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

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ClinicalTrials.gov Identifier: NCT00092235
Recruitment Status : Recruiting
First Posted : September 22, 2004
Last Update Posted : November 7, 2023
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

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

Condition or disease
Chronic Graft vs. Host Disease

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

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Study Type : Observational
Estimated Enrollment : 650 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease
Actual Study Start Date : October 26, 2004


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



Primary Outcome Measures :
  1. 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. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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.
Criteria
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00092235


Contacts
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Contact: Steven Z Pavletic, M.D. (240) 760-6174 sp326h@nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Steven Z Pavletic, M.D. National Cancer Institute (NCI)
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00092235    
Obsolete Identifiers: NCT00331968
Other Study ID Numbers: 040281
04-C-0281
First Posted: September 22, 2004    Key Record Dates
Last Update Posted: November 7, 2023
Last Verified: November 1, 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
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.
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.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
cGVHD-associated graft-versus-tumor effects (GVT)
Allo-HSCT controls
Leukapheresis
Natural History
Additional relevant MeSH terms:
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Bronchiolitis Obliterans Syndrome
Graft vs Host Disease
Immune System Diseases
Organizing Pneumonia
Bronchiolitis Obliterans
Bronchiolitis
Bronchitis
Bronchial Diseases
Respiratory Tract Diseases
Lung Diseases, Obstructive
Lung Diseases