Dexamethasone to Prevent Oral Chronic Graft-versus-Host Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Orthopedic, Hematology |
| Therapuetic Areas: | Hematology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 12 - 110 |
| Updated: | 4/3/2019 |
| Start Date: | November 24, 2006 |
| End Date: | December 31, 2023 |
| Contact: | Sara E Hauffe |
| Email: | sara.hauffe@nih.gov |
| Phone: | (301) 402-3595 |
Pilot Study of Topical Dexamethasone 0.01% Solution for Prevention of Oral Chronic Graft Versus Host Disease
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 recipient's 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 "cosynthropin," 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.
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 recipient's 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 "cosynthropin," 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.
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 oral 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 50/50 to receive dexamethasone 0.01% 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 0.01% 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 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.
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 oral 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 50/50 to receive dexamethasone 0.01% 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 0.01% 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 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.
- INCLUSION CRITERIA:
1. History of allogeneic hematopoietic stem cell transplantation within 60-90 days
of enrollment.
2. Age 12 or older.
3. Ability to rinse and expectorate study medication rather than swallow it.
4. Ability and willingness to come to Clinical Center for follow-up appointments and
at the time of development of symptoms/signs suggestive of oral GVHD.
EXCLUSION CRITERIA:
1. Clinically significant oral chronic GVHD at the time of the screening.
2. Active viral or fungal infection involving oral cavity not resolving by day 90.
3. Platelet count less than 20,000/ml at the time of the screening appointment.
4. Life expectancy less than 4 months at the time of enrollment.
5. Documented hypersensitivity to dexamethasone.
6. Pregnancy or lactation.
7. Inability to understand the investigational nature of the study.
8. Inability to provide informed consent.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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