MULTICENTER TRIAL OF FETAL MYELOMENINGOCELE REPAIR

Project: Research project

Description

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.
StatusFinished
Effective start/end date7/1/019/30/12

Funding

  • National Institutes of Health: $474,429.00
  • National Institutes of Health: $238,500.00
  • National Institutes of Health: $710,090.00
  • National Institutes of Health: $462,017.00
  • National Institutes of Health: $577,292.00

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Meningomyelocele
Multicenter Studies
Cesarean Section
Pregnancy
Biostatistics
Muscle Strength
Cerebellum
Cognition
Brain Stem
Walking
Fetus
San Francisco
Mothers
Quality of Life
Morbidity
Incidence
Magnetic Resonance Imaging
Clinical Trials
Spinal Dysraphism
Safety

ASJC

  • Medicine(all)