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Study of Axatilimab for Patients with Chronic Graft Versus Host Disease After Two Prior 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 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 treatment being tested in this study is a medication called Axatilimab, which is given as a solution through an intravenous infusion. The study aims to evaluate the effectiveness, safety, and tolerability of Axatilimab at three different doses in patients who have cGVHD that has not responded to at least two previous treatments.

Participants in the study will receive Axatilimab at one of three doses: 0.3 mg/kg every two weeks, 1 mg/kg every two weeks, or 3 mg/kg every four weeks. The study will observe how well the treatment works and monitor any side effects. The goal is to see if Axatilimab can help reduce the symptoms of cGVHD and improve the quality of life for those affected by this condition.

The study will take place over a period of time, with regular check-ups and assessments to track the participants' progress. This includes monitoring vital signs, conducting physical examinations, and evaluating the response to the treatment. The information gathered from this study will help determine the potential benefits of Axatilimab for patients with cGVHD who have limited treatment options.

The research process

The trial runs in 6 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, eligibility is confirmed based on specific criteria, including age, medical history, and current health status.

    Informed consent is required, ensuring understanding of the study's requirements and restrictions.

  2. Step 2

    Randomization and initial assessment

    Participants are randomly assigned to one of three dosage groups for the medication axatilimab.

    Initial health assessments are conducted to establish baseline health metrics.

  3. Step 3

    Treatment phase

    Participants receive axatilimab through intravenous infusion.

    Dosage varies: 0.3 mg/kg every two weeks, 1 mg/kg every two weeks, or 3 mg/kg every four weeks.

    The treatment continues for a specified number of cycles, with each cycle lasting a set period.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring of health status, including vital signs and laboratory tests, is conducted to assess the medication's effects.

    Participants may continue using certain other medications, such as corticosteroids, if already prescribed.

  5. Step 5

    Evaluation of response

    The primary goal is to evaluate the overall response rate to axatilimab during the first six cycles.

    Secondary evaluations include assessing specific health parameters and any side effects experienced.

  6. Step 6

    Completion of study participation

    Upon completing the treatment cycles, a final assessment is conducted to evaluate the overall health outcomes.

    Participants receive information on any further steps or follow-up care required.

Who can join the trial?

11 criteria

  • Patient must be 18 years of age or older at the time of signing the informed consent.
  • Patients who have received a type of transplant called allogeneic HSCT and have active chronic graft versus host disease (cGVHD) that needs treatment with medicines that suppress the immune system.
  • Patients with refractory (not responding to treatment) or recurrent (comes back after treatment) active cGVHD, even after trying at least 2 different treatments.
  • Patients may have ongoing symptoms of both acute and chronic GVHD at the same time, known as overlap syndrome.
  • Patients must have a Karnofsky Performance Scale score of 60 or higher if they are 16 years or older, or a Lansky Performance Score of 60 or higher if they are younger than 16. These scores measure how well a person can perform daily activities.
  • Patients must have adequate organ and bone marrow function, which will be checked within 14 days before starting the study.
  • Patients must have a creatinine clearance (a measure of kidney function) of 30 milliliters per minute or higher, calculated using specific formulas for adults and children.
  • Both male and female participants are allowed. If applicable, participants must use contraception according to local guidelines.
  • Patients can use a type of medication called calcineurin inhibitors or mTOR inhibitors (like sirolimus or everolimus), but it is not required.
  • Patients can use systemic corticosteroids (a type of medication) but it is not required. If they are using corticosteroids, the dose must be stable for at least 2 weeks before starting the study.
  • Patients must be able to give signed informed consent, which means they understand and agree to follow the study's rules. For children, a parent or guardian must provide consent, and if possible, the child should also agree.
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Investigated drugs

Axatilimab is a medication being studied for its effectiveness, safety, and tolerability in patients with chronic graft versus host disease (cGVHD) who have not responded to at least two previous treatments. This medication is administered through an intravenous infusion and is being tested at different doses to determine the best approach for managing cGVHD symptoms.

What is already known about the treatment

Axatilimab – Axatilimab is administered intravenously, typically at varying doses depending on the treatment plan. It is currently being studied in clinical trials for its effectiveness in treating chronic graft versus host disease (cGVHD), particularly in patients who have not responded to at least two previous systemic therapies. The medication works by targeting specific immune cells to reduce inflammation and tissue damage associated with cGVHD. Axatilimab is classified as a monoclonal antibody, which is a type of biologic therapy designed to interfere with specific pathways in the immune system.

Investigated diseases

Chronic Graft Versus Host Disease (cGVHD) – This is a condition that can occur after a stem cell or bone marrow transplant, where the donated cells attack the recipient's body. It typically develops several months after the transplant and can affect various organs, including the skin, liver, and digestive tract. Symptoms may include skin rashes, dry mouth, and joint pain. The disease can vary in severity, with some individuals experiencing mild symptoms and others facing more significant health challenges. Over time, cGVHD can lead to chronic inflammation and tissue damage in the affected areas. The progression of the disease can be unpredictable, with periods of improvement and worsening symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-512978-99-00Protocol codeSNDX-6352-0504Estimated enrolment240 patientsSponsorSyndax Pharmaceuticals Inc.

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