Clinical trial NCT03936322: Minimally Invasive Fetoscopic Regenerative Repair of Spina Bifida - A Pilot Study
Official title: Minimally Invasive Fetoscopic Regenerative Repair of Spina Bifida - A Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Spina Bifida
Myelomeningocele
Neural Tube Defects
Lead sponsor: University of Miami
Sponsor class: OTHER
Collaborators: name: Mayo Clinic
First posted: 2019-05-03
Enrollment: 3
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Researchers are studying a new minimally invasive technique (fetoscopic repair) for repair of spina bifida (MMC) during the second trimester of pregnancy. Researchers are trying to determine if this less invasive surgical approach will have less risk to the mother and at the same time adequate closure of the fetal spina bifida defect.


Eligibility criteria: Inclusion Criteria

* Pregnant women - maternal age 18 years or older
* Gestational age at the time of the procedure between 19 0/7 weeks and 25 6/7 weeks
* Singleton pregnancy.
* MMC diagnosis with the upper boundary located between Thoracic 1 (T1) and Sacral

  1 (S1).
* Evidence of hindbrain herniation (confirmed on MRI to have an Arnold-Chiari type II malformation).
* Absence of chromosomal abnormalities and associated anomalies.
* Normal karyotype and/or normal chromosomal microarray (CMA) by invasive testing (amniocentesis or CVS). If there is a balanced translocation with normal CMA with no other anomalies the candidate can be included.
* Family has considered and declined the option of termination of the pregnancy at less than 24 weeks.
* Family meets psychosocial criteria (sufficient social support, ability to understand requirements for this study).
* Pregnant subject capable of consenting for their own participation in this study.
* Willingness to undergo an open MMC repair, if necessary
* Parental/guardian permission (informed consent) for follow up of child after birth.

Exclusion Criteria:

* Exclusion Criteria

  * Fetal anomaly unrelated to MMC.
  * Multiple gestation
  * Declined invasive testing for karyotype (amniocentesis or CVS)
  * Severe kyphosis (defined as curvature of the spina (vertebras) higher than 30o degree measured by ultrasonography or magnetic resonance imaging).
  * Increased risk for preterm labor including short cervical length (\<2.0 cm), history of incompetent cervix with or without cerclage, and previous preterm birth.
  * Placental abnormalities (previa, abruption, accreta) known at time of enrollment.
  * A body-mass index ≥40 at first prenatal visit.
  * Contraindications to surgery including previous hysterotomy (whether from a previous classical cesarean, uterine anomaly such as an arcuate or bicornuate uterus, major myomectomy resection, or previous fetal surgery) in active uterine segment.
  * Technical limitations precluding fetoscopic surgery, such as uterine fibroids, fetal membrane separation, uterine anomalies incompatible with fetoscopy.
  * Amniotic Fluid Index (AFI) \< 6 cm if deemed to be due to fetal anomaly, poor placental perfusion or function, or membrane rupture. Low amniotic fluid volume that responds to maternal hydration is not an exclusion.
  * Maternal-fetal Rh isoimmunization, Kell sensitization or neonatal alloimmune thrombocytopenia affecting the current pregnancy.
  * Maternal HIV, Hepatitis-B, Hepatitis-C status positive because of the increased risk of transmission to the fetus during maternal-fetal surgery. If the patients HIV or Hepatitis status is unknown, the patient must be tested and found to have negative results before enrollment.
  * Maternal medical condition that is a contraindication to surgery or anesthesia.
  * A score of ≥ 29 on the Beck Depression Inventory (BDI) at screening
  * Maternal hypersensitivity to collagen
  * Patient does not have a support person (i.e. Spouse, partner, mother) available to support the patient for the duration of the pregnancy.
  * Inability to comply with the travel and follow-up requirements of the trial.
  * Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy.

Primary outcomes:
measure: Maternal Adverse Events
timeFrame: From time of surgery until delivery (up to 21 weeks)
measure: Neonatal Adverse Events
timeFrame: From the time of surgery until 28 days of life (up 25 weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic in Rochester
state: Minnesota
Study officials:
affiliation: University of Miami
name: Rodrigo Ruano, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: