Fetoscopic Surgery for Spina Bifida

Recruiting at 1 trial location

Overseen By Ramen Chmait, MD

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: USFetus

Disqualifiers: Diabetes, Obesity, Hypertension, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 21 weeks

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new surgical technique for treating spina bifida before birth. Spina bifida occurs when the spine and spinal cord do not form properly, and the trial aims to correct it using a minimally invasive procedure called fetoscopy. The trial will explore two surgical methods based on the placenta's position. Pregnant women between 19 and 27 weeks, with a diagnosis of spina bifida in their unborn child, may be suitable candidates, especially if they can remain near the designated hospitals for the study and follow-up visits. As an unphased trial, this study offers a unique opportunity to contribute to pioneering research that could improve outcomes for future families facing spina bifida.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that this fetoscopic surgical technique is safe for antenatal correction of fetal myelomeningocele?

A previous study on fetoscopic repair of spina bifida identified some risks. Specifically, 26% of pregnancies experienced a separation of the membranes around the baby, and there was a 46% chance of premature membrane rupture.

Another study found no difference in complication rates between different surgical methods, suggesting that the type of surgery might not significantly impact safety. However, surgery before birth was linked to better outcomes for the baby compared to surgery after birth.

Overall, despite some risks, fetoscopic surgery has demonstrated potential benefits. Prospective participants should consider these factors when deciding about joining such a trial. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Fetoscopic surgery for spina bifida is unique because it offers a minimally invasive approach to repair the spinal defect while the baby is still in the womb. Unlike traditional open fetal surgery, which involves a larger incision in the mother's abdomen and uterus, fetoscopy uses tiny instruments and a camera inserted through small incisions. This method aims to reduce risks for both the mother and the baby, such as preterm labor and complications from large surgical wounds. Researchers are excited about this technique because it holds the potential to improve neurological outcomes for the baby while minimizing recovery time and stress for the mother.

What evidence suggests that fetoscopic surgery is effective for spina bifida?

Studies have shown that fetoscopic surgery for spina bifida, which participants in this trial will undergo, can improve outcomes for babies. Research indicates that this technique reduces the likelihood of needing a wheelchair later in life and leads to better physical abilities compared to traditional open surgery. In one study, fetoscopic surgery resulted in 130 more cases where babies had much better movement abilities than expected. This method is less invasive, potentially making it safer for both the mother and the baby compared to open surgery. Overall, fetoscopic surgery shows promise in enhancing the quality of life for children with spina bifida. ^{2 6 7 8 9}

Who Is on the Research Team?

Ruben Quintero, MD

Principal Investigator

Wellington Regional Medical Center

Ramen Chmait, MD

Principal Investigator

University of Southern California/ Huntington Memorial Hospital

Are You a Good Fit for This Trial?

This trial is for pregnant women over 18 with a fetus diagnosed with myelomeningocele, between 19-27 weeks' gestation. They must be able to stay near Wellington or Pasadena for the pregnancy and delivery, and return for follow-ups. Exclusions include multiple pregnancies, certain health conditions like HIV/Hepatitis-B, obesity (BMI of 35+), nickel allergy, pregestational diabetes, uterine anomalies, previous early deliveries.

Inclusion Criteria

Willingness to remain in the greater Wellington or Pasadena area (within a 30-minute car ride) for remainder of the pregnancy and deliver at Wellington Regional Medical Center or Huntington Memorial Hospital for postnatal management

I can come back for check-ups at 12, 24, 30, 48, and 60 months.

My test results confirm a balanced karyotype, acceptable through FISH if I'm 24 weeks along or more.

Exclusion Criteria

I want to have surgery for my baby's spina bifida before birth at this hospital.

The mother has a medical condition that makes surgery or anesthesia unsafe.

Participation in another intervention study influencing maternal and fetal morbidity and mortality

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo fetoscopic surgical correction of myelomeningocele using either laparotomy or percutaneous techniques

Up to 21 weeks

Surgery and follow-up assessments until delivery

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Fetoscopy

Trial Overview The study tests a fetoscopic surgical technique versus traditional surgery to correct fetal myelomeningocele before birth. The percutaneous approach is used if the placenta is at the back; otherwise laparotomy/uterine exteriorization can be done regardless of placental location.

How Is the Trial Designed?

Group I: Fetoscopic repair Experimental Treatment 1 Intervention

Fetoscopy is already approved in United States, European Union for the following indications:

Approved in United States as Fetoscopic surgery for:

• Myelomeningocele repair
• Spina bifida correction

Approved in European Union as Fetoscopic surgery for:

• Myelomeningocele repair
• Spina bifida correction

Who Is Running the Clinical Trial?

USFetus

Lead Sponsor

Trials

2

Recruited

120+

Wellington Hospital

Collaborator

Trials

11

Recruited

1,500+

University of Southern California

Collaborator

Trials

956

Recruited

1,609,000+

Published Research Related to This Trial

Minimally invasive fetoscopic surgery for spina bifida aperta aims to protect exposed neural tissue and preserve neurological function, highlighting its potential as a new therapeutic approach.

In a retrospective analysis of 61 cases, a carefully developed anesthetic protocol was shown to effectively ensure safe maternal and fetal anesthesia, uterine relaxation, and prevention of complications such as pulmonary edema and spontaneous labor.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anesthetic Management for Percutaneous Minimally Invasive Fetoscopic Surgery of Spina Bifida Aperta: A Retrospective, Descriptive Report of Clinical Experience. Arens, C., Koch, C., Veit, M., et al.[2018]

Minimally invasive fetoscopic surgery for spina bifida aims to enhance the neurological outcomes for affected fetuses while reducing maternal trauma compared to traditional open fetal surgery.

The article provides insights into the perioperative and postoperative management practices for these cases, highlighting the advancements in surgical techniques for better patