Recruiting

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

Sponsor:

National Cancer Institute (NCI)

Code:

NCT00092235

Conditions

Chronic Graft vs. Host Disease

Eligibility Criteria

Sex: All

Age: 1 - 70+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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).
Long tem data collection for evaluation of long-term outcomes will be conducted anually as feasible

Conditions

Chronic Graft vs. Host Disease

Study ID

NCT00092235

Start date

Oct 26, 2004

Status verified date

Aug 31, 2026

Eligibility Criteria

Sex: All

Age: 1 - 70+

Healthy Volunteers: Not accepted

INCLUSION CRITERIA:

1. 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
2. Patient or the patient's legal representative is able and willing to provide consent.

EXCLUSION CRITERIA:

1. 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
2. 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.
3. 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

Study Design

Enrollment

650 participants

Anticipated

Interventions and Outcome Measures

Arms

Cohort 1

Patients who have undergone an allogeneic stem cell transplant and are diagnosed with cGVHD

Cohort 2

Pediatric patients who have undergone an allogeneic stem cell transplant and are diagnosed with cGVHD

Cohort 3

Patients who have undergone an allogeneic stem cell transplant and choose to submit biopsy, blood and urine samples only

Cohort 4

Patients who have undergone an allogeneic stem cell transplant and are not diagnosed with cGVHD

Primary outcome measure

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 ]
To improve our current understanding of the biology of cGVHD-associated graft-versus-tumor effects (GVT). [ Time Frame: ongoing ]
To identify potential clinical and biological markers of cGVHD activity [ Time Frame: ongoing ]
To identify novel biological characteristics of cGVHD and to describe them in the context of clinical history and presentation [ Time Frame: ongoing ]
To establish a multidisciplinary clinic infrastructure for study of pathogenesis and natural history of cGVHD [ Time Frame: ongoing ]
To develop clinically relevant cGVHD grading scales [ Time Frame: ongoing ]

Central Contacts and Locations

Central contacts

Najla El Jurdi, M.D.

(240) 992-4033 najla.eljurdi@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

More Information

Sponsor

National Cancer Institute (NCI)

Last update posted

Sep 8, 2026

Last verified

Aug 31, 2026

Keywords

cGVHD-associated graft-versus-tumor effects (GVT)
Allo-HSCT controls
Leukapheresis
Natural History

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Cancer Institute (NCI) on 2026-09-08.