RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 1
- 主要终点
- Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
研究概览
简要总结
The purpose of this research study is to find out what effects RAD001 has on angiomyolipomas of a person with Tuberous Sclerosis Complex and to determine the safe dose of RAD001 without toxicity.
The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.
详细描述
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.
Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM.
The primary goal of this study is to evaluate the clinical effectiveness of RAD001 in a 24 month trial. Although the primary goal is to determine if the drug RAD001 has effects on angiomyolipomas, other diseases associated with tuberous sclerosis will be monitored too, specifically any change in involvement of your brain or lungs with tuberous sclerosis. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or lymphangioleiomyomatosis
- •Angiomyolipoma one centimeter or greater in largest diameter
- •Between the ages of 18 and 65 years old.
- •If female, documentation of negative pregnancy test prior to enrollment and, where applicable, use of appropriate non-estrogen containing birth control contraceptive regimen while on study.
- •Adequate renal function (creatinine < 3 mg/dl)
排除标准
- •Pregnant or lactating women
- •Continuous requirement for supplemental oxygen
- •Surgery within past 2 months
- •Use of an investigational drug within last 30 days.
研究组 & 干预措施
Administration of RAD001
干预措施: RAD001 (Drug)
结局指标
主要结局
Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline
时间窗: 12 months
次要结局
未报告次要终点