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Study Comparing Axatilimab to Best Available Therapy for Patients with Chronic Graft-Versus-Host Disease After Two or More Treatments

Verified siteInvestigationalNo 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 Chronic Graft-Versus-Host Disease (cGVHD), a condition that can occur after a stem cell or bone marrow transplant. In this disease, the donated cells attack the recipient's body, leading to various symptoms. The study will compare the effectiveness of a new treatment called Axatilimab (INCA034176), which is given as a solution for infusion, against the best available therapy currently used for cGVHD.

The purpose of the study is to see how well Axatilimab works compared to other treatments. Participants in the study will receive either Axatilimab or one of the existing treatments, which may include medications like cyclosporine, tacrolimus, or other therapies. The study will monitor participants over a period to assess their response to the treatment and any changes in their condition.

Throughout the study, participants will be observed for improvements in their symptoms and any side effects they may experience. The goal is to determine if Axatilimab can provide a better outcome for those with cGVHD who have already tried at least two other treatments. This research aims to offer new hope for managing this challenging condition.

The research process

The trial runs in 4 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, participants will be randomly assigned to receive either axatilimab or the best available therapy (BAT). This is a randomized study, meaning the treatment is assigned by chance, like flipping a coin.

    Participants will be informed about the treatment they will receive, as this is an open-label study, meaning both the participants and the researchers know which treatment is being administered.

  2. Step 2

    Treatment administration

    Participants receiving axatilimab will have it administered through an intravenous infusion. This means the medication is given directly into a vein using a needle.

    The frequency and dosage of axatilimab will be determined by the study protocol and communicated to the participants by the study team.

  3. Step 3

    Monitoring and assessments

    Participants will undergo regular monitoring and assessments to evaluate the effectiveness and safety of the treatment. This includes clinical assessments and laboratory tests.

    The study will assess the response to treatment at specific time points, such as 6 months and 12 months, to determine if there is a complete or partial response to the therapy.

  4. Step 4

    End of treatment evaluation

    At the end of the treatment period, participants will have a final evaluation to assess the overall response to the therapy and any changes in their condition.

    Participants will be informed about the results of their treatment and any further steps or follow-up that may be necessary.

Who can join the trial?

10 criteria

  • Must be at least 12 years old.
  • Must understand and be willing to sign a written consent form to participate in the study. If the participant is a child, a parent or guardian must provide consent, and the child should also sign a form if possible.
  • Must have active, moderate to severe chronic Graft-Versus-Host Disease (cGVHD) that requires treatment with medications that affect the immune system.
  • Must have had a history of a type of stem cell transplant called allo-HCT from any donor type, using any source of stem cells (like bone marrow, blood, or cord blood).
  • Must have cGVHD that has not responded to or has returned after at least two different treatments, including corticosteroids and ruxolitinib.
  • May have a type of cGVHD that includes features of both acute and chronic forms, as defined by specific criteria.
  • Must have a certain level of physical ability, measured by a score of 60 or higher on a scale used for people aged 16 and older, or a similar score for those younger than 16.
  • Can continue using systemic corticosteroids during the study.
  • Must agree to be treated with one of the following options at the start of the study: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, pentostatin, proteasome inhibitors, imatinib, or ibrutinib.
  • Must be willing to avoid becoming pregnant or fathering children during the study.

Who cannot join the trial?

10 criteria

  • Patients with any other serious medical condition that might affect their participation in the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have received another investigational drug within a certain period before the study.
  • Patients with a history of severe allergic reactions to similar medications.
  • Patients who are unable to comply with the study procedures.
  • Patients with active infections that require treatment.
  • Patients with uncontrolled high blood pressure.
  • Patients with significant heart disease.
  • Patients with liver or kidney problems that are not stable.
  • Patients who have had a recent organ transplant.
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Investigated drugs

  • Axatilimab

    is a medication being studied for its potential to help people with chronic graft-versus-host disease (cGVHD), a condition that can occur after a stem cell or bone marrow transplant. This medication works by targeting specific cells in the immune system that may be causing inflammation and damage in the body. The goal of using axatilimab in this trial is to see if it can reduce the symptoms of cGVHD and improve the quality of life for patients who have not responded well to other treatments.

  • Best Available Therapy (BAT)

    refers to the standard treatments that are currently used for managing chronic graft-versus-host disease. These treatments can vary depending on the patient's specific needs and the healthcare provider's recommendations. The purpose of including BAT in this study is to compare its effectiveness with axatilimab, to determine which option provides better outcomes for patients with cGVHD. BAT may include a variety of medications or therapies that are commonly used to control the symptoms of this condition.

What is already known about the treatment

Axatilimab – Axatilimab is administered as an injection, typically given under the skin. It is currently being studied in clinical trials and is not yet widely available in medical practice. The main use of axatilimab is to treat chronic graft-versus-host disease, a condition that can occur after a stem cell or bone marrow transplant. At the molecular level, axatilimab works by targeting and blocking a specific protein involved in the immune response, which helps reduce inflammation and tissue damage. It is classified as a monoclonal antibody, a type of medication designed to target 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 multiple organs, including the skin, liver, and digestive tract. The disease often begins with skin rashes and can progress to cause joint stiffness and dry eyes or mouth. Over time, it may lead to more severe complications such as liver dysfunction and lung problems. The progression varies widely among individuals, with some experiencing mild symptoms and others facing more significant challenges. The disease can persist for months or even years, requiring ongoing management.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-518973-32-00Protocol codeINCA034176-355Estimated enrolment300 patientsSponsorIncyte Corp.

sourced from the EU Clinical Trials Register and site verification

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