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.
1joining 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.
2treatment 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.
3monitoring 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.
4end 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 Study?
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 Study?
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.
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.
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.
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