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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.

FDA-approved medicines

4

Imbruvica, Jakafi, Niktimvo, Rezurock

Active trials

95

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Next FDA decision

None set

FDA calendar

Medicines are those whose current FDA label names Chronic Graft-versus-Host Disease, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

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About Chronic Graft-versus-Host Disease

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.

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.

The treatment landscape has expanded significantly since 2017. 4 FDA-approved therapies are now available for chronic GVHD after failure of prior systemic treatment: 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.

Common Symptoms of Chronic Graft-versus-Host Disease

Recognizing the signs of Chronic Graft-versus-Host Disease early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • 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 Chronic Graft-versus-Host Disease Affects

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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Find trials I might fit Answer a few questions and see which open Chronic Graft-versus-Host Disease trials fit your age, location and treatment history.

FDA-Approved Treatments for Chronic Graft-versus-Host Disease

There are currently 4 FDA-approved medications for Chronic Graft-versus-Host Disease. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

ibrutinib
Pharmacyclics / AbbVie / Janssen
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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.

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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 12, 2026.

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Side Effect Explorer

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 Testing

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 21 studies recruiting patients and 4 opening soon in the United States and worldwide, with eligibility criteria in plain English. Of these, 17 test a treatment and 8 are observational studies or registries; each card says which. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
TrialsSite mapPipeline 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.

95 active trials worldwide
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RECRUITINGObservational study, no treatment givenRecently updatedNCT00106925

Long-term Evaluation and Follow-up Care of Patients Treated With Stem Cell Transplants

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

This study will provide follow-up evaluation and care of patients who have undergone allogeneic (donor) stem cell transplantation at the NIH Clinical Center. Patients are monitored for their response to treatment, disease relapse, and later-occurring effects of the transplant.

Ages 7 Years to 80 Years1 location
Started Apr 2005Updated 2 days agoCompletion date not listed
NOT YET RECRUITINGTreatment trialRecently updatedNCT05617625

CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

Intervention: Busulfan, Melphalan, Fludarabine, CliniMACS CD34+ enriched, T-cell depleted peripheral blood stem cell (PBSC)

Sponsor: Guenther Koehne

This study will evaluate whether processing blood stem cell transplants using an investigational device (the CliniMACS system) results in fewer complications for patients who undergo transplant to treat myelodysplastic syndrome (MDS).

Ages 18 Years to 72 Years1 location
Expected to start Jan 2027Updated 2 days agoEst. Jan 2034 (~7y 3m)
RECRUITINGObservational study, no treatment givenRecently updatedNCT00128973

Evaluation of Patients With Immune Function Abnormalities

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation. This may include inherited conditions, such as X-linked severe combined immunodeficiency (XSCID), chronic granulomatous disease (CGD), and leuk...

Ages not specified1 location
Started Sep 2005Updated 2 days agoCompletion date not listed
RECRUITINGTreatment trialPhase 2Recently updatedNCT06799195

Optimizing GVHD Prophylaxis After Allogeneic Hematopoietic Cell Transplantation

Intervention: Attenuated-dose Cyclophosphamide, High-dose Cyclophosphamide, Sirolimus, Mycophenolate Mofetil (MMF)

Sponsor: University of Nebraska

This study will compare post-transplant health-related quality of life following the use of standard versus attenuated dose of post-transplant cyclophosphamide in addition to two-drug graft-versus-host disease (GVHD) prophylaxis among recipients of allogeneic hematopoietic stem cell transplant.

Ages 60 Years+1 location
Started Jun 2025Updated 6 days agoEst. Apr 2031 (~4y 6m)
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+131 locations
Started Jan 2025Updated 6 days agoEst. Sep 2027 (~11 months)
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Find Chronic Graft-versus-Host Disease trials near you, ranked by distance →
Active trial locations62 cities in the US
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Trial Pipeline

Jan 2021 to Oct 2031
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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Chronic Graft-versus-Host Disease patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Chronic Graft-versus-Host Disease treatments, clinical trial participation, and day-to-day disease management.

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Related Blood & Immune Conditions

Other rare diseases in the blood & immune category. Patients with Chronic Graft-versus-Host Disease may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Chronic Graft-versus-Host Disease Treatments

12 pharmaceutical companies have Chronic Graft-versus-Host Disease in their rare disease portfolio

Frequently Asked Questions About Chronic Graft-versus-Host Disease