Efficacy and safety of oral sirolimus in vascular malformation: A prospective pilot assessor-blinded interventional study.
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- to assess the percentage reduction in volume of vascular malformation at the end of 1 year of oral sirolimus
研究概览
简要总结
Management of vascular malformation is crucial to prevent the compression of vital organs, ulceration, and limb length discrepancies. Various complex and interlinking molecular pathways are involved in the pathogenesis of vascular malformation, thereby targeted therapy plays a pivotal role in the treatment. Sirolimus also known as rapamycin is a mTOR inhibitor, blocks the RAS-RAF-MEK and PIK3CA-AKT-mTOR pathways. Therefore, resulting in apoptosis and inhibition of vascular pathways.
研究设计
- 研究类型
- Interventional
- 分配方式
- Other
- 盲法
- Outcome Assessor Blinded
入排标准
- 年龄范围
- 1.00 Day(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Complex microcystic and macrocystic lymphatic malformations, venolymphatic or mixed malformations and or capillary malformations, vascular malformations associated overgrowth syndrome.
排除标准
- •Concurrent use of drug with cytochrome P450 3A4 inducer such as barbiturates, glucocorticoids, rifampicin, clarithromycin, and erythromycin.
- •Immunosuppressed patient or patients known case of human immunodeficiency virus infection, ateriovenous and capillary malformations.
结局指标
主要结局
to assess the percentage reduction in volume of vascular malformation at the end of 1 year of oral sirolimus
时间窗: baseline, 1 month, 3 months, 6 months, 9 months and 12 months
次要结局
- To assess time to attain resolution & safety of oral sirolimus in vascular malformation(baseline, 1 month, 3 months, 6 months, 9 months & 12 months)
研究者
Gomathy Sethuraman
MD Dermatology, professor, dept of dermatology, All India Institute of Medical Sciences, New Delhi