Ignite Creation Date:
2024-05-05 @ 5:08 PM
Last Modification Date:
2024-10-26 @ 9:28 AM
Study NCT ID:
NCT00391170
Status:
COMPLETED
Last Update Posted:
2022-02-10
First Post:
2006-10-20
Brief Title:
Dexamethasone to Prevent Oral Chronic Graft-versus-Host Disease
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pilot Study of Topical Dexamethasone 001 Solution for Prevention of Oral Chronic Graft Versus Host Disease
Status:
COMPLETED
Status Verified Date:
2021-02-24
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will determine if a dexamethasone mouth rinse can reduce the risk of developing oral chronic graft-versus-host disease cGVHD in patients who have undergone a bone marrow stem cell transplant procedure cGVHD is a common complication of stem cell transplantation resulting from the donor cells attacking the transplant recipients tissues In oral cGVHD the tissues in the mouth are damaged causing painful mouth sores Dexamethasone is a corticosteroid that is commonly used to treat inflammation It is the only corticosteroid available that can be used as a mouth rinse
Patients 12 years of age or older who have received a stem cell transplant may be eligible to participate if they are enrolled within 70 to 90 days of their transplant Candidates are screened with a medical history and oral exam
Participants are randomly assigned to receive either the dexamethasone rinse or a placebo a solution that looks and tastes like the dexamethasone rinse but has no active medication They undergo the following procedures
Treatment with the study solution Patients rinse their mouth with the dexamethasone solution or placebo three times a day for 3 months
Clinic visits before starting treatment and at 1 2 and 3 months after starting the study drug for the following procedures
Oral exam before starting treatment and at each visit
Photographs of the mouth before starting treatment and at 3 months
Biopsy from inside the cheek before starting treatment The inside of the cheek is numbed and a small piece of tissue is removed for examination by a pathologist
Saliva sample collection before starting treatment
Blood draw before starting treatment and at each visit
Quality-of-life questionnaires before starting treatment and at 3 months
Questionnaire to assess level of dry mouth and mouth pain before starting treatment and at each visit
Review of medications at each visit
ACTH stimulation test to evaluate adrenal gland function at 3 months Patients are given an injection of a drug called ACTH or cosyntropin which is a version of a hormone normally produced by the pituitary gland Blood samples are drawn before the injection and at 30 and 60 minutes after the injection to measure levels of the hormone cortisol
After treatment ends participants are contacted by telephone every month for 6 months to report any symptoms of cGVHD and they return to the clinic at 6 months for a final evaluation
Patients 12 years of age or older who have received a stem cell transplant may be eligible to participate if they are enrolled within 70 to 90 days of their transplant Candidates are screened with a medical history and oral exam
Participants are randomly assigned to receive either the dexamethasone rinse or a placebo a solution that looks and tastes like the dexamethasone rinse but has no active medication They undergo the following procedures
Treatment with the study solution Patients rinse their mouth with the dexamethasone solution or placebo three times a day for 3 months
Clinic visits before starting treatment and at 1 2 and 3 months after starting the study drug for the following procedures
Oral exam before starting treatment and at each visit
Photographs of the mouth before starting treatment and at 3 months
Biopsy from inside the cheek before starting treatment The inside of the cheek is numbed and a small piece of tissue is removed for examination by a pathologist
Saliva sample collection before starting treatment
Blood draw before starting treatment and at each visit
Quality-of-life questionnaires before starting treatment and at 3 months
Questionnaire to assess level of dry mouth and mouth pain before starting treatment and at each visit
Review of medications at each visit
ACTH stimulation test to evaluate adrenal gland function at 3 months Patients are given an injection of a drug called ACTH or cosyntropin which is a version of a hormone normally produced by the pituitary gland Blood samples are drawn before the injection and at 30 and 60 minutes after the injection to measure levels of the hormone cortisol
After treatment ends participants are contacted by telephone every month for 6 months to report any symptoms of cGVHD and they return to the clinic at 6 months for a final evaluation
Detailed Description:
Prevention of oral chronic graft versus host disease GVHD by topical agents is an attractive strategy because it would potentially avoid the adverse effects associated with systemic immunosuppression Topically administered dexamethasone solution is a commonly used agent for the prophylaxis of oral inflammatory conditions including GVHD However the efficacy and systemic effects of topically administered dexamethasone solution are unknown We therefore propose this trial designed to evaluate the efficacy and safety of topical dexamethasone solution for prevention of oral chronic GVHD in stem cell transplant recipients
This pilot phase II study will follow a randomized double-blind placebo controlled parallel group design Consenting subjects who have undergone hematopoietic stem cell transplantation at the NIH Clinical Center and the surrounding transplant clinics will be randomized 5050 to receive dexamethasone 001 solution or placebo as an oral rinse for 3 months starting 90-100 days post-transplant Subjects will be evaluated monthly after the start of intervention Diagnostic and research evaluations will include a complete oral examination oral mucosal biopsy prior to the beginning of the intervention day -7 and at the time of development of oral chronic GVHD or at the completion of intervention in the absence of clinical GVHD We will measure serum dexamethasone levels and perform short cosyntropin ACTH stimulation test at the end of the 3 months of intervention or onset of clinically significant GVHD
The primary objective of the study is to evaluate the safety and efficacy of topical dexamethasone 001 solution used as an oral rinse for prevention of oral chronic GVHD Our primary endpoint will be the proportion of subjects that develop clinically significant severity score 3 or higher oral chronic GVHD after three months
Secondary objectives will include the impact of oral chronic GVHD on the quality of life characterization of the changes in tissue and salivary biomarkers associated with development of oral graft versus host disease and measures of the effects of topical dexamethasone on hypothalamo-pituitary-adrenal axis Secondary outcomes will include oral cavity specific quality of life as measured by Oral Health Impact Profile OHIP-14 questionnaire oral discomfort levels improvement in general quality of life scores and severity of oral chronic GVHD as measured by the site-specific GVHD scoring system
This pilot phase II study will follow a randomized double-blind placebo controlled parallel group design Consenting subjects who have undergone hematopoietic stem cell transplantation at the NIH Clinical Center and the surrounding transplant clinics will be randomized 5050 to receive dexamethasone 001 solution or placebo as an oral rinse for 3 months starting 90-100 days post-transplant Subjects will be evaluated monthly after the start of intervention Diagnostic and research evaluations will include a complete oral examination oral mucosal biopsy prior to the beginning of the intervention day -7 and at the time of development of oral chronic GVHD or at the completion of intervention in the absence of clinical GVHD We will measure serum dexamethasone levels and perform short cosyntropin ACTH stimulation test at the end of the 3 months of intervention or onset of clinically significant GVHD
The primary objective of the study is to evaluate the safety and efficacy of topical dexamethasone 001 solution used as an oral rinse for prevention of oral chronic GVHD Our primary endpoint will be the proportion of subjects that develop clinically significant severity score 3 or higher oral chronic GVHD after three months
Secondary objectives will include the impact of oral chronic GVHD on the quality of life characterization of the changes in tissue and salivary biomarkers associated with development of oral graft versus host disease and measures of the effects of topical dexamethasone on hypothalamo-pituitary-adrenal axis Secondary outcomes will include oral cavity specific quality of life as measured by Oral Health Impact Profile OHIP-14 questionnaire oral discomfort levels improvement in general quality of life scores and severity of oral chronic GVHD as measured by the site-specific GVHD scoring system
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 07-H-0005 | None | None | None |