Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot Trial
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Evolution of angiomyolipomas volume
研究概览
简要总结
Treatment of angiomyolipomas is based on invasive techniques such as surgery or embolization. Development of anti-angiogenic therapies is a major and growing field of research in hypervascularized tumors. Angiomyolipomas have been shown to regress after prolonged treatment with mTOR inhibitors (Sirolimus), but with a large proportion of secondary effects. We showed recently that beta-blockers were able to induce regression of infantile hemagiomas. Consequently, we looked for and found, histologically, in a few cases of angiomyolipomas the presence of beta2 receptors.
The aim of the study is to estimate if beta-blockers could induce regression or stabilization of renal angiomyolipomas in tuberous sclerosis in a pilot study.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Tuberous sclerosis patients with one or several angiomyolipomas of a size of at least 4 cms.
排除标准
- •Patients whom CT or MR scan shows one or several intra-lesional aneurisms requiring a preventive embolization.
- •Patients with a retroperitoneal hemorragic complication requiring a preventive embolization.
- •Patients whom biopsy will show an adenocarcinoma, hypertension non controlled, renal failure and severe liver.
- •Diabetic subjects insufficiently controlled.
- •Beta-blockers contra-indication.
- •Psychosis, severe mental disorder.
- •Patient already treated with beta-blockers or mTOR inhibitors.
- •Pregnant or nursing women.
研究组 & 干预措施
Patient
干预措施: Propranolol (Drug)
结局指标
主要结局
Evolution of angiomyolipomas volume
时间窗: 6 months and 1 year after inclusion
Stabilization or even regression of angiomyolipomas volume after 6 months and 1 year of treatment with a quantification of the vascular component.
次要结局
- Renal function evolution(6 months and 1 year after inclusion)
- Effect on the potential haemorraghic transformation(6 months and 1 year after inclusion)
- Improvement of the quality of life(6 months and 1 year after inclusion.)
- Effect on face angiofibromas(6 months and 1 year after inclusion)