Protocol Number: 04-NR-0069

Title:

Evaluation of Efficacy and Mechanisms of Topical Thalidomide for Chronic Graft-Versus-Host-Disease Related Stomatitis

Number:

04-NR-0069

Summary:

This study will be conducted in two parts. The first part is a pilot study testing the effects of a thalidomide ointment in patients who have developed oral chronic graft-versus-host-disease (GVHD)-related ulcerative stomatitis following allogeneic bone marrow transplantation (ABMT). Stomatitis is an inflammation of the lining of the throat and mouth that may lead to ulcers and pain in the mouth and throat. GVHD - a condition in which the donor cells see patient's cells as foreign and mount an immune response to them - may be related to increased levels of a substance called TNF-alpha following ABMT. Thalidomide's anti-inflammatory effects may lower TNF-alpha levels and decrease chronic GVHD-related stomatitis and oral pain in these patients.

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.)

Sponsoring Institute:

(NINR)

Recruitment Detail

Type: Active Accrual Of New Subjects

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA:

Allogeneic transplant patients who are enrolled in the participating NHLBI ABMT protocols, and are willing to participate in this study concurrently;

Have a diagnosis of oral cGVHD as confirmed by surgical biopsy results;

Able to understand and sign protocol consent;

Ages 18 to 80 years.

EXCLUSION CRITERIA:

Pregnant or lactating females for main study;

Unwilling to follow precautions for use of thalidomide;

Unable to demonstrate appropriate use of study medication;

Concurrent use of non-protocol-related medications confounding assessment of the inflammatory response (antihistamines, non-steroidal anti-inflammatory drugs, antidepressants);

Allergic reaction to thalidomide;

Pre-existing oral infection, which might maximize the possibility of an infection or sepsis contributing to a drug-related adverse event;

Unwilling to forego concurrent treatment for mucosal lesions and/or related oral pain (including topical steroids, viscous lidocaine, topical anti0fungals, or mouthwashes);

Requiring addition of new systemic therapy including thalidomide, steroids, or radiation therapy;

Use of sedatives (including CNS depressants).

Special Instructions: Currently Not Provided

Keywords:

Stomatitis

Oropharyngeal Pain

TNF-Alpha

Allogeneic BMT

Inflammation

Recruitment Keywords:

None

Conditions:

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citations: Not Provided

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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