Silent Cerebral Infarct Multi-Center Clinical Trial -SCC

无症状脑梗塞多中心临床试验-SCC

基本信息

批准号：
7892030
负责人：
Bruce A Barton
金额：
$ 7.06万
依托单位：
MARYLAND MEDICAL RESEARCH INSTITUTE, INC
依托单位国家：
美国
项目类别：
财政年份：
2003
资助国家：
美国
起止时间：
2003-09-30 至 2010-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7892030
关键词：
12 year old 13 year old 6 year old Accounting Acute Affect Age Alloimmunization Archives Benefits and Risks Blood Blood Transfusion Brain Brain Injuries Cerebral Infarction Cerebrum Chest Pain Child Childhood Chronic Clinical Clinical Trials Cognitive Companions Controlled Study Data Data Analyses Data Collection Data Quality Detection Distal Doctor of Medicine Documentation Dropout Drops Effectiveness Enrollment Ensure Erythrocytes Evaluation Event Family Fostering Glutamic Acid Goals Healthcare Hematology Hemoglobin Hemoglobin SS Hemolytic Anemia Hospitalization Hour Infarction Infusion procedures Injury Internal carotid artery structure Intervention Intervention Trial Iron Iron Chelating Agents Ischemic Brain Injury Lead Lesion Magnetic Resonance Imaging Manuals Maryland Master of Public Health Measurement Measures Medical Medical Research Monitor Morbidity - disease rate Motor Multi-Institutional Clinical Trial Neurocognitive Neurologic Neurologic Examination Neurologist Neurology Operative Surgical Procedures Paralysed Parents Participant Patients Performance Phase Phase III Clinical Trials Point Mutation Population Positioning Attribute Preparation Prevention therapy Principal Investigator Procedures Process Publications Quality Control Quality of life Random Allocation Randomized Randomized Controlled Clinical Trials Reaction Recording of previous events Reporting Research Design Research Institute Research Personnel Risk Schedule Screening procedure Sickle Cell Anemia Site Standardization Stroke Stroke prevention Testing Thalassemia Time Study Training Transfusion United States National Institutes of Health Upper arm Valine Veno-Occlusive Disease Virus Diseases Work base clinical research site follow-up middle cerebral artery mortality nervous system disorder prevent programs prophylactic prospective protocol development risk benefit ratio sickling standard care subcutaneous

项目摘要

DESCRIPTION (provided by the applicant): Silent Cerebral Infarct is the most common cause of severe neurological disease in children with Sickle Cell Anemia, occurring in 22% of this population prior to their 16th birthday. The overall goal of this trial is to determine whether blood transfusion therapy will decrease further neurologic morbidity in children with Silent Cerebral Infarcts and, if so, the magnitude of this benefit. We propose a multi-center, randomized trial with 22 Clinical Sites, a Clinical Coordinating Center, and a Statistical Coordinating Center to test the following hypothesis: Prophylactic Blood Transfusion therapy in children with Silent Cerebral Infarcts will result in at least an 86% reduction in the rate of subsequent overt strokes or new cerebral infarcts, as defined by Magnetic Resonance Imaging (MRI) of the brain. The intervention is blood transfusion therapy vs. observation, with a goal to keep the maximum hemoglobin-S concentration less than 30% in the transfused patients. As many as 3,020 children with Sickle Cell Anemia, greater than 6 years of age and less than 13 years of age, are available to the Clinical Consortium, and will be eligible for screening evaluations. Among this group of eligible patients, approximately 1,880 children will be asked to participate with MRIs of the brain. In this group, we estimate that 376 children (20%) will have Silent Cerebral Infarcts, among whom 38 children (10%) will have an elevated TCD measurement greater than or equal to 200 cm./sec., and will not be eligible for the study. The remaining 338 children will be eligible for random allocation to either observation or blood transfusion therapy. Based on a 60% parent and child acceptance rate among children with Silent Cerebral Infarcts, a study size of 203 participants (at least 101 in each treatment assignment) ensures 90% power to detect the effect necessary to make transfusion therapy worth recommending (86% reduction), after accounting for 10% dropout and 20% crossover rates. The standardization of the trial will occur during the first six months, enrollment for 24 months, and the intervention for 36 months. Twelve months after enrollment, each patient will receive a MRI and TCD. Each study participant will receive MRI and cognitive assessment at study exit (month 36 after enrollment). The primary aim of this trial is to determine the effectiveness of blood transfusion therapy for prevention of stroke or new, Silent Cerebral Infarct assessed by MRI of the brain. The secondary aims of this trial are determining whether: 1) Prophylactic Blood Transfusion therapy will limit further decline in general intellectual abilities; and 2) the overall benefits of blood transfusion therapy for silent cerebral infarcts outweigh risks associated with this therapy in a formal risk-benefit analysis. We anticipate results of this study could lead to a change in standard care practices for children affected with both Sickle Cell disease and Silent Cerebral Infarcts.

描述（由申请人提供）：沉默的脑梗塞是镰状细胞贫血儿童严重神经系统疾病的最常见原因，在16岁生日之前发生在该人群的22％。该试验的总体目的是确定输血疗法是否会降低静音脑梗塞儿童的进一步神经系统发病率，如果是这样，则该益处的幅度。 We propose a multi-center, randomized trial with 22 Clinical Sites, a Clinical Coordinating Center, and a Statistical Coordinating Center to test the following hypothesis: Prophylactic Blood Transfusion therapy in children with Silent Cerebral Infarcts will result in at least an 86% reduction in the rate of subsequent overt strokes or new cerebral infarcts, as defined by Magnetic Resonance Imaging (MRI) of the 脑。干预措施是输血疗法与观察疗法，目的是使输血患者的最大血红蛋白-S浓度低于30％。临床财团可用多达3,020名患有镰状细胞贫血的儿童，大于6岁，年龄少于13岁，年龄少于13岁，并且有资格进行筛查评估。在这组符合条件的患者中，将要求大约1,880名儿童与大脑MRI一起参加。在这一组中，我们估计有376名儿童（20％）将具有无声的脑梗塞，其中38名儿童（10％）将具有大于或等于200 cm./sec。的TCD测量升高，并且不符合该研究的资格。其余的338名儿童将有资格进行观察或输血疗法的随机分配。基于沉默脑梗塞的儿童的父母和儿童接受率的60％，研究规模为203名参与者（在每个治疗分配中至少101个）确保了90％的功率，以检测值得推荐的输血疗法所需的效果（减少86％），在核算10％的辍学和20％的交叉率后。试验的标准化将在前六个月内进行，注册24个月，干预持续36个月。入学率十二个月后，每位患者将获得MRI和TCD。每个研究参与者将在研究退出时接受MRI和认知评估（入学后的第36个月）。该试验的主要目的是确定输血疗法对预防中风或由大脑MRI评估的新的，无声的脑梗塞的有效性。该试验的次要目的是确定：1）预防性输血疗法将限制一般智力能力的进一步下降； 2）在正式的风险效益分析中，输血疗法对静音脑梗塞的总体益处大于与这种疗法相关的风险。我们预计这项研究的结果可能会导致患有镰状细胞疾病和无声脑梗塞的儿童的标准护理实践发生变化。