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Blood & Immune

Chronic Graft-versus-Host Disease Clinical Trials

Also called chronic GVHD, cGVHD, chronic graft vs host disease, GVHD chronic

Chronic GVHD is driven by donor T cells and B cells that recognize recipient tissue antigens, initiating a multi-phase immune response involving both alloreactive immunity and aberrant immune reconstitution. The pathophysiology involves thymic damage (preventing normal tolerance mechanisms), B-cell dysregulation with autoantibody production, tissue fibrosis mediated by transforming growth factor beta and platelet-derived growth factor pathways, and macrophage-driven inflammation.

In plain words, chronic graft-versus-host disease is a problem that can happen after a stem cell or bone marrow transplant from a donor. The donor's immune cells attack the person's body and can harm the skin, eyes, mouth and other organs.

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Chronic Graft-versus-Host Disease

Where treatments stand, from ClinicalTrials.gov and FDA labels

FDA review0

No decision pending. FDA calendar

FDA-approved medicines4

Imbruvica · Jakafi · Niktimvo · Rezurock

Chronic Graft-versus-Host Disease has 4 FDA-approved medicines, 93 open or active clinical trials, and no pending FDA decision.

Labels checked October 21, 2026. Corticosteroids and immune globulins count once each.Download the data

Latest for Chronic Graft-versus-Host Disease

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More about Chronic Graft-versus-Host Disease

The disease is graded as mild, moderate, or severe based on the NIH Consensus Criteria, which assess the number of organs involved, the severity of involvement in each organ, and the functional impact. Mild disease may require only topical treatment, while moderate-to-severe disease typically requires systemic immunosuppression. Bronchiolitis obliterans syndrome, a manifestation of chronic GVHD in the lungs, is particularly concerning because it causes irreversible airway obstruction.

Keep reading about Chronic Graft-versus-Host Disease

Treatment options have grown since 2017. The FDA has approved 4 therapies for chronic GVHD after earlier systemic treatment has failed: ibrutinib (Imbruvica, approved 2017), ruxolitinib (Jakafi, approved 2021), belumosudil (Rezurock, approved 2021), and axatilimab-csfr (Niktimvo, approved August 2024). Each targets a different node in the GVHD immune cascade, providing options for patients who progress through first-line corticosteroid therapy.

What are the symptoms of Chronic Graft-versus-Host Disease?

These are common symptoms of Chronic Graft-versus-Host Disease, and how strong they are varies from person to person.

  • Skin rashes, thickening, tightening, or discoloration resembling scleroderma
  • Severe dry mouth, mouth sores, and difficulty eating
  • Dry, irritated eyes and light sensitivity
  • Shortness of breath from bronchiolitis obliterans (irreversible airway scarring)
  • Liver dysfunction with elevated bilirubin and liver enzymes
  • Joint and muscle stiffness limiting range of motion

Who gets Chronic Graft-versus-Host Disease?

Occurs exclusively in patients who have received an allogeneic (donor) stem cell or bone marrow transplant for blood cancers, bone marrow failure syndromes, or other hematologic conditions. Typically develops 3 months to 2 years after transplant.

Risk factors include older donor or recipient age, HLA mismatch, prior acute GVHD, peripheral blood stem cell source (vs. bone marrow), and female donor to male recipient.

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Which medicines are FDA-approved for Chronic Graft-versus-Host Disease?

There are currently 4 FDA-approved medications for Chronic Graft-versus-Host Disease.

ibrutinib
Pharmacyclics / AbbVie / Janssen
Search
ruxolitinib
Incyte Corporation
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axatilimab
Syndax Pharmaceuticals / Incyte Corporation
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belumosudil
Kadmon / Sanofi
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

How to get help paying for Chronic Graft-versus-Host Disease treatment

Charity funds and drugmaker programs for Chronic Graft-versus-Host Disease, checked at the source.

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Your insurance
Charity funds
  • From a charity · TotalAssist (formerly PAN Foundation)
    Graft-vs-Host Disease (GVHD) fund
    Open

    Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).

