A Randomized Double-Blind Pilot Study of Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-Versus-Host-Disease
12 Years
N/A
Both
Oral Chronic Graft vs Host Disease
Trial Information
A Randomized Double-Blind Pilot Study of Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-Versus-Host-Disease
BACKGROUND:
- 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
of morbidity.
- 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.
OBJECTIVES:
- 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
dryness.
- 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.
ELIGIBILITY:
- Patients age 12-99 years with clinically significant oral cGVHD.
DESIGN:
- 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.
Inclusion Criteria
- INCLUSION CRITERIA:
- Age: 12 years - 99 years.
- Diagnosis: clinically significant oral cGVHD after allogeneic HSCT with severity
score of at least 2 on erythema subset and/or at least 1 on ulceration subset and a
composite score greater than or equal to 20 of the Oral Mucositis Rating Scale (OMRS)
scale confirmed by the principal investigator (PI), clinical study chair (CSC), or
lead associate investigator (LAI).
- Hematologic Function: Patients must have a platelet count greater than or equal to
20,000/microL at the time of the initial evaluation.
- Informed Consent: All patients or their legal representative (for patients < 18 years
old) must sign an IRB approved informed consent document (cGVHD natural history
protocol 04-C-0281 or any NCI protocol allowing for screening procedures) prior to
performing studies to determine patient eligibility. After confirmation of patient
eligibility all patients or their legal representative must sign the protocol
specific informed consent. For pediatric patients age appropriate assent will be
obtained in accordance with NIH guidelines.
- Patients must be able to rinse and expectorate study medication rather than swallow
it. Female patients must be willing to practice birth control (including abstinence)
during and for two months after treatment, if of childbearing potential.
- Patients must have the ability and willingness to come to Clinical Center for
bi-weekly follow-up appointments.
- No change in systemic immunosuppressive therapy (type or intensity level) within 2
weeks prior to enrollment.
- A 7-day washout period is required if patients are currently using another oral
topical treatment for mouth lesions. Patients currently using clobetasol oral topical
treatment are not eligible for this study.
EXCLUSION CRITERIA:
- Documented hypersensitivity to clobetasol.
- Use of clobetasol ointment intra-orally at any time during the last 6-month period.
- Pregnant or breast-feeding females due to possible toxicity to the fetus or infant.
- Inability to understand the investigational nature of the study to provide informed
consent.
- Patients who, for medical or other reasons, are unable to comply with the study
procedures.
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment
Outcome Measure:
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).
Principal Investigator
Steven Z Pavletic, M.D.
Investigator Role:
Principal Investigator
Investigator Affiliation:
National Cancer Institute (NCI)
Authority:
United States: Federal Government
Study ID:
120068
NCT ID:
NCT01557517
Start Date:
January 2012
Completion Date:
January 2017
Related Keywords:
- Oral Chronic Graft vs Host Disease
- cGVHD
- Oral cGVHD
- Topical
- Oral Rinse
- Clobetasol
- Oral Graft-Versus-Host Disease
- GVHD
- Graft vs Host Disease
Name | Location |
|---|---|
| National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda, Maryland 20892 |
