Ongoing Clinical Trials for Chronic Graft Versus Host Disease

Chronic graft versus host disease is a complication that can occur after a stem cell or bone marrow transplant, where donated immune cells attack the recipient’s body tissues. Currently, 12 clinical trials are investigating new treatments and comparing different medication approaches across multiple European countries. These studies focus on various medications including axatilimab, ruxolitinib, belumosudil, and ibrutinib, examining their effectiveness in patients who have not responded to previous treatments or are newly diagnosed with this condition.

Clinical trial locations

Comparison of Tacrolimus alone versus Tacrolimus, Mycophenolate mofetil and Prednisone combination in elderly kidney transplant patients to reduce infections

This study focuses on elderly patients aged 60 years and older who have received a kidney transplant from either a deceased or living donor. Patients can participate if they have had a previous kidney transplant, provided they meet all other criteria. Importantly, patients must not have donor-specific antibodies at the time of transplantation.

The trial compares two approaches to preventing organ rejection. One group receives tacrolimus alone, while the other receives the standard triple combination therapy of tacrolimus, mycophenolate mofetil, and prednisone. All medications are taken orally. The study aims to determine whether using fewer medications can reduce infection risks while maintaining adequate protection against transplant rejection.

Patients are excluded if they have severe heart, liver, or lung disease, active cancer or a history of cancer within the past 5 years, uncontrolled diabetes, or severe kidney dysfunction despite transplantation. Those with active or chronic infections, current pregnancy or breastfeeding, known allergies to immunosuppressive medications, or a history of organ rejection episodes in the past 6 months cannot participate.

The study will follow participants for three years after their kidney transplant, regularly monitoring kidney function, checking for viral infections such as cytomegalovirus and BK-virus, and assessing quality of life. Researchers will track the occurrence of infections, transplanted kidney function, and any complications that may arise during this period.

Continued Treatment Study for Patients with Myelofibrosis, Post-Lung Transplant BOS, or Chronic Graft-Versus-Host Disease Using Itacitinib

This study provides continued access to itacitinib for patients who have been participating in previous Incyte-sponsored clinical trials and are benefiting from this treatment. Itacitinib is a JAK inhibitor that works by blocking specific proteins involved in inflammation and immune responses.

Patients eligible for this continuation study must currently be enrolled in an Incyte-sponsored trial involving itacitinib, be tolerating the treatment well, and receiving clinical benefit as determined by their doctor. They must have demonstrated compliance with the previous study requirements and be willing to continue following study procedures.

The main focus is ensuring the medication remains safe and continues to provide benefits to those already experiencing positive effects. Participants take itacitinib in tablet form according to the prescribed schedule. Throughout the study, they have scheduled visits to monitor any side effects or adverse events that may occur.

The study is designed to run until 2027, providing long-term support and monitoring for participants who are benefiting from itacitinib. Regular assessments evaluate both the safety of the treatment and any changes in symptoms or overall health.

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

This trial evaluates a new treatment for moderate or severe chronic graft-versus-host disease using axatilimab combined with corticosteroids. Axatilimab is a monoclonal antibody that targets specific proteins involved in the immune response, potentially reducing inflammation and tissue damage.

Participants must be at least 12 years old with newly diagnosed moderate or severe disease requiring systemic treatment. They must have received an allogeneic hematopoietic cell transplant from any donor type and have adequate blood cell levels. The study uses a double-blind design, meaning neither participants nor researchers know who receives axatilimab or placebo.

Patients are excluded if they are pregnant or breastfeeding, have another medical condition that might interfere with the study, are taking certain medications that could affect study treatment, or have participated in another clinical trial recently. Those with severe liver or kidney problems, or active or untreated cancer, also cannot participate.

The study monitors participants through regular check-ups and assessments, tracking symptom improvements and any side effects. At six months, researchers evaluate whether patients have achieved complete or partial response without needing additional therapy. The trial continues until March 2030, gathering comprehensive data on treatment effectiveness and safety.

Study on Axatilimab and Ruxolitinib for Patients with Newly Diagnosed Chronic Graft-Versus-Host Disease

This research examines the combination of axatilimab and ruxolitinib for patients with newly diagnosed chronic graft-versus-host disease. Axatilimab is administered either intravenously or subcutaneously, while ruxolitinib is taken orally as tablets. Together, these medications target different aspects of the immune response.

