Myelomeningocele Repair Randomized Trial
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 183
- 试验地点
- 6
- 主要终点
- Infant Death or Need for Ventricular Shunt by 1 Year of Life
研究概览
简要总结
Spina bifida (myelomeningocele) is a complex birth defect in which a portion of the spinal cord is not fully developed. The overlying bones and skin are incompletely formed and the underdeveloped area of the spinal cord is exposed on the surface of the back. Spina bifida defects are closed soon after birth to prevent further damage to the spinal cord and nerves. The Management of Myelomeningocele Study (MOMS) is a research study comparing two approaches to the treatment of babies with spina bifida: surgery before birth (prenatal surgery) and the standard closure, surgery after birth (postnatal surgery).
详细描述
Since 1997, more than 200 fetuses have had in utero closure of myelomeningocele by open maternal-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 prenatal surgery for myelomeningocele are based on comparisons with historical controls and examine only efficacy, not safety. MOMS will determine if intrauterine repair of fetal myelomeningocele at 19 to 25 weeks of gestation improves outcomes as compared to standard postnatal repair. Outcomes assessed include death, the need for ventricular decompressive shunting by one year of life and neurologic function at 30 months of age.
One hundred eighty-three women, whose fetuses have spina bifida, were enrolled in the study and randomized to have either prenatal surgery or postnatal surgery. After a central screening process which included a medical record review, all women had an extensive baseline evaluation that included ultrasound, MRI, physical exam, social work evaluation, psychological screening, and education about spina bifida and prenatal surgery.
For women who were eligible following the central screening process, all screening, surgery and follow-up visits were performed at one of three MOMS Centers. The mother, if eligible, and her support person traveled (at the expense of the study) to the MOMS Center for screening and randomization.
Women assigned to have prenatal surgery were scheduled for surgery within 1 to 3 days after they were randomized. They stayed near the MOMS Center until they delivered. Women in the postnatal group traveled back to their assigned MOMS Center to deliver. Both groups delivered their babies by C-section around the 37th week of their pregnancies. Babies born to women in the postnatal surgery group had their spina bifida defects closed when they were medically stable, usually within 48 hours of birth.
Children and their parents returned to their assigned MOMS Center at 1 year and 2 ½ years of age for follow-up evaluation. Motor function, developmental progress, and bladder, kidney, and brain development were assessed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 是
入选标准
- •Pregnant women carrying a fetus diagnosed with myelomeningocele
- •Myelomeningocele lesion that starts no higher than T1 and no lower than S1 with hindbrain herniation present
- •Gestational age at randomization of 19 weeks 0 days to 25 weeks 6 days
- •Normal karyotype
- •Singleton pregnancy
- •United States resident
- •Able to travel to study site for study evaluation, procedures, and visits (if randomized to prenatal surgery, must stay near center until delivery)
- •Support person to travel and stay with participant
排除标准
- •Maternal insulin-dependent pregestational diabetes
- •Short or incompetent cervix or cervical cerclage
- •Placenta previa
- •Body mass index of 35 or more
- •Previous spontaneous delivery prior to 37 weeks
- •Maternal HIV, Hepatitis-B or Hepatitis-C status positive
- •Uterine anomaly
- •Maternal medical condition which is a contraindication to surgery or general anesthesia
- •Other fetal anomaly
结局指标
主要结局
Infant Death or Need for Ventricular Shunt by 1 Year of Life
时间窗: 12 months of age
Bayley Scales of Infant Development MDI and the Difference Between the Functional and Anatomical Level of Lesion at 30 Months of Age
时间窗: 30 months of age
Individual outcome score is the sum of the following: 1. Rank for the Bayley score which was constructed from the Bayley Scales of Infant Development Mental Development Index standardized score for each child at 30 months. Deaths had the lowest score of 0, lower than the lowest standardized score of 49. Scores were then ranked from 1 to 182 (1 is worst,182 is best). 2. Rank for the difference between the anatomic and functional lesion levels of the spine was generated by a plain x-ray obtained at the 12-month visit for the anatomic level and the physical examination at 30 months for the functional level. The difference between the two was calculated where a positive difference means that the child is functioning better than expected by the level of his/her lesion. Deaths received the lowest score of -25, lower than all other possible differences. The differences were then ranked from 1 to 182 (1 is worst, 182 is best). For the overall score, 2 is the worst and 364 is the best.
次要结局
- Number of Participants Walking Independently at Examination(30 months of age)