  • From a charity · NMDP (formerly Be The Match / National Marrow Donor Program)
    NMDP Patient Financial Assistance Grants fund
    Apply directly

    Pays for: Out-of-pocket costs before and after blood or marrow transplant, ongoing GVHD treatment costs, clinical-trial travel and financial-crisis grants.

    The foundation says: “Status not shown on page”
Status as each foundation showed it on October 26, 2026.

Every program and fund for Chronic Graft-versus-Host Disease, by insurance →

Side effects people have reported to the FDA

Side effects people have reported to the FDA

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Diagnosis and genetic testing

How Chronic Graft-versus-Host Disease is diagnosed

Caused by donor T cells reacting against recipient tissue after allogeneic transplant; risk rises with HLA mismatch, but there is no diagnostic genetic test for the disease itself.

Trusted Chronic Graft-versus-Host Disease resources

Reputable organizations and medical references for learning more about Chronic Graft-versus-Host Disease, including disease registries, foundation resources, and clinical guidelines.

Active clinical trials for Chronic Graft-versus-Host Disease

Use this Chronic Graft-versus-Host Disease clinical trial finder to see the 22 studies recruiting patients and 3 not yet recruiting in the United States and worldwide, with eligibility criteria in plain English. Of these, 16 test a treatment and 9 are observational studies or registries; each card says which. Every trial comes from ClinicalTrials.gov.

Jump to the trial list, the site map or the pipeline timeline.

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

93 active trials worldwide
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RecruitingTreatment trialPhase 2Recently updatedNCT07124078

A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

Intervention: INCA034176, Best available Treatment (BAT)

Sponsor: Incyte Corporation

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Ages 2 Years to 17 Years
Started May 2026Updated todayEst. Jan 2029 (~2y 2m)
RecruitingTreatment trialPhase 3Recently updatedNCT06585774

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

Intervention: INCA034176, Placebo, Corticosteroids

Sponsor: Incyte Corporation

This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).

Ages 12 Years+
Started Jan 2025Updated todayEst. Sep 2027 (~10 months)
RecruitingObservational study, no treatment givenRecently updatedNCT00092235

Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease

Sponsor: National Cancer Institute (NCI)

Background:

Ages 1 Year to 120 Years1 location
Started Oct 2004Updated yesterdayCompletion date not listed
Not yet recruitingTreatment trialPhase 1Recently updatedNCT07611370

Evaluating the Effects of Propionate and Butyrate Supplementation on the Intestinal Health of Healthy Volunteers

Intervention: Biospecimen Collection, Microencapsulated Sodium Butyrate, Microencapsulated Sodium Propionate

Sponsor: City of Hope Medical Center

This clinical trial evaluates how propionate and butyrate supplementation alters intestinal health in healthy volunteers and whether it would be feasible to administer these supplements to patients undergoing allogeneic hematopoietic stem cell transplant in the future. Propionate...

Ages 18 Years to 75 Years1 location
Expected to start Jan 2027Updated 3 days agoEst. Jun 2027 (~7 months)
RecruitingTreatment trialPhase 1Recently updatedNCT06996119

Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant

Intervention: Biospecimen Collection, Busulfan, Computed Tomography, Cyclophosphamide, Echocardiography Test, Emapalumab, Fludarabine, Hematopoietic Cell Transplantation, Melphalan, Multigated Acquisition Scan, Mycophenolate Mofetil, Questionnaire Administration, Tacrolimus

Sponsor: City of Hope Medical Center

This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome ...

Ages 18 Years to 75 Years1 location
Started Jun 2026Updated 4 days agoEst. Nov 2028 (~1y 11m)
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Active trial locations70 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Chronic Graft-versus-Host Disease patient communities

Facebook groups, Reddit communities and YouTube channels where Chronic Graft-versus-Host Disease patients, caregivers and advocacy groups talk.

Companies developing Chronic Graft-versus-Host Disease treatments

Drugmakers that list Chronic Graft-versus-Host Disease in their rare disease portfolio, with makers of its treatments first.

Questions people ask about Chronic Graft-versus-Host Disease