Oral Propanolol for Surgically Inaccessible Cerebral and Spinal Cavernous Malformations
试验速览
- 阶段
- 1 期
- 入组人数
- 346
- 试验地点
- 1
- 主要终点
- Number of symptomatic and silent hemorrhages on MRI
研究概览
简要总结
The purpose of this study is to evaluate the role of propranolol as an alternative treatment for cavernous malformation in patients that may not be ideal candidates for surgery.
详细描述
Patients between the ages of 1 and 80 years of age, that have been diagnosed with a spinal or cerebral cavernous malformation(s) that can not be surgically treated, will be enrolled in this randomized study. All eligible patients will be enrolled in one of two groups. Group 1 will receive oral propranolol tablets 3 times a day for 36 months. Group 2 will receive oral placebo tablets 3 times per day for 36 months. Prior to beginning treatment, all imaging, including MRIs, will be evaluated. During the first visit a blood sample will be collected for genomic analysis. Patients will return to clinic every 6 months. Each time the patient returns to clinic monitoring of how the patient is feeling and evaluation of how the patient is tolerating the propranolol will take place. At the end of the three years, evaluators will determine if the propranolol was able to stop the growth of the cavernous malformation and change the imaging features of the lesion on MRI. Once the study is completed patients will no longer receive the study drug.
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
- None
入排标准
- 年龄范围
- 1 Year 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of isolated or familial cavernous malformation syndrome:
- •symptomatic cavernous malformation not amenable to surgical resection.
- •familial cavernous malformation with seizure, other neurological symptom or surgically inaccessible lesion.
- •Written and informed consent obtained prior to study enrollment.
- •Subject is able and willing to return for outpatient visits.
- •Negative pregnancy test at time of enrollment for women and child-bearing potential.
排除标准
- •Age less than 1 year-old.
- •Propranolol allergy or allergy to other b-blockers.
- •Estimated life expectancy of less than 1 year.
- •History of severe anemia, cardiac dysfunction, or diabetes.
- •A psychiatric or substance abuse problem that may interfere with study compliance.
- •Pregnant and lactating women.
研究组 & 干预措施
Group 1: Propranolol Group
Group 1: Propranolol - group of randomized patients will receive one propranolol pill tid for 36 months.
干预措施: Propranolol Oral Tablet (Drug)
Group 2: Placebo Group
Group 2: Placebo - group of randomized patients will receive one placebo pill tid for 36 months.
干预措施: Placebo Oral Tablet (Drug)
结局指标
主要结局
Number of symptomatic and silent hemorrhages on MRI
时间窗: 36 months
Using MRI imaging the size of the cavernous malformation will be measured. The primary outcome is the number of symptomatic and silent hemorrhages as assessed by review of a fine-cut, axial T2-weighted MRI obtained at each visit.
次要结局
- Rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits(36 months)
研究者
Yashar Kalani, MD
Co-Director of Neurovascular Surgery, Director of Skull Base Surgy and Director of Stroke Center
University of Virginia