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Protocol Details

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

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 12
Max Age: 110

Referral Letter Required


Population Exclusion(s)

Pregnant Women


Oral Chronic GVHD;
Topical Dexamethasone;
Tissue Markers;
Salivary Proteomics;
Quality of Life

Recruitment Keyword(s)

Oral Chronic Graft Versus Host Disease;
Oral Pain;
Mouth Pain


Graft vs Host Disease

Investigational Drug(s)


Investigational Device(s)



Drug: Topical Dexamethasone
Other: Placebo

Supporting Site

National Heart, Lung, and Blood Institute

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 "cosyntropin," 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.

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1. History of allogeneic hematopoietic stem cell transplantation within 60-90 days of enrollment.

2. Age 12 or older.

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

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


1. Clinically significant oral chronic GVHD at the time of the screening.

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

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

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

5. Documented hypersensitivity to dexamethasone.

6. Pregnancy or lactation.

7. Inability to understand the investigational nature of the study.

8. Inability to provide informed consent.

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

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.

Thongprasom K, Luengvisut P, Wongwatanakij A, Boonjatturus C. Clinical evaluation in treatment of oral lichen planus with topical fluocinolone acetonide: a 2-year follow-up. J Oral Pathol Med. 2003 Jul;32(6):315-22. doi: 10.1034/j.1600-0714.2003.00130.x. PMID: 12787037.

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Principal Investigator

Referral Contact

For more information:

Joseph A. Clara, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CRC BG RM 5-5264
(301) 312-3731

Sara E. Hauffe
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 6512
10 Center Drive
Bethesda, Maryland 20892
(301) 385-2828

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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