Participants must be 12 years or older with new-onset moderate or severe disease according to 2014 NIH Consensus Criteria. They must have a history of one allogeneic stem cell transplant and require treatment with corticosteroids for their condition. Previous systemic treatments for the disease are not permitted, though certain immunosuppressive medications at low doses may be continued.

The study excludes patients who are pregnant or breastfeeding, have participated in another clinical trial recently, are taking medications that might interfere with study drugs, or have had a recent infection or serious health condition that might affect results. Those outside the specified age range or unable to follow study procedures also cannot participate.

The primary goal is achieving an overall response at 6 months, defined as complete or partial response without the need for new systemic therapy. Participants undergo regular monitoring to assess treatment safety and effectiveness, including evaluation of clinical and laboratory parameters. Extended observation continues for up to 12 months to assess long-term outcomes.

Study on Ruxolitinib and Methoxsalen for Patients with Steroid-Refractory Chronic Graft-versus-Host Disease

This trial combines ruxolitinib with extracorporeal photopheresis for patients whose disease has not responded to steroid treatments. Ruxolitinib is a JAK inhibitor taken orally that helps reduce inflammation by targeting specific immune system pathways. Extracorporeal photopheresis is a process where blood is treated outside the body to modify immune responses.

Eligible participants are adults 18 years or older who have received a stem cell transplant and have clinically diagnosed moderate to severe disease. They must have been treated with systemic glucocorticoids for less than 6 months and meet specific criteria for steroid-refractory disease. Blood cell counts must reach certain levels, and women of childbearing potential must have negative pregnancy tests.

Patients who have not been diagnosed with steroid-refractory disease, are outside the specified age range, or belong to vulnerable populations are excluded from the study. The trial aims to evaluate the safety and effectiveness of this combined treatment approach for patients who have not responded to standard steroid treatments.

Treatment is organized into cycles, with the primary evaluation occurring at week 25. Researchers assess the Overall Response Rate, measuring the proportion of patients showing complete or partial response relative to baseline. Secondary assessments include failure-free survival, changes in symptom scores, overall survival, and the best overall response at any time during the trial.

Study of Axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments

This research compares axatilimab to current standard treatments in children and teenagers aged 2 to 17 years who have already tried at least two other treatments. Axatilimab is a monoclonal antibody that targets the CSF-1R pathway, which plays a role in immune cell development and function related to the disease.

Participants must have active moderate to severe disease requiring systemic treatment and a history of allogeneic stem cell transplant. They must have received at least two previous treatments, including corticosteroids and ruxolitinib, and have a performance score of 60 or higher. Adequate blood cell counts are required, and participants may continue taking corticosteroids at a stable dose.

The study excludes patients with active or uncontrolled infections, severe liver or kidney problems, uncontrolled heart conditions, active or untreated cancer, pregnancy or breastfeeding, or use of other experimental medications within 30 days. Those with severe mental health conditions, known allergic reactions to similar medications, or very low blood cell counts also cannot participate.

Participants receive either axatilimib through intravenous infusion or one of several commonly used standard treatments. The study monitors response to treatment at 6 months and 12 months, tracks how well participants can reduce corticosteroid use, and assesses quality of life. The trial is expected to continue until January 2029.

Study of belumosudil in children aged 1 to 18 years with moderate to severe chronic graft versus host disease requiring systemic treatment

This trial investigates belumosudil, a ROCK2 kinase inhibitor, in children with moderate to severe disease requiring systemic treatment. The medication helps manage immune system responses that cause inflammation and tissue damage after stem cell or bone marrow transplantation. Belumosudil is available as film-coated tablets or oral suspension.

The study is divided into two phases. Phase 1 establishes the appropriate dose for children aged 1 to 12 years, while Phase 2 evaluates effectiveness in participants aged 1 to 18 years. Participants must have a life expectancy exceeding 6 months, weigh at least 8 kilograms, and have received an allogeneic stem cell transplant. The disease must not be responding to current treatment or has returned after at least 2 different treatments.

Patients are excluded if they have active and uncontrolled infections requiring treatment, severe liver problems, severe kidney problems requiring dialysis, uncontrolled heart conditions, active or untreated cancer, pregnancy or breastfeeding, or use of other experimental medications within 30 days. Those with blood disorders resulting in very low blood cell counts or history of serious bleeding problems also cannot participate.

