Protocol Number: 04-NR-0069
If this first part of the study is successful, then the second part of the study will be conducted. The second part of this study will test the effects of a thalidomide mouthwash in treating stomatitis in patients who have developed oral chronic graft-versus-host-disease (GVHD)-related stomatitis following allogeneic bone marrow transplantation (ABMT). Applying thalidomide directly to the GVHD-related mouth ulcer in ointment form or to the entire oral cavity by mouthwash form rather than taking it as a pill may reduce the amount of drug that enters the blood stream and cause fewer side effects.
Patients between 18 and 80 years of age who have received an ABMT and developed oral chronic GVHD-related stomatitis as confirmed by surgical biopsy may be eligible for this study. The only eligible female participants for the pilot study will be women who are unable to have children. In the pilot study, participants will be randomly assigned to receive thalidomide ointment or placebo (an ointment with no thalidomide) to use four times a day. In the mouthwash study, participants will be randomly assigned to receive thalidomide mouthwash or placebo (a mouth rinse with no thalidomide) to use four times a day. Participants will also undergo the following procedures before beginning medication, 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 ratings.
- Dental examination.
- Quality of life questionnaire (The questionnaire is repeated only at week 8 of the study.).
- Mouth photography to measure and record the response to treatment.
- Saliva sampling to look for chemicals, including TNF-alpha.
- Ulcer exudate collected by filter paper to obtain fluid for measuring TNF-alpha levels.
- Gentle swabbing of 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 TNF-alpha (The punch biopsy is repeated only at week 4 of the study.)
- Blood sampling to monitor thalidomide and TNF-alpha levels.
- A urine pregnancy test for women who are able to have children. (The pregnancy test is repeated at weeks 2, 4, and 8.)
National Institutes of Health Clinical Center
Bethesda, Maryland 20892. Last update: 01/30/2009