Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Change in size of lesion
研究概览
简要总结
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient 2 years of age and older
- •Venous, lymphatic, or venolymphatic malformations
排除标准
- •Children with contraindication to use of sirolimus
- •Children with history of transplant
- •Children with a history of natural immunodeficiency
- •Children with a history of artificially induced immunodeficiency
- •Children with a history of a serious or life-threatening infection
- •Children taking CYP3A4 inhibiting medications
- •Children taking strong CYP3A4 inducers to avoid subtherapeutic dosing/exposure.
- •Inability or unwillingness of subject or legal guardian/representative to give informed consent
- •Women that are or may become pregnant o Sirolimus is a Pregnancy Category C drug. No randomized controlled studies have been done on pregnant women. Women of childbearing potential must be on effective contraception prior to, during, and for 12 weeks following sirolimus therapy.
研究组 & 干预措施
Treatment Group
干预措施: Sirolimus (Drug)
结局指标
主要结局
Change in size of lesion
时间窗: Baseline and 6 months
Will be measuring the size of the lesions (mm) at each patient visit
Change in size of lesion through photograph
时间窗: Baseline and 6 months
Will be evaluating clinical photographs of lesions at each patient visit
次要结局
- Number of participants with laboratory abnormalities(From baseline visit to 2 month visit)
- Change in quality of life as assessed by questionnaire(Baseline and 6 months)
- Number of side effects experienced(Month Six)
- Number of side effects experienced(Month One)
- Number of side effects experienced(Month Two)
- Number of side effects experienced(Month Three)
- Number of side effects experienced(Month Four)
- Number of side effects experienced(Month Five)
研究者
Chelsea Shope
Principal Investigator
Medical University of South Carolina