Protocol Number: 07-H-0005

Title:

Pilot Study of Topical Dexamethasone 0.01% Solution for Prevention of Oral Chronic Graft Versus Host Disease

Number:

07-H-0005

Summary:

This study will determine if a dexamethasone mouth rinse can reduce the risk of developing oral chronic graft-versus-host disease (cGVHD) in patients who have undergone a bone marrow (stem cell) transplant procedure. cGVHD is a common complication of stem cell transplantation, resulting from the donor cells attacking the transplant recipient's tissues. In oral cGVHD, the tissues in the mouth are damaged, causing painful mouth sores. Dexamethasone is a corticosteroid that is commonly used to treat inflammation. It is the only corticosteroid available that can be used as a mouth rinse.

Patients 12 years of age or older who have received a stem cell transplant may be eligible to participate if they are enrolled within 70 to 90 days of their transplant. Candidates are screened with a medical history and oral exam.

Participants are randomly assigned to receive either the dexamethasone rinse or a placebo (a solution that looks and tastes like the dexamethasone rinse but has no active medication). They undergo the following procedures:

Treatment with the study solution. Patients rinse their mouth with the dexamethasone solution or placebo three times a day for 3 months.

Clinic visits before starting treatment and at 1, 2 and 3 months after starting the study drug for the following procedures:

-Oral exam (before starting treatment and at each visit).

-Photographs of the mouth (before starting treatment and at 3 months).

-Biopsy from inside the cheek (before starting treatment). The inside of the cheek is numbed and a small piece of tissue is removed for examination by a pathologist.

-Saliva sample collection (before starting treatment).

-Blood draw (before starting treatment and at each visit).

-Quality-of-life questionnaires (before starting treatment and at 3 months).

-Questionnaire to assess level of dry mouth and mouth pain (before starting treatment and at each visit).

-Review of medications (at each visit).

-ACTH stimulation test to evaluate adrenal gland function (at 3 months). Patients are given an injection of a drug called "ACTH" or "cosynthropin," which is a version of a hormone normally produced by the pituitary gland. Blood samples are drawn before the injection and at 30 and 60 minutes after the injection to measure levels of the hormone cortisol.

After treatment ends, participants are contacted by telephone every month for 6 months to report any symptoms of cGVHD, and they return to the clinic at 6 months for a final evaluation.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: Yes

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA:

History of allogeneic hematopoietic stem cell transplantation at the NIH Clinical Center within 70-90 days of enrollment.

Age 12 or older.

Ability to rinse and expectorate study medication rather than swallow it.

Ability and willingness to come to Clinical Center for monthly follow-up appointments and at the time of development of symptoms/signs suggestive of oral GVHD.

EXCLUSION CRITERIA:

Clinically significant oral GVHD at the time of the screening.

Active viral or fungal infection involving oral cavity not resolving by day 90.

Platelet count less than 20,000/ml at the time of the screening appointment.

Life expectancy less than 4 months at the time of enrollment.

Documented hypersensitivity to dexamethasone.

Pregnancy or lactation.

Inability to understand the investigational nature of the study ability to provide informed consent.

Special Instructions:

Currently Not Provided

Keywords:

Mucositis

Topical Steroids

Transplant Complication

Oral Pain

Recruitment Keyword(s):

Oral Chronic Graft Versus Host Disease

Oral Pain

Mouth Pain

Condition(s):

Graft vs Host Disease

Investigational Drug(s):

None

Investigational Device(s):

None

Intervention(s):

Drug: Dexamethasone 0.01% Solution

Supporting Site:

National Heart, Lung and Blood Institute

Contact(s):

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

Citation(s):

Chakrabarti S, Childs R. Allogeneic immune replacement as cancer immunotherapy. Expert Opin Biol Ther. 2003 Oct;3(7):1051-60. Review.

Tykodi SS, Warren EH, Thompson JA, Riddell SR, Childs RW, Otterud BE, Leppert MF, Storb R, Sandmaier BM. Allogeneic hematopoietic cell transplantation for metastatic renal cell carcinoma after nonmyeloablative conditioning: toxicity, clinical response, and immunological response to minor histocompatibility antigens. Clin Cancer Res. 2004 Dec 1;10(23):7799-811.

Lee SJ, Vogelsang G, Flowers ME. Chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2003 Apr;9(4):215-33. Review.

• 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 Clinical Research Informatics, CC.

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