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

May 7, 2024 updated by: National Cancer Institute (NCI)

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

Study Overview

Status

Recruiting

Conditions

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

Study Type

Observational

Enrollment (Estimated)

650

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Steven Z Pavletic, M.D.
  • Phone Number: (240) 760-6174
  • Email: sp326h@nih.gov

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Recruiting
        • National Institutes of Health Clinical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients will be selected via referrals or from patients who were on companion clinical protocols at the NIH.

Description

  • 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 less than 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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
Studying mechanisms of how cGVHD exerts its anti-cancer effects via laboratory analysis.
ongoing
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.
ongoing
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.
ongoing
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.
ongoing
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.
ongoing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Cancer Institute (NCI)

Investigators

  • Principal Investigator: Steven Z Pavletic, M.D., National Cancer Institute (NCI)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 26, 2004

Study Registration Dates

First Submitted

September 21, 2004

First Submitted That Met QC Criteria

September 21, 2004

First Posted (Estimated)

September 22, 2004

Study Record Updates

Last Update Posted (Actual)

May 8, 2024

Last Update Submitted That Met QC Criteria

May 7, 2024

Last Verified

February 7, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 040281
  • 04-C-0281

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

.All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.

IPD Sharing Time Frame

Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.

IPD Sharing Access Criteria

Data from this study may be requested by contacting the PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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