Effectiveness of Topical Thalidomide to Treat Chronic Graft-Versus-Host-Disease Related Stomatitis
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ClinicalTrials.gov Identifier: NCT00075023 |
Recruitment Status
:
Terminated
(unable to enroll adequate subjects)
First Posted
: December 31, 2003
Results First Posted
: November 4, 2015
Last Update Posted
: November 4, 2015
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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)
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Graft-versus-Host Disease Stomatitis | Drug: Thalidomide Gel Drug: Placebo Gel | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 10 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | Evaluation of Efficacy and Mechanisms of Topical Thalidomide for Chronic Graft-Versus-Host-Disease Related Stomatitis |
Study Start Date : | December 2003 |
Actual Primary Completion Date : | April 2010 |
Actual Study Completion Date : | April 2010 |

Arm | Intervention/treatment |
---|---|
Experimental: Thalidomide gel
Thalidomide gel 20 mg applied topically 4 times daily for 4 weeks to a maximum of 3 target oral ulcers
|
Drug: Thalidomide Gel |
Experimental: Placebo
Placebo gel with no Thalidomide applied topically 4 times daily for 4 weeks to a maximum of 3 target oral ulcers
|
Drug: Placebo Gel |
- Mean Percentage Change in Total Surface Area of Oral Ulceration. [ Time Frame: baseline to 4 weeks ]Mean percentage change in total surface area of oral ulceration

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Ages Eligible for Study: | 18 Years to 80 Years (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- INCLUSION CRITERIA:
Participating in HSCT or cGVHD protocols and willing to participate in this study concurrently;
Diagnosed with oral cGVHD stomatitis confirmed by surgical biopsy results;
Oral ulceration present
Able to understand and sign protocol informed consent;
Ages 18 to 80 years of age.
EXCLUSION CRITERIA:
Pregnant or lactating females;
For the proof of concept study, females who are not surgically sterilized by means of hysterectomy or tubal ligation;
For the main study, females of childbearing potential who do not agree to the use of two forms of highly effective contraception for at least four weeks prior, during, and for four weeks following the last dose of study drug;
Sexually active males who do not agree to the use of a latex condom while receiving study drug and for four weeks following the last dose of study drug;
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);
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 or unable to forego concurrent treatment for mucosal lesions and/or related oral pain (including topical steroids, viscous lidocaine, topical anti-fungals);
Requiring addition of new systemic therapy including thalidomide, steroids, or radiation therapy;
Use of sedatives (including CNS depressants);
Absolute neutrophil count (ANC) less than 750/mm(3)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00075023
United States, Maryland | |
National Institutes of Health Clinical Center, 9000 Rockville Pike | |
Bethesda, Maryland, United States, 20892 | |
United States, Washington | |
Fred Hutchinson Cancer Research Center | |
Seattle, Washington, United States, 28104 |
Principal Investigator: | Jane Fall-Dickson, RN, PhD | NIH/NINR |
Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | National Institute of Nursing Research (NINR) |
ClinicalTrials.gov Identifier: | NCT00075023 History of Changes |
Other Study ID Numbers: |
040069 04-NR-0069 |
First Posted: | December 31, 2003 Key Record Dates |
Results First Posted: | November 4, 2015 |
Last Update Posted: | November 4, 2015 |
Last Verified: | October 2015 |
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Nursing Research (NINR) ):
Stomatitis Oropharyngeal pain TNF-alpha Allogeneic hematopoietic stem cell transplant |
Inflammation Thalidomide Oral chronic graft-versus-host disease |
Additional relevant MeSH terms:
Graft vs Host Disease Stomatitis Immune System Diseases Mouth Diseases Stomatognathic Diseases Thalidomide Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |
Leprostatic Agents Anti-Bacterial Agents Anti-Infective Agents Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Growth Inhibitors Antineoplastic Agents |