Clobetasol for Oral Graft-Versus-Host Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2013 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )
ClinicalTrials.gov Identifier:
NCT01557517
First received: March 16, 2012
Last updated: June 5, 2014
Last verified: October 2013
  Purpose

Background:

- Oral graft-versus-host disease (GVHD) is a possible complication of bone marrow transplants. It is the result of the donor cells trying to attack the recipient s body. Symptoms include dry mouth, sensitivity and pain when tasting certain spices and flavors, and painful swallowing. Steroids are a possible effective treatment for GHVD, but they can cause side effects when given as pills or injections. Steroids given in a cream or rinse form, applied directly to the site of the symptoms, can have fewer side effects. However, their effectiveness as a rinse has not been tested in the mouth. Researchers want to see if a steroid called clobetasol can be used as a mouth rinse to treat oral GHVD.

Objectives:

- To see if a clobetasol rinse is a safe and effective treatment for oral graft-versus-host disease.

Eligibility:

- Individuals at least 12 years of age who have oral GHVD and are not allergic to clobetasol.

Design:

  • Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. They will also have an oral exam, a mouth tissue biopsy, and other tests before starting the study drug.
  • Participants will be separated into two groups. One group will receive clobetasol; the other will have a placebo liquid.
  • Participants will rinse their mouths with the study liquid three times a day after meals for 2 weeks.
  • After 2 weeks, participants will have another study visit with blood tests and other exams.
  • After the study visit, all participants will start to use the clobetasol rinse. Those who originally had clobetasol will use the rinse for another 2 weeks. Those who originally had a placebo will use the rinse for 4 weeks.
  • Participants will have a follow-up exam after the end of treatment.

Condition Intervention Phase
Oral Chronic Graft vs Host Disease
Drug: Clobetasol Oral Rinse
Procedure: Chronic GVHD organ staging
Procedure: Assessment of Oral GVHD
Behavioral: Numeric rating scales for oral pain
Behavioral: Oral Global Scale
Behavioral: Painometer (POM) Assessment
Behavioral: Oral Health Impact Profile-OHIP-14
Procedure: Biopsy of buccal mucosa
Procedure: Short ACTH stimulation test
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: A Randomized Double-Blind Pilot Study of Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-Versus-Host-Disease

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • 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).

Secondary Outcome Measures:
  • 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.

Estimated Enrollment: 34
Study Start Date: January 2012
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Clobetasol Oral Rinse
    N/A
    Procedure: Chronic GVHD organ staging
    N/A
    Procedure: Assessment of Oral GVHD
    N/A
    Behavioral: Numeric rating scales for oral pain
    N/A
    Behavioral: Oral Global Scale
    N/A
    Behavioral: Painometer (POM) Assessment
    N/A
    Behavioral: Oral Health Impact Profile-OHIP-14
    N/A
    Procedure: Biopsy of buccal mucosa
    N/A
    Procedure: Short ACTH stimulation test
    N/A
Detailed Description:

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.
  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01557517

Contacts
Contact: Licia Masuch (301) 435-9352 licia.masuch@nih.gov
Contact: Steven Z Pavletic, M.D. (301) 402-4899 sp326h@nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office    (888) NCI-1937      
Sponsors and Collaborators
Investigators
Principal Investigator: Steven Z Pavletic, M.D. National Cancer Institute (NCI)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )
ClinicalTrials.gov Identifier: NCT01557517     History of Changes
Other Study ID Numbers: 120068, 12-C-0068
Study First Received: March 16, 2012
Last Updated: June 5, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
cGVHD
Oral cGVHD
Topical
Oral Rinse
Clobetasol
Oral Graft-Versus-Host Disease
GVHD

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases
Clobetasol
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 22, 2014