CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 123
- 试验地点
- 1
- 主要终点
- Rate of Recurrent Symptomatic Hemorrhage During the Two Year Follow-up Period in Patients With CASH.
研究概览
简要总结
Brain Cavernous Angiomas with Symptomatic Hemorrhage (CASH) are rare, but they exact a heavy burden of neurologic disability from recurrent bleeding, for which there is no proven therapy. This trial readiness project aims to address current critical obstacles in identifying cases at multiple sites, characterizing their relevant features, and measuring their outcome. The timing cannot be more opportune, with therapeutic targets already identified, exceptional collaboration among researchers and with the patient community, and several drugs ready to benefit from a track to clinical testing in the next five years.
详细描述
The Trial Readiness grant mechanism, funded by NINDS, proposes to address knowledge gaps and establish a research network as infrastructure for future research. This project includes an observational cohort study of 181 patients with an operational goal of demonstrating the feasibility of screening, enrollment rates, baseline disease categorization and follow-up of CASH using common data elements at multiple sites, and to assess the following endpoints for 123 participants enrolled at centers prespecified to do prospective follow-up: (1) the rates of recurrent hemorrhage; (2) the reliability of imaging biomarkers including quantitative susceptibility mapping and permeability measures which have been shown to correlate with lesion activity, and (3) change in functional status during prospective follow-up.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- 未提供
排除标准
- 未提供
结局指标
主要结局
Rate of Recurrent Symptomatic Hemorrhage During the Two Year Follow-up Period in Patients With CASH.
时间窗: 2 year
Number of new bleeds reported during the two year follow-up period in the cohort with CASH. We will assess the change in the number of new symptomatic hemorrhage from baseline to year 1 and year 1 to year 2 follow-up.
次要结局
- Median Score of European Quality of Life Visual Analogue Scale (EQ-VAS) During the 2 Year Follow-up Period.(2 years of follow-up)
- Percent Change in QSM Value (Lesional Iron Content)(2 years of follow-up)
- Percent Change in Contrast-enhanced Quantitative Perfusion (DCEQP) Value (Vascular Permeability)(2 years of follow-up)
- Compare the Number of Patients With MRS 2 or Higher Who Had Prospective Symptomatic Hemorrhage to Those With MRS 2 or Higher Without Prospective Symptomatic Hemorrhage During the 2 Year Follow-up Period.(2 years of follow-up)
- Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Self-care" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Fatigue" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Pain" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Physical Function" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Depression" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Percentage of Patients With National Institutes of Health Stroke Scale (NIHSS) 0-4, 5-14 and 14+ During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Anxiety" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Sleep Disturbance" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)
- Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Mobility" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period.(2 years of follow-up)
- Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Usual Activities" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period.(2 years of follow-up)
- Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Pain / Discomfort" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period.(2 years of follow-up)
- Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Anxiety / Depression" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period.(2 years of follow-up)
- Median T-score for "Social" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.(2 years of follow-up)