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: 7940506
• 负责人: Diana Lee Farmer
• 金额: $ 57.73万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 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 2012-09-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7940506
• 关键词: Academic Medical Centers; Affect; Age; Age-Months; Age-Years; Biometry; Brain Stem; California; Cerebellum; Cesarean section; Cessation of life; Child; Clinical; Cognitive; Consent; Data; Diagnosis; Eligibility Determination; Enrollment; Fetus; Home environment; Hydrocephalus; Incidence; Incontinence; Individual; Infant; Infant Development; Intervention; Lesion; Life; Lower Extremity; Magnetic Resonance Imaging; Measures; Meningomyelocele; Mothers; Motor; Movement; Multicenter Trials; Neonatal; Nervous System Physiology; Neurological outcome; Open Spina Bifida; Operative Surgical Procedures; Orthopedic Procedures; Outcome; Parents; Patients; Pediatric Hospitals; Philadelphia; Pregnancy; Procedures; Psychological Tests; Quality of life; Questionnaires; Randomized; Randomized Clinical Trials; Randomized Controlled Clinical Trials; Reflex action; Risk; Safety; San Francisco; Shunt Device; Spastic; Spinal Dysraphism; Universities; Ventricular; Visit; Washington; base; cognitive function; evaluation/testing; fetal; fetus surgery; follow-up; improved; in utero; indexing; malformation; mental development; muscle strength; neonatal morbidity; postnatal; psychologic; psychosocial; repaired; reproductive; reproductive function; urinary

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（由申请人提供）：自1997年以来，子宫内有180名胎儿 通过开放式胎儿手术闭合脊髓脑膜膨出（MMC）。初步临床 有证据表明，此过程降低了分流依赖性的发生率 脑积水并将小脑和脑干恢复到更正常的 配置。但是，MMC胎儿手术的临床结果是基于 与历史控制的比较，仅检查功效而不是安全性。 脊髓脑膜静脉维修随机试验是一个多中心的未盲 200例患者的随机临床试验将在三个 加利福尼亚大学弗朗西斯科分校的胎儿手术单元（FSU）， 费城儿童医院和范德比尔特大学医学中心 以及独立的数据和研究协调中心（DSCC）， 乔治华盛顿大学生物统计学中心。主要目标 该试验是确定胎儿内修复胎儿脊髓脑膜纤维的修复 18至25周的妊娠改善了预后，如（1）死亡或 需要一年的心室减压分流和（2）死亡 与标准的产后修复相比，或Bayley心理发展指数。 同意满足资格标准的患者将是中央 随机分配给MMC的宫内或标准后修复。 分配给胎儿手术组的患者将被出院到附近 手术后的住宿，除非不可行，在这种情况下，他们将 在32周妊娠37周时返回其分配的FSU 剖宫产部分的妊娠。分配到产后手术的患者 小组将返回家园，并在37周内返回他们的指定的FSU 剖宫产部分交付。磁共振成像将在 注册，出院或定期妊娠以及一岁以确定是否是否 宫内修复改善了Chiari II畸形的程度。 新生儿发病率将被记录为手术程序的数量 对于与MMC相关的条件，肌肉力量，移动状况和 泌尿和粪便持续性。婴儿将在十二岁时进行后续访问 详细神经运动功能分析的30个月校正年龄， 神经发育状况的认知测试和评估。此外， 母亲的长期社会心理和生殖后果将是 评估。 总而言之，拟议的试验有望证明胎儿是否 干预提供了改进的结果，并具有合理的生活质量 脊柱裂儿童。