Chronic Graft-versus-Host Disease in the Oral Cavity of Patients Following Allogeneic Hematopoietic Stem Cell Transplant and Including Healthy Controls
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|ClinicalTrials.gov Identifier: NCT03602599|
Recruitment Status : Recruiting
First Posted : July 27, 2018
Last Update Posted : October 18, 2019
This study follows people who have had, or will soon have, a transplant using stem cells from another person. This is known as an allogeneic hematopoietic stem cell transplant (HSCT). Graft-versus-host disease (cGVHD) can happen after HSCT. cGVHD can cause mouth problems and more serious issues. Researchers want to study changes in the mouth that might indicate cGVHD.
To identify cGVHD in the mouth and better understand the development, treatment, and progress of post-transplant changes in the mouth.
Adults at least 18 years old who will soon undergo HSCT or have had one in the past 3 years Healthy adults at least 18 years old
All participants will have a screening visit and baseline visit. They will last 60-90 minutes. Over these two visits, participants will have:
Medical and dental history
Questions about their eating habits and general health
Blood drawn through a needle in the arm
Vital signs taken
Pictures of their mouth and lips taken
Questions about their oral health, including about pain, sensitivity, or dryness
Saliva samples taken. Participants will spit into a sterile plastic tube.
Swabs taken of the mouth and some of the saliva, plaque, and fluid from the spaces between teeth and gums.
Participants may also have:
A piece of skin taken (biopsy) from the inner lining of the cheeks
A piece of skin taken (biopsy) from the lower lip
Urine pregnancy test
Most participants will have at least 7 study visits over 3 years. They will meet with a dentist and repeat baseline tests.
|Condition or disease|
|Graft Versus Host Disease|
- Chronic graft-versus-host disease (cGVHD) is a multi-organ severe alloimmune and - autoimmune disorder that occurs after allogeneic hematopoietic stem cell transplantation (HSCT). It is characterized by immune dysregulation, immunodeficiency, impaired organ function, and decreased survival.
- Manifestations of cGVHD in the oral cavity are observed in 45-85% of cGVHD patients and include 3 components: limitation of mouth opening, oral mucosal changes (lichenoid lesions, ulceration) and salivary gland changes.
- There is an urgent need for new tools to diagnose and treat oral cGVHD. To develop targeted therapies and non-invasive methods for serial screening, an improved understanding of the clinical course and underlying mechanisms of oral cGVHD are required.
- Advance understanding of cGVHD pathophysiology and generate hypotheses for future cGVHD studies by tracking the longitudinal development of cGVHD in the oral cavity and transplant-related changes in the oral microenvironment.
- Leverage knowledge about the impact of transplant on the oral cavity to develop better protocols for supportive dental care, to refine clinical definitions and classifications of oral cGVHD, and to test and improve criteria and tools for clinical trials.
- Include healthy volunteer cohorts both as a direct control group for HSCT patients, and to characterize measures of interest that were not previously defined in the normal oral cavity.
- Patients who are scheduled to undergo allogeneic HSCT at the National Institutes of Health (NIH) or who have already undergone allogeneic HSCT at the NIH or elsewhere.
- Healthy individuals who have not undergone transplant and are willing to undergo oral exam and sample collection will be included in a control group.
- All study subjects, including patients and healthy individuals, must be able to provide written informed consent and be willing to return to the NIH Dental Clinic for scheduled evaluations.
- This is a single site, observational study with four cohorts. There are 2 transplant cohorts: the New Transplant Cohort ("Cohort NT"; approximate n=300) consists of patients who are scheduled to undergo allogeneic HSCT (under another protocol at the NIH) and the Prior Transplant Cohort ("Cohort PT"; approximate n=100) consists of patients who have already undergone allogeneic HSCT. These Cohorts will have up to 8 regularly scheduled visits across 3 years, along with a variable number of acute-episode visits. Additionally, subjects will have the opportunity to participate in and optional annual follow-up visits in years 4-10 post transplant.
- The other 2 Cohorts include healthy volunteers: the Healthy-controls Longitudinal (HL) Cohort ("Cohort HL"; approximate n=20) includes subjects who will participate in up to the full set of 8 study visits across 3 years, and the Healthy-controls Short-term (HS) Cohort ("Cohort HS"; approximate n=80) will participate in a single baseline visit.
- Each visit will include collection of biologic samples, patient-reported data and clinical assessments.
- Initial statistical analysis will begin with unsupervised clustering methods and simple logistic regression to identify biomarkers important in oral cGVHD. Based upon the results of these initial analyses, more complex multivariate statistical models will be developed to both classify and predict onset of oral cGVHD. Given the hypothesisgenerating nature of this study, additional prospective statistical analysis plans will be developed in consultation with a statistician as the study progresses to address specific scientific questions.
|Study Type :||Observational|
|Estimated Enrollment :||500 participants|
|Official Title:||Natural History of Chronic Graft-versus-Host Disease in the Oral Cavity of Patients Following Allogeneic Hematopoietic Stem Cell Transplant and Including Healthy Controls|
|Actual Study Start Date :||November 15, 2018|
|Estimated Primary Completion Date :||May 31, 2023|
|Estimated Study Completion Date :||May 31, 2023|
Healthy-controls Longitudinal Cohort
("Cohort HL"; approximate n=20) includes subjects who will participate in up to the full set of 8 study visits across 3 years.
Healthy-controls Short-term Cohort
("Cohort HS"; approximate n=80) will participate in a single baseline visit.
New Transplant Cohort
("Cohort NT"; approximate n=300) consists of patients who are scheduled to undergo allogeneic HSCT (under another protocol at the NIH).
Prior Transplant Cohort
("Cohort PT"; approximate n=100) consists of patients who have already undergone allogeneic HSCT.
- oral cGVHD onset [ Time Frame: 3 years ]This study is designed to track the longitudinal development of chronic graft-versus-host disease in the oral cavity.
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): NCT03602599
|Contact: Jacqueline W Mays, D.D.S.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Jacqueline W Mays, D.D.S.||National Institute of Dental and Craniofacial Research (NIDCR)|