Effectiveness of Topical Thalidomide to Treat Chronic Graft-Versus-Host-Disease Related Stomatitis
Status:
Terminated
Trial end date:
2010-04-01
Target enrollment:
Participant gender:
Summary
This study was designed to be conducted in 2 parts. The first part is a pilot study to test
the effects of topical thalidomide gel 20mg applied to up to 3 oral ulcers in patients who
have developed oral chronic graft-versus-host-disease (cGVHD)-related ulcerative stomatitis
following allogeneic bone marrow/peripheral blood stem cell transplant (HSCT). Chronic GVHD
may be related to increased levels of a cytokine called TNF-alpha (TNFa) following HSCT.
Thalidomide's anti-inflammatory effects may lower TNFa levels, lead to healing of these oral
ulcers, and decrease oral pain.
If the pilot study is successful, the second part of the study will be done. This will test
the effects of a 0.1% (20mg) thalidomide mouthwash in treating oral cGVHD-related stomatitis
in patients following allogeneic HSCT. Applying thalidomide directly to the GVHD-related
mouth ulcer in gel form or to the entire oral cavity in mouthwash form rather than taking it
in pill form may reduce the amount of drug that enters the blood stream and cause less side
effects.
In the pilot study, participants will be randomly assigned to receive thalidomide gel 20mg or
placebo (identical gel with no thalidomide) to use 4 times a day for 4 weeks. In the
mouthwash study, participants will be randomly assigned to receive 0.1% 20mg thalidomide
mouthwash or placebo (identical mouth rinse with no thalidomide) to use 4 times a day for 4
weeks. Participants will undergo the following procedures before beginning experimental
treatment, then once a week for 4 weeks, and then approximately 8 weeks after the first
visit:
- Interview about current medications and use of alcohol and cigarettes
- Self-report of mouth and throat pain
- Oral examination for stomatitis rating, and oral ulcer(s) measurement
- Quality of life questionnaire (repeated only at week 8 of the study)
- Mouth photography to measure and record the oral ulcer response to treatment
- Saliva sampling to look for proinflammatory cytokines (small proteins), including TNFa
- Oral ulcer exudate collected by filter paper to obtain fluid for measuring TNFa levels
- Gentle swabbing of oral ulcers to culture for virus, fungus, and bacteria that may be
present
- Small punch biopsy of the area near the ulcer or affected area to check for presence of
TNFa (repeated only at week 4 of the study)
- Blood sampling to monitor TNFa levels
- A urine pregnancy test for women who are able to have children (repeated at weeks 2, 4,
and 8)