Surgical correction of spina bifida in the womb

In-Utero Endoscopic Correction of Spina Bifida: Laparotomy-Assisted or Percutaneous

This study is testing two different surgical methods to see which one works better for fixing spina bifida in babies while they are still in the womb.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 110 (estimated)
• Ages: 18 Years to 52 Years
• Sex: Female
• Sponsor: University of Southern California (other)
• Locations: 2 sites (Los Angeles, California and 1 other locations)
• Trial ID: NCT04362592 on ClinicalTrials.gov

What this trial studies

This study evaluates the feasibility and effectiveness of fetoscopic surgical correction for fetal spina bifida using two different surgical techniques: a percutaneous approach and a laparotomy/uterine exteriorization technique. The goal is to determine which method is more effective in treating myelomeningocele, a severe form of spina bifida. The study will involve pregnant women with specific eligibility criteria, including gestational age and maternal health assessments. Participants will undergo follow-up evaluations to assess outcomes related to the procedure.

Who should consider this trial

Good fit: Ideal candidates are pregnant women aged 18 and older, with a diagnosis of myelomeningocele at specific spinal levels and gestational age between 19 to 27 weeks.

Not a fit: Patients with multiple gestations or those whose conditions do not meet the specific inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve outcomes for fetuses diagnosed with spina bifida.

How similar studies have performed: Other studies have shown promise in similar surgical approaches for treating spina bifida, indicating potential for success in this trial.

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.

Where this trial is running

Los Angeles, California and 1 other locations

• Hollywood Presbyterian Medical Center — Los Angeles, California, United States (RECRUITING)
• Wellington Regional Medical Center — Wellington, Florida, United States (RECRUITING)

Study contacts

• Principal investigator: Ruben Quintero, MD — US Fetus
• Study coordinator: Ruben Quintero, MD

Conditions: Neural Tube Defects, Spina Bifida, Myelomeningocele