Clinical Trial: Fetoscopic Repair of Isolated Fetal Spina Bifida

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Study of Fetoscopic Repair of Myelomeningocele in Fetuses With Isolated Spina Bifida

Brief Summary:

The purpose of this investigation is to evaluate maternal and fetal outcomes following fetoscopic repair of fetal spina bifida at the Johns Hopkins Hospital.

The hypothesis of this study is that fetoscopic spina bifida repair is feasible and has the same effectiveness as open repair of fetal spina bifida, but with the benefit of significantly lower maternal and fetal complication rates. The fetal benefit of the procedure will be the prenatal repair of spina bifida. The maternal benefit of fetoscopic spina bifida repair will be the avoidance of a large uterine incision. This type of incision increases the risk of uterine rupture and requires that all future deliveries are by cesarean section. The use of the minimally invasive fetoscopic surgical technique may also lower the risk of preterm premature rupture of membranes and preterm birth compared to open fetal surgery. Finally, successful fetoscopic spina bifida repair also makes vaginal delivery possible.


Detailed Summary:

Spina bifida is a congenital anomaly that results from incomplete closure of the neural tube between 22 and 28 embryological days. Its incidence is approximately 2-4 cases per 10,000 births, and it is considered the most common congenital central nervous system anomaly that is compatible with life (CDC). Open spina bifida can present as a flat defect without a covering (myeloschisis), it may have a membranous covering (meningocele), or the fluid may be extruded into a fluid filled sac (myelomeningocele or MMC). Spina bifida can lead to lifelong sequelae that are the result of additional insult to the nervous system that occurs during fetal life as a consequence of the anomaly in the spinal cord. Downward displacement of the brain stem results in hindbrain herniation and the Chiari II malformation during fetal life leading to non-communicating hydrocephalus. Concurrently, intrauterine injury to exposed neural elements leads to neurologic dysfunction.

Despite improved care and technology, 2-year survival of affected individuals is 75%. The need for ventriculoperitoneal shunting for hydrocephaly is related to the level of the lesion and ranges between 88-97% for thoracolumbar lesions. Shunt placement in and of itself is associated with complications such as obstruction, infection, and displacement requiring repeated shunt revisions as early as the first year of life. The majority (75%) of patients with hydrocephaly have radiologic evidence of the Arnold-Chiari II malformation (hindbrain herniation, brain stem abnormalities, and small posterior fossa), which are associated with symptoms of apnea, swallowing difficulties, quadriparesis, balance issues, and coordination difficulties. The lesion level also correlates to the functional motor level; in general, the rate of being wheelchair-bound increases from 17% in sacral lesions to 90% of patients with a thoracic level lesions.
Sponsor: Johns Hopkins University

Current Primary Outcome: Ability to perform fetoscopic spina bifida repair [ Time Frame: From time of surgery until delivery (up to 21 weeks) ]

Successful complete closure of the defect fetoscopically and reversal of hindbrain herniation on ultrasound and MRI prior to delivery


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Maternal obstetric outcome as evidenced by preterm premature rupture of membranes [ Time Frame: From time of surgery until 37 weeks of gestation (up to 18 weeks) ]
    Preterm premature rupture of membranes occuring any time from surgery until 37 weeks of gestation
  • Maternal obstetric outcome as evidenced by preterm labor leading to delivery at less than 34 weeks of gestation [ Time Frame: From time of surgery until 34 weeks of gestation (up to 15 weeks) ]
    Preterm labor occuring at any time from surgery leading to delivery before 34 weeks of gestation
  • Maternal obstetric outcome as evidenced by gestational age at delivery [ Time Frame: From time of surgery until delivery (up to 21 weeks) ]
    Gestational age of delivery regardless of indication
  • Maternal obstetric outcome as evidenced by the ability to delivery vaginally [ Time Frame: From time of surgery until delivery (up to 21 weeks) ]
    Mode of delivery - either vaginal or cesarean section
  • Adverse fetal or neonatal outcome as evidenced by fetal or neonatal death [ Time Frame: From the time of surgery until 28 days of life (up 25 weeks) ]
    Composite of fetal or neonatal death
  • Adverse early childhood outcome as evidenced by need for a cerebrospinal fluid shunt [ Time Frame: From the time of birth until 12 months of life ]
    Need for a cerebrospinal fluid shunt within the first year of life
  • Neurodevelopmental outcome as evaluated by the Bayley Scales of Infant Development II [ Time Frame: 30 months of age ]
    Score of the Mental Developmental Index of the Bayley Scales of Infant Development II at 30 months of age. The score ranges from 50 (minimum) to 150 (maximum). A score of <70 indicates severe developmental delay; 70-84 indicates moderate delay; >85 indicates no delay.
  • Early childhood motor function on physical examination [ Time Frame: 30 months of age ]
    Difference between the anatomic upper border of the lesion level and motor function based on the physical examination at 30 months of age. A positive score of 2 indicates a functional level 2 vertebrae higher than lesion level. A score of -2 indicates a function level 2 vertebrae lower than the lesion level.


Original Secondary Outcome: Same as current

Information By: Johns Hopkins University

Dates:
Date Received: March 6, 2017
Date Started: April 2017
Date Completion: April 2027
Last Updated: March 20, 2017
Last Verified: March 2017