Treatment response is evaluated by Week 25 or Cycle 7 Day 1. Participants take the medication orally, through a nasogastric tube, or through a feeding tube. Current medications for the disease must remain stable for at least 2 weeks before starting this treatment. Regular check-ups assess response and document any health changes.

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

This open-label, randomized study compares axatilimab to best available therapy in patients aged 12 and older whose disease has not responded to or has returned after at least two different treatments. Best available therapy may include medications such as cyclosporine, tacrolimus, extracorporeal photopheresis, mycophenolate mofetil, or other commonly used treatments.

Participants must have active, moderate to severe disease requiring immunosuppressive treatment and a history of allogeneic stem cell transplant. The disease must not have responded to or has recurred after at least two previous treatments, including corticosteroids and ruxolitinib. Participants need a physical ability score of 60 or higher and can continue using systemic corticosteroids during the study.

Exclusion criteria include other serious medical conditions affecting study participation, pregnancy or breastfeeding, recent use of investigational drugs, history of severe allergic reactions to similar medications, inability to comply with study procedures, active infections requiring treatment, uncontrolled high blood pressure, significant heart disease, or unstable liver or kidney problems.

Axatilimab is administered through intravenous infusion according to the study protocol. Participants undergo regular monitoring and assessments to evaluate treatment effectiveness and safety through clinical assessments and laboratory tests. The study assesses response at specific time points such as 6 months and 12 months to determine complete or partial response.

Study of Axatilimab for Patients with Chronic Graft Versus Host Disease After Two Prior Treatments

This trial evaluates axatilimab at three different doses in patients aged 18 and older whose disease has not responded to at least two previous treatments. The doses being tested are 0.3 mg/kg every two weeks, 1 mg/kg every two weeks, or 3 mg/kg every four weeks, all administered through intravenous infusion.

Participants must have received an allogeneic hematopoietic stem cell transplant and have active disease requiring systemic immunosuppressive treatment. The disease must be refractory or recurrent after trying at least 2 different treatments. Patients may have overlap syndrome with symptoms of both acute and chronic forms. A Karnofsky Performance Scale score of 60 or higher is required, along with adequate organ and bone marrow function.

The study monitors how well the treatment works and tracks any side effects. Regular check-ups include vital signs, physical examinations, and evaluation of response to treatment. Participants can continue using calcineurin inhibitors, mTOR inhibitors, or systemic corticosteroids if the corticosteroid dose has been stable for at least 2 weeks before starting the study.

The primary goal is evaluating the overall response rate during the first six cycles of treatment. Secondary evaluations include assessing specific health parameters and any side effects experienced. Upon completing treatment cycles, a final assessment evaluates overall health outcomes and determines any further steps or follow-up care required.

Study of Belumosudil and Prednisone for Patients Aged 12 and Older with Newly Diagnosed Chronic Graft Versus Host Disease

This double-blind, randomized trial compares belumosudil combined with corticosteroids against placebo combined with corticosteroids in patients aged 12 and older with newly diagnosed disease. Belumosudil is a kinase inhibitor that works by blocking enzymes involved in immune response signaling pathways that lead to inflammation.

Participants must have newly diagnosed moderate to severe disease requiring corticosteroid treatment and should not have received any previous systemic treatment for the condition, including extracorporeal photopheresis. They must have had an allogeneic stem cell transplant, weigh at least 40 kilograms, and use appropriate birth control methods according to local guidelines.

If participants are taking other immunosuppressive medications to prevent or treat acute graft-versus-host disease, the dose should be below a certain level specified in the study protocol. Both men and women can participate, and participants or their legally authorized representative must provide signed informed consent after understanding what the study involves.

The study’s primary focus is measuring event-free survival, which is the time from randomization to the occurrence of any predefined event. Participants take the study medication or placebo orally according to the protocol. Regular monitoring assesses treatment effectiveness and safety, and the study is estimated to conclude by September 2028.

Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies

This open-label, multi-center trial evaluates long-term safety of treatments for patients who previously participated in studies involving ruxolitinib, either alone or combined with panobinostat, siremadlin, or rineterkib. All medications are administered orally in capsule or tablet form.

Participants must currently be enrolled in a Novartis or Incyte-sponsored study receiving treatment with these medications and benefiting from the treatment as determined by the investigator. They must have met all requirements of their original study and continue to follow all study guidelines.

