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Study on Axatilimab and Corticosteroids for Initial Treatment of Chronic Graft-Versus-Host Disease in Patients

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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 chronic graft-versus-host disease (cGVHD). This is a complication that can occur after a stem cell or bone marrow transplant, where the donated cells attack the recipient's body. The study will explore the effectiveness of a new treatment involving a medication called Axatilimab (INCA034176), used in combination with corticosteroids, which are a type of steroid medication. The trial will also include a comparison with a placebo to evaluate the treatment's effectiveness.

The purpose of the study is to compare how well Axatilimab works in combination with corticosteroids compared to a placebo in treating moderate or severe cases of cGVHD. Participants in the study will receive either the new treatment or a placebo, and their progress will be monitored over time. The study will involve regular check-ups and assessments to track the condition and any changes in symptoms.

Participants will be randomly assigned to different groups to ensure a fair comparison between the new treatment and the placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo, to prevent bias in the results. The trial will continue for a set period, during which the safety and effectiveness of the treatment will be closely monitored.

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

    Randomization

    Upon joining the study, participants are randomly assigned to one of two groups. One group receives the medication axatilimab along with corticosteroids, while the other group receives a placebo with corticosteroids. This process is double-blind, meaning neither the participants nor the researchers know who is receiving which treatment.

  2. Step 2

    Initial treatment phase

    Participants begin the initial treatment phase. Those in the axatilimab group receive the medication either through an intravenous (IV) or subcutaneous (SC) route. The corticosteroids, such as prednisone or methylprednisolone, are administered orally or intravenously, depending on the specific medication and dosage required.

    The dosage and frequency of administration are determined by the study protocol and the participant's specific needs.

  3. Step 3

    Monitoring and assessment

    Throughout the study, participants undergo regular monitoring to assess the effectiveness of the treatment. This includes evaluating the response to the medication and any side effects experienced.

    The primary goal is to determine the time from the start of the study to the occurrence of any significant events, such as the need for additional therapy, treatment failure, or any other adverse outcomes.

  4. Step 4

    Response evaluation

    At six months, participants' responses to the treatment are evaluated. This involves assessing whether there has been a complete or partial response to the treatment without the need for new systemic therapy.

    The evaluation is based on established criteria for chronic graft-versus-host disease, focusing on improvements in symptoms and overall health.

  5. Step 5

    Follow-up

    Participants continue to be monitored for any long-term effects of the treatment. This includes regular check-ups and assessments to ensure the ongoing health and safety of the participants.

    The study is expected to continue until March 2030, with participants being followed up throughout this period to gather comprehensive data on the treatment's effectiveness and safety.

Who can join the trial?

7 criteria

  • Must be at least 12 years old at the time of giving consent to participate in the study.
  • Must be able to understand and willing to sign a written consent form. If the participant is a child, a parent or guardian must provide consent, and the child should also sign an assent form if possible.
  • Must have a new case of moderate or severe **chronic graft-versus-host disease (cGVHD)**, which is a condition that can occur after a bone marrow or stem cell transplant. This condition requires treatment that affects the whole body. Moderate cGVHD involves at least one organ with a certain level of severity, or multiple organs with milder involvement. Severe cGVHD involves at least one organ with a high level of severity.
  • Must have a history of receiving an **allogeneic hematopoietic cell transplant (allo-HCT)**, which is a type of transplant using cells from a donor. The donor can be related or unrelated, and the cells can come from bone marrow, blood, or umbilical cord blood.
  • Must have a **Karnofsky Performance Status (KPS)** score of 60% or higher if 16 years or older, or a **Lansky Performance Status (LPS)** score of 60% or higher if younger than 16. These scores measure the ability to perform daily activities.
  • Must have adequate blood cell levels, specifically an **absolute neutrophil count (ANC)** of at least 0.5 x 109/L without the help of growth factors for at least 7 days before joining the study. Neutrophils are a type of white blood cell important for fighting infections.
  • Must agree to avoid pregnancy or fathering children during the study and for a period after the study. Men must take precautions to avoid fathering children and not donate sperm. Women who can become pregnant must have negative pregnancy tests and agree to use effective birth control methods. Women who cannot become pregnant are also eligible.

Who cannot join the trial?

7 criteria

  • Patients who have a different condition than chronic graft-versus-host disease cannot participate. This is a condition that can occur after a transplant, where the donated cells attack the patient's body.
  • Patients who are not in the age range specified for the study cannot participate. The study includes certain age groups, so if you are outside these groups, you cannot join.
  • Patients who are not willing or able to follow the study procedures cannot participate. This means you need to be able to attend appointments and follow instructions given by the study team.
  • Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
  • Patients who have another medical condition that might interfere with the study cannot participate. This is to make sure the study results are accurate and not affected by other health issues.
  • Patients who are taking certain medications that might interfere with the study cannot participate. Some medications can affect how the study treatment works.
  • Patients who have participated in another clinical trial recently cannot participate. This is to avoid any interference from other treatments or medications.
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Investigated drugs

  • Axatilimab

    This is an investigational medication being studied for its potential to help treat chronic graft-versus-host disease (cGVHD). It is being tested to see if it can improve symptoms when used alongside corticosteroids.

  • Corticosteroids

    These are a type of medication commonly used to reduce inflammation and suppress the immune system. In this trial, they are used in combination with axatilimab to treat chronic graft-versus-host disease.

What is already known about the treatment

Axatilimab – Axatilimab is administered through an intravenous infusion, meaning it is given directly into a vein. It is currently being studied in clinical trials as a potential treatment for chronic graft-versus-host disease (cGVHD), a condition that can occur after a stem cell or bone marrow transplant. The medication works by targeting and inhibiting a specific protein involved in the immune response, which may help reduce inflammation and tissue damage associated with cGVHD. Axatilimab is classified as a monoclonal antibody, a type of drug designed to interact with specific proteins in the body.

Investigated diseases

Chronic Graft-Versus-Host Disease – This condition occurs when donor cells attack the recipient's body after a stem cell or bone marrow transplant. It can affect various organs, including the skin, liver, and digestive tract. Symptoms may include skin rashes, dry eyes, and mouth, as well as joint pain. The disease can develop months or even years after the transplant. It often progresses slowly, with periods of worsening and improvement. The severity and specific symptoms can vary widely among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510292-65-00Protocol codeINCA34176-357Estimated enrolment240 patientsSponsorIncyte Corp.

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