NCT00092235 · National Cancer Institute (NCI)
Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease
What this study is about
Background: * Chronic graft-versus-host disease (cGVHD) is a multi-organ alloimmune and autoimmune disorder that occurs following allogeneic hematopoietic stem cell transplantation (alloHSCT). It is characterized by immune dysregulation, immunodeficiency, impaired organ function, and decreased survival.
View original scientific description
Background: * Chronic graft-versus-host disease (cGVHD) is a multi-organ alloimmune and autoimmune disorder that occurs following allogeneic hematopoietic stem cell transplantation (alloHSCT). It is characterized by immune dysregulation, immunodeficiency, impaired organ function, and decreased survival. * Each year about 8000 patients receive allogeneic hematopoietic stem cell transplant (alloHSCT) in North America and about 50% of patients who are transplanted develop cGVHD. * Chronic GVHD is also a disorder that simultaneously affects many organ systems in highly variable fashion and requires complex and coordinated medical management by multiple medical specialties. There is an urgent need for progress in understanding and effective treatments for cGVHD as it is one of the most serious complications of cancer therapy and hematopoietic stem cell transplantation. Objectives: * To establish a multidisciplinary clinic infrastructure for study of the pathogenesis and natural history of cGVHD. * To prospectively identify clinical and biological prognostic markers in patients with cGVHD * To develop clinically relevant cGVHD grading scales * To identify novel biological characteristics of cGVHD and to describe them in the context of clinical history and presentation * To identify potential clinical and biological markers of cGVHD activity * To improve understanding of the biology of cGVHD-associated graft-versus-tumor effects * To identify potential patients for cGVHD treatment protocols at the NCI and NIH Eligibility: -Patients age 1 and older referred by the primary transplant physician for the evaluation of chronic graft-versus-host disease independent of underlying diagnosis. Design: * Patient undergoes initial clinical and laboratory multispecialty work-up at the NCI cGVHD clinic. * Minimally invasive biopsies and rarely, deep tissue biopsy may be obtained to confirm the diagnosis and/or rule-out other pathologic process (in adults only).
Primary outcome measures
To prospectively identify candidate markers for clinical and biological prognostic factors in patients with cGVHD and develop a prognostic model
Time frame: 2 years + 3 months after protocol entry
Patient evaluations resulting in collection of data via several medical specialties; data will be examined individually and against clinical outcomes.
To improve our current understanding of the biology of cGVHD-associated graft-versus-tumor effects (GVT).
Time frame: ongoing
Studying mechanisms of how cGVHD exerts its anti-cancer effects via laboratory analysis.
To identify potential clinical and biological markers of cGVHD activity
Time frame: ongoing
Assessment of risk and outcome as related to molecular markers of pathogenesis and/or stage of disease.
To identify novel biological characteristics of cGVHD and to describe them in the context of clinical history and presentation
Time frame: ongoing
Through collection of data via several medical specialties, assess the weight of specific clinical and biological characteristics and disease severity scales for predicting major clinical outcomes.
To establish a multidisciplinary clinic infrastructure for study of pathogenesis and natural history of cGVHD
Time frame: ongoing
Assessment of clinical and biological characteristics of cGVHD.
To develop clinically relevant cGVHD grading scales
Time frame: ongoing
Develop appropriate staging as a tool for measuring responses or outcomes in clinical studies through prospective collection and analysis of data.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Any patient age 1 and older referred by the primary transplant physician for the evaluation of chronic graft-versus-host disease independently of age or underlying diagnosis
- Patient or the patient's legal representative is able and willing to provide consent.
Exclusion criteria
- Significant medical condition or any other significant circumstance that could in the PIs assessment affect the patient's ability to tolerate, comply, or complete the study
- Patients who in the PIs assessment have a life expectancy \<3 months. Note: Because it is not always possible to make a clear clinical distinction between acute and chronic GVHD, patients with acute GVHD are not a-priori excluded until the possibility of chronic GVHD is reliably excluded on the basis of the clinical assessments in the cGVHD clinic.
- Pregnant women are excluded from this study because multiple tests would need to be excluded for safety of the patient and the fetus. Inclusion Criteria for control subjects without cGVHD:
- Age 1 and older
- Patient has undergone Allo-HSCT
- Patient or the patient's parent/guardian is able and willing to provide consent Exclusion Criteria for control subjects without cGVHD:
- Active GVHD
- In the previous three months have received systemic immunosuppressant therapy for the treatment of GVHD
- In the previous three months have received therapy for malignancy
Where
- Bethesda, Maryland
Collaborators
Emory University, Department of Pediatrics
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Frequently asked questions
What is a clinical trial?
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Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
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Will I receive a placebo instead of treatment?
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Data: ClinicalTrials.gov · synced Jul 16, 2026 · Source of record for eligibility and locations