The main objective is evaluating long-term safety data, focusing on serious adverse events and adverse events. The frequency and severity of these events are monitored throughout the study. Regular visits allow investigators to assess clinical benefits of treatment and determine the proportion of patients experiencing such benefits.

Ruxolitinib is a JAK inhibitor used to treat certain blood disorders. Panobinostat is a histone deacetylase inhibitor often used in combination with other treatments for cancer. Siremadlin is an MDM2 inhibitor being investigated for cancer treatment. Rineterkib is a kinase inhibitor also under investigation for cancer. The study is expected to continue until September 2027.

Summary

The current clinical trial landscape for chronic graft-versus-host disease shows significant research activity across Europe, with the majority of trials concentrated in Germany, Italy, Spain, France, and Belgium. A notable trend is the strong focus on axatilimab, which appears in multiple studies either as monotherapy or in combination with other agents, suggesting considerable interest in this monoclonal antibody approach.

Several trials specifically target pediatric populations, addressing an important gap in treatment options for children and young adults with this condition. These include studies investigating belumosudil, axatilimab, and ibrutinib in younger age groups, with some trials including participants as young as 1 year old.

The research covers a range of treatment scenarios: some trials focus on newly diagnosed patients requiring initial systemic therapy, while others target patients whose disease has not responded to at least two previous treatments. This variety reflects different stages of disease management and the need for multiple treatment options.

Combination therapies represent another important research direction, with several trials examining whether adding novel agents to standard corticosteroid treatment or combining different investigational medications can improve outcomes. Studies comparing new treatments to best available therapy provide important evidence about whether novel approaches offer advantages over current standards of care.

The geographic distribution shows strong representation in Western and Southern European countries, with limited participation from Eastern European nations. Most trials are expected to continue through 2027-2030, indicating a long-term commitment to understanding these treatments’ safety and effectiveness. The diversity of medications being studied, including JAK inhibitors, ROCK inhibitors, and monoclonal antibodies, demonstrates multiple therapeutic approaches being explored simultaneously.

#1 Clinical Trials Search in Europe

Chronic graft versus host disease – Trials in Disease

Ongoing Clinical Trials for Chronic Graft Versus Host Disease

Clinical trial locations

Comparison of Tacrolimus alone versus Tacrolimus, Mycophenolate mofetil and Prednisone combination in elderly kidney transplant patients to reduce infections

Continued Treatment Study for Patients with Myelofibrosis, Post-Lung Transplant BOS, or Chronic Graft-Versus-Host Disease Using Itacitinib

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

Study on Axatilimab and Ruxolitinib for Patients with Newly Diagnosed Chronic Graft-Versus-Host Disease

Study on Ruxolitinib and Methoxsalen for Patients with Steroid-Refractory Chronic Graft-versus-Host Disease

Study of Axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments

Study of belumosudil in children aged 1 to 18 years with moderate to severe chronic graft versus host disease requiring systemic treatment

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

Study of Axatilimab for Patients with Chronic Graft Versus Host Disease After Two Prior Treatments

Study of Belumosudil and Prednisone for Patients Aged 12 and Older with Newly Diagnosed Chronic Graft Versus Host Disease

Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies

Summary

Ongoing Clinical Trials on Chronic graft versus host disease

Study of Axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments

A Study of Belumosudil for Patients with Steroid-Resistant Overlap Syndrome Graft-versus-Host Disease

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

Study on Ruxolitinib and Methoxsalen for Patients with Steroid-Refractory Chronic Graft-versus-Host Disease

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

Study of Axatilimab for Patients with Chronic Graft Versus Host Disease After Two Prior Treatments

Study on Axatilimab and Ruxolitinib for Patients with Newly Diagnosed Chronic Graft-Versus-Host Disease

Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies

Study on the Safety and Effectiveness of Ibrutinib for Children and Young Adults with Chronic Graft Versus Host Disease

Continued Treatment Study for Patients with Myelofibrosis, Post-Lung Transplant BOS, or Chronic Graft-Versus-Host Disease Using Itacitinib

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Chronic graft versus host disease – Trials in Disease

Ongoing Clinical Trials for Chronic Graft Versus Host Disease

Clinical trial locations

Summary

Ongoing Clinical Trials on Chronic graft versus host disease

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