Myelomeningocele Repair Randomized Trial-DSCC

脊髓脊膜膨出修复随机试验-DSCC

基本信息

批准号：
7568611
负责人：
ELIZABETH ANN THOM
金额：
$ 123.51万
依托单位：
GEORGE WASHINGTON UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2002
资助国家：
美国
起止时间：
2002-04-11 至 2012-09-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7568611
关键词：
Academic Medical Centers Adherence Advertisements Affect Age Age-Months Age-Years Archives Biometry Brain Stem Case Report Form Cerebellum Cessation of life Child Clinical Closure Cognitive Condition Data Data Base Management Data Coordinating Center Data Quality Data Reporting Diagnosis Documentation Eligibility Determination Enrollment Ensure Evaluation Fetus Goals Hydrocephalus Incidence Incontinence Individual Infant Infant Development Internet Intervention Leadership Learning Lesion Life Lower Extremity Magnetic Resonance Imaging Mails Manuals Manuscripts Measures Meningomyelocele Monitor Mothers Motor Movement Neonatal Nervous System Physiology Neurological outcome Numbers Online Systems Open Spina Bifida Operative Surgical Procedures Orthopedic Procedures Outcome Pamphlets Patients Pediatric Hospitals Philadelphia Physicians Pregnancy Preparation Printing Procedures Protocols documentation Psychological Tests Publications Purpose Quality Control Quality of life Questionnaires Radiology Specialty Randomized Randomized Clinical Trials Randomized Controlled Clinical Trials Reflex action Reporting Research Design Research Personnel Risk Safety San Francisco Screening procedure Shunt Device Site Spastic Spinal Dysraphism Standards of Weights and Measures System Training Universities Update Ventricular Washington Week abstracting base cognitive function computerized computerized data processing data management evaluation/testing fetal fetus surgery improved in utero indexing malformation member mental development muscle strength neonatal morbidity patient oriented postnatal programs psychologic repaired reproductive reproductive function urinary

项目摘要

Since 1997, 180 fetuses have had in utero closure of myelomeningocele (MMC) by open fetal surgery. Preliminary clinical evidence suggests that this procedure reduces the incidence of shunt-dependent hydrocephalus and restores the cerebellum and brainstem to more normal configuration. However, clinicalresults of fetal surgery for MMC are based on comparisons with historical controls and examine only efficacy and not safety. The Myelomeningocele Repair Randomized Trial is a multi-centerunblinded randomized clinical trial of 200 patients that will be conducted at three Fetal Surgery Units (FSU), the University of Califomia-San Francisco, Children's Hospital of Philadelphia, and Vanderbilt University Medical Center. The primary objective of the trial is to determine if intrauterine repair of fetal myelomeningocele at 18¿to 256 weeks gestation improves outcome, as measured by 1) death or the need for ventricular decompressive shunting by one year of life and 2) death or Bayley Mental Development Index, as compared to standardpostnatal repair This proposal is for the George Washington University Biostatistics Center to serve as the Data and Study Coordinating Center (DSCC) for the MMC Repair Trial. The purpose of the DSCC, an important but independent member of the multi-centercollaborative study group, is to provide expertise and support in study design, study conduct and statistical analysis. We will provide scientific leadership in the design of the study and prepare the final study documents includingthe protocol, manual of operations and case report forms. The DSCC will be responsible for all publicity for the MMC Repair Trial such as establishing a central web site, mailing of physician brochures, presenting trial information at appropriate professional meetings and placing print advertisements in medicaljournals and patient oriented publications. We will also serve as the central referral site for patients to learn more about the trial, conduct preliminary review of patient eligibility and assign the patient to a Fetal Surgery Unit for final evaluation. The DSCC will maintain an Internetrandomization system and web-based data entry system for the patient eligibility data. We will provide a comprehensive data processing system including central data entry, data base management and data quality control. The DSCC will use appropnate statisticaltechniques to conductinterim and final analyses. We will assist the investigators in preparationof manuscriptsand abstracts from studyresults. In summary, we will participate in cooperation with the FSUs on the proposed trial with the goal of demonstrating whether fetal intervention offers improvedoutcome with a reasonable quality- of life for spina bifida children.

自 1997 年以来，已有 180 名胎儿通过开放胎儿手术在子宫内进行了脊髓脊膜膨出 (MMC) 闭合术。临床证据表明，该手术可降低分流依赖性脑积水的发生率并恢复小脑和脑干的形态更加正常，但是，MMC 胎儿手术的临床结果是基于的。与历史对照进行比较，仅检查疗效而不检查安全性。脊髓脊膜膨出修复随机试验是一项多中心、非盲法随机临床试验，纳入了 200 名患者患者将在加州大学旧金山分校的三个胎儿外科中心 (FSU) 进行手术，费城儿童医院和范德比尔特大学医学中心试验的主要目的是。确定胎儿脊髓脊膜膨出是否在 18° 进行宫内修复根据测量，妊娠 256 周可改善结局 1) 死亡或需要进行心室减压分流，生命满一年；2) 死亡或贝利精神疾病发育指数，与标准产后修复相比本提案由乔治华盛顿大学生物统计中心作为数据和研究中心 MMC 修复试验协调中心 (DSCC) DSCC 的目的是一个重要但独立的机构。多中心协作研究组成员，在研究设计、研究实施等方面提供专业知识和支持我们将在研究设计和准备最终研究方面提供科学领导。 DSCC 将负责所有文件，包括方案、操作手册和病例报告表。 MMC 修复试验的宣传，例如建立中央网站、邮寄医师手册、展示在适当的专业会议上提供试验信息，并在医学期刊和患者上刊登印刷广告我们还将作为患者了解更多有关试验、进行试验的中心转诊网站。初步审查患者资格，并将患者分配至胎儿手术室进行最终评估。我们将维护一个互联网随机化系统和基于网络的患者资格数据输入系统。提供全面的数据处理系统，包括中央数据输入、数据库管理和数据质量 DSCC 将使用适当的统计技术进行中期和最终分析。研究人员准备研究结果的手稿和摘要。总之，我们将参与与 FSU 合作开展拟议的试验，目的是证明胎儿干预是否可以为脊柱裂儿童提供改善的结果和合理的生活质量。