procedure where you receive blood through a vein.
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Investigated drugs

Luspatercept (ACE-536) is a medication being studied for its ability to treat anemia in patients with myeloproliferative neoplasm-associated myelofibrosis. This condition often requires patients to receive red blood cell transfusions. Luspatercept works by helping the body produce more red blood cells, potentially reducing the need for transfusions. It is being tested in combination with JAK2 inhibitor therapy, which is commonly used to manage symptoms of myelofibrosis.

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 myeloproliferative neoplasm-associated myelofibrosis. It is not yet fully established in medical practice but shows promise in ongoing research. The main therapeutic indication for luspatercept is to reduce the need for red blood cell transfusions in patients with this specific type of anemia. At the molecular level, luspatercept works by binding to certain proteins involved in red blood cell production, thereby enhancing the maturation of these cells. It is classified pharmacologically as an erythroid maturation agent.

Investigated diseases

Anemia associated with myeloproliferative neoplasm-associated myelofibrosis – This condition occurs when the bone marrow, which is responsible for producing blood cells, becomes scarred and fibrotic, leading to a decrease in the production of red blood cells. As a result, individuals experience anemia, characterized by fatigue, weakness, and pallor due to insufficient oxygen being transported throughout the body. Myeloproliferative neoplasms are a group of diseases where the bone marrow makes too many blood cells, and when associated with myelofibrosis, it can lead to further complications in blood cell production. The progression of this condition involves worsening anemia and may require regular blood transfusions to manage symptoms. Over time, the bone marrow's ability to produce healthy blood cells continues to decline, exacerbating the symptoms of anemia.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507890-17-00Protocol codeACE-536-MF-002Estimated enrolment224 patientsSponsorCelgene Corp.

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