Multicenter Trial of Fetal Myelomeningocele Repair

胎儿脊髓脊膜膨出修复的多中心试验

• 批准号: 6629395
• 负责人: N SCOTT ADZICK
• 金额: $ 102.64万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-03-15 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6629395
• 关键词: clinical trials; cognition; cooperative study; developmental psychology; embryo /fetus surgery; functional ability; human pregnant subject; human therapy evaluation; hydrocephalus; infant mortality; longitudinal human study; magnetic resonance imaging; meningomyelocele; neuromuscular function; neurosurgery; orthopedics; patient oriented research; psychological tests; psychology; quality of life; reproduction; spina bifida

DESCRIPTION (provided by applicant): 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, clinical results 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-center unblinded randomized clinical trial of 200 patients that will be conducted at three Fetal Surgery Units (FSUs), the University of California-San Francisco, Children s Hospital of Philadelphia, and Vanderbilt University Medical Center, along with an independent Data and Study Coordinating Center (DSCC), the George Washington University Biostatistics Center. The primary objective of the trial is to determine if intrauterine repair of fetal myelomeningocele at 18 to 25 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 standard postnatal repair. Consenting patients who satisfy eligibility criteria will be centrally randomized to either intrauterine or standard postnatal repair of the MMC. Patients assigned to the fetal surgery group will be discharged to nearby accommodation following surgery, unless unfeasible, in which case they will return to their assigned FSU at 32 weeks gestation for delivery at 37 weeks gestation by cesarean section. Patients assigned to the postnatal surgery group will return home and at 37 weeks, return to their assigned FSU for delivery by cesarean section. Magnetic resonance imaging will be conducted at enrollment, discharge or term gestation, and one year of age to determine if intrauterine repair improves the degree of the Chiari II malformation. Neonatal morbidity will be recorded as will the number of surgical procedures for conditions related to the MMC, muscle strength, ambulation status and urinary and fecal continence. Infants will make follow-up visits at twelve and thirty months corrected age for detailed neuromotor function analysis, cognitive testing and evaluation of neurodevelopmental status. In addition, the long term psychosocial and reproductive consequences in mothers will be evaluated. In summary, the proposed trial is expected to demonstrate whether fetal intervention offers improved outcome with a reasonable quality of life for spina bifida children.

描述(申请人提供)：自1997年以来，已有180名胎儿在宫内分娩 开放胎儿手术治疗脊髓脊膜膨出(MMC)初步临床 有证据表明，这种手术减少了分流依赖的发生率。 并使小脑和脑干恢复到更正常的状态 配置。然而，胎儿外科治疗MMC的临床结果是基于 与历史对照进行比较，只检查疗效而不检查安全性。 脊髓脊膜膨出修复术的随机试验是一项多中心非盲法试验 200名患者的随机临床试验将于3点进行 胎儿外科单位(FSU)，加州大学旧金山分校， 费城S医院和范德比尔特大学医学中心的儿童， 与独立的数据和研究协调中心(DSCC)一起， 乔治华盛顿大学生物统计中心。的主要目标是 该试验旨在确定胎儿脊髓脊膜膨出的宫内修复是否在 妊娠18至25周可改善结局，其衡量标准为：(1)死亡或 需要一年生命和(2)死亡的脑室减压术 或者贝利智力发展指数，与标准的产后修复相比。 符合资格标准的同意患者将集中在 随机接受宫内修复或标准产后修复。 分配到胎儿手术组的患者将出院到附近 手术后的住宿，除非不可行，在这种情况下，他们会 在怀孕32周时返回指定的FSU，在37周分娩 剖腹产怀孕。被分配到产后手术的患者 小组将返回家中，并在37周后返回分配给他们的FSU 剖腹产。磁共振成像将在以下时间进行 登记、退伍或足月妊娠，以及一岁以确定是否 宫腔内修补术可改善Chiari II畸形的程度。 新生儿发病率和手术次数将被记录下来 对于与MMC相关的情况，肌肉力量，步行状态和 大小便失禁。婴儿将在12岁和12岁时进行随访 对30个月的校正年龄进行详细的神经运动功能分析， 神经发育状态的认知测试和评估。此外, 母亲的长期心理社会和生殖后果将是 已评估。 总而言之，拟议的试验预计将证明胎儿 干预提供改善的结局和合理的生活质量 脊柱裂儿童。