A Randomized Double-Blind Pilot Study of Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-Versus-Host-Disease
- Chronic Graft versus Host Disease (cGVHD) is a major late complication of allogeneic
hematopoietic stem cell transplantation.
- The oral cavity is the second most commonly affected area in cGVHD and is a major cause
- Clobetasol is a high-potency topical corticosteroid widely used for a variety of
inflammatory disorders of the skin and oral mucosa.
- Treatment of oral cGVHD by topical agents is an attractive strategy to potentially
avoid adverse effects associated with systemic immunosuppression.
- To investigate efficacy of topical clobetasol 0.05% oral rinse for oral chronic
graftversus-host disease (cGVHD)
- To assess the effect of topical clobetasol on oral cGVHD related pain, sensitivity, and
- To evaluate the basic pharmacodynamics and pharmacokinetics of clobetasol mouth rinse
in cGVHD patients.
- To obtain a pilot assessment of patient perceived oral health in cGHVD and its response
to treatment with topical clobetasol.
- To evaluate the psychometrics of the NIH Oral cGVHD Clinical Scoring instrument.
- To evaluate the immunological profile present at baseline and after clobetasol
treatment in oral tissue to identify potential biomarkers for disease response or
resistance to clobetasol.
- To evaluate blood and saliva to identify potential biomarkers for oral cGVHD disease
activity and response to topical clobetasol.
- Patients age 12-99 years with clinically significant oral cGVHD.
- This is a randomized, double blind, placebo-controlled, pilot study of clobetasol 0.05%
topical oral rinse with an open label extension period.
- Patients will rinse oral cavity with 10cc of clobetasol 0.05% or placebo oral rinse for
2 minutes 3 times a day.
- Treatment duration will be for 2 weeks in the randomized phase and 2-4 weeks in the
open label phase.
- A maximum of 34 evaluable patients will be enrolled on this pilot trial.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment
To investigate efficacy of topical clobetasol 0.05% oral rinse for oral chronic graft-versus-host-disease (cGVHD) during a four-week treatment period as assessed by Oral Mucositis Rating Scale (OMRS).
Steven Z Pavletic, M.D.
National Cancer Institute (NCI)
United States: Federal Government
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Bethesda, Maryland 20892|