Myelomeningocele Repair Randomized Trial-DSCC

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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.