Clinical trial NCT04362592: In-Utero Endoscopic Correction of Spina Bifida
Official title: In-Utero Endoscopic Correction of Spina Bifida: Laparotomy-Assisted or Percutaneous
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Neural Tube Defects
Spina Bifida
Myelomeningocele
Lead sponsor: University of Southern California
Sponsor class: OTHER
Collaborators: name: USFetus
First posted: 2020-04-27
Enrollment: 110
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 52
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the feasibility and effectiveness of performing fetoscopic surgical correction of fetal spina bifida. Two surgical approaches will be utilized: the percutaneous technique versus the laparotomy/uterine exteriorization technique.


Eligibility criteria: Inclusion Criteria:

1. Myelomeningocele (including myeloschisis) at level T1 through S1 with hindbrain herniation. Lesion level and hindbrain herniation will be confirmed by MRI and ultrasonography.
2. Maternal age ≥18 years.
3. Gestational age of 19 to 27 6/7 weeks' gestation as determined by clinical information and evaluation of first ultrasound.
4. Balanced karyotype and/or normal mircoarray with written confirmation of culture results. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is at 24 weeks or more.
5. Positive evaluation of social work consult indicating the patient is capable of consenting to the procedure and has the appropriate social support system to participate in the study.
6. Positive evaluation from pediatric neurology consult.
7. Willing to return to our center, or to a multi-disciplinary spina bifida clinic closer to their home for the 6, 12, 24, 30, 48, and 60 months follow-up evaluations.

Exclusion Criteria:

1. Multiple gestation
2. Insulin-dependent pregestational diabetes
3. Presence of a fetal anomaly not related to Chiari II Malformation. A fetal echocardiogram will be conducted before surgery and if the finding is abnormal, the patient will be excluded.
4. Fetal kyphosis of 30 degrees or more, assessed by ultrasound or MRI.
5. Presence of uterine cervical cerclage or history of incompetent cervix.
6. Placenta previa or placental abruption.
7. Short cervix \< 25 mm measured by cervical ultrasound.
8. Obesity as defined by body mass index (BMI) of 40 or greater.
9. History of previous spontaneous singleton delivery prior to 37 weeks.
10. Maternal-fetal Rh isoimmunization, Kell sensitization or a history of neonatal alloimmune thrombocytopenia.
11. Maternal HIV or Hepatitis-B status positive because of the increased risk of transmission to the fetus during maternal-fetal surgery. If the patient's HIV or Hepatitis B status is unknown, the patient must be tested and found to have negative results before she can be enrolled.
12. Known Hepatitis-C positivity. If the patient's Hepatitis C status is unknown, she does not need to be screened.
13. Uterine anomaly such as large (greater than 6 cm) fibroids, cervical fibroids or multiple fibroids or Mullerian duct abnormality.
14. Other maternal medical condition which is a contraindication to surgery or anesthesia.
15. Patient does not have a support person (e.g., husband, partner, parents).
16. Inability to comply with the travel and follow-up requirements of the study.
17. Patient does not meet psychosocial criteria as determined by the social worker evaluation.
18. Participation in another intervention study that influences maternal and fetal morbidity and mortality.
19. Maternal hypertension as determined by the investigator, which would increase the risk of preeclampsia or preterm delivery (including, but not limited to: uncontrolled hypertension, chronic hypertension with end organ damage and new onset hypertension in current pregnancy).
20. Bicornuate uterus or any other uterine malformation the PI decides is not safe for surgery.
21. Nickel allergy.
22. Maternal request to undergo open fetal surgery for the antenatal correction of OSB at our institution primarily or after failed fetoscopic approach.
23. Known maternal hypersensitivity to bovine collagen or chondroitin materials.

Primary outcomes:
measure: Ability to perform the endoscopic procedure
timeFrame: At time of surgery
Secondary outcomes:
measure: Chiari II malformation reversal
timeFrame: Prior to delivery
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Hollywood Presbyterian Medical Center
state: California
city: Wellington
country: United States
facility: Wellington Regional Medical Center
state: Florida
Study officials:
affiliation: US Fetus
name: Ruben Quintero, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Southern California
name: Ramen Chmait, MD
role: PRINCIPAL_INVESTIGATOR
References: