A Randomized Double-Blind Pilot Study of Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-Versus-Host-Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period
研究概览
简要总结
Background:
- Oral graft-versus-host disease (GVHD) is a possible complication of bone marrow transplants. It is the result of the donor cells trying to attack the recipients body. Symptoms include dry mouth, sensitivity and pain when tasting certain spices and flavors, and painful swallowing. Steroids are a possible effective treatment for GHVD, but they can cause side effects when given as pills or injections. Steroids given in a cream or rinse form, applied directly to the site of the symptoms, can have fewer side effects. However, their effectiveness as a rinse has not been tested in the mouth. Researchers want to see if a steroid called clobetasol can be used as a mouth rinse to treat oral GHVD.
Objectives:
- To see if a clobetasol rinse is a safe and effective treatment for oral graft-versus-host disease.
Eligibility:
- Individuals at least 12 years of age who have oral GHVD and are not allergic to clobetasol.
Design:
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. They will also have an oral exam, a mouth tissue biopsy, and other tests before starting the study drug.
- Participants will be separated into two groups. One group will receive clobetasol; the other will have a placebo liquid.
- Participants will rinse their mouths with the study liquid three times a day after meals for 2 weeks.
- After 2 weeks, participants will have another study visit with blood tests and other exams.
- After the study visit, all participants will start to use the clobetasol rinse. Those who originally had clobetasol will use the rinse for another 2 weeks. Those who originally had a placebo will use the rinse for 4 weeks.
- Participants will have a follow-up exam after the end of treatment....
详细描述
BACKGROUND:
- Chronic Graft versus Host Disease (cGVHD) is a major late complication of allogeneic hematopoietic stem cell transplantation.
- The oral cavity is the second most commonly affected area in cGVHD and is a major cause of morbidity.
- Clobetasol is a high-potency topical corticosteroid widely used for a variety of inflammatory disorders of the skin and oral mucosa.
- Treatment of oral cGVHD by topical agents is an attractive strategy to potentially avoid adverse effects associated with systemic immunosuppression.
OBJECTIVES:
- To investigate efficacy of topical clobetasol 0.05% oral rinse for oral chronic graft versus-host disease (cGVHD)
ELIGIBILITY:
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Clobetasol
Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day.
干预措施: Clobetasol Oral Rinse (Drug)
Placebo
Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.
干预措施: Placebo oral rinse (Drug)
结局指标
主要结局
Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period
时间窗: At 4 weeks on active treatment
Mucosal changes were assessed by the Oral Mucositis Rating Scale. The primary endpoint was evaluated using the OMRS. Oral tissue changes are rated on a scale of 0-3 compared with normal oral tissue (0-normal/no change, 1-mild change, 2-moderate change, and 3-severe change). Total score is the sum of all OMRS items with a possible range of 0. Total score is the sum of all OMRS items with a possible range of 0-273. Lower score=more normal oral mucosa. There is no standard definition of response in this field. Definitions we used in this pilot study to grade the response to study intervention are: Progress of 25% of initial score (rounded to the closest number) on the OMRS scale. Completion (PD) is defined as an increase of 25% of initial score (rounded to the closest number). Partial Response (PR) is defined as a decrease Response (CR) is defined as a PR plus a score of 0 on the erythema and ulceration components. Stable Disease (SD) does not meet criteria for progression or response.
次要结局
- Percent Change in Participants' Raw Score of Oral cGVHD Related Sensitivity on a 0-10 Rating Scale(Baseline and 4 weeks on active treatment)
- Plasma Concentrations of Clobetasol Mouth Rinse in cGVHD Patients at Baseline (Day 0) and Day 28(Baseline Day 0 and Day 28)
- Maximum Plasma Concentration (Cmax) of Clobetasol During Pharmacokinetic Testing(pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse)
- Time to Maximum Plasma Concentration (Cmax) of Clobetasol(pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse)
- Percent Change in Participants' Raw Score of Oral cGVHD Related Pain on a 0-10 Rating Scale(Baseline to Day 14 and baseline to Day 28)
- Percent Change in Participants' Raw Score of Oral cGVHD Related Dryness on a 0-10 Rating Scale(Baseline and 4 weeks on active treatment)
- Area Under the Plasma Concentration vs Time Curve for All Time Points(pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse)
- Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)(Date treatment consent signed to date off study, approximately 62 months and 12 days.)
研究者
Najla El Jurdi
Principal Investigator
National Cancer Institute (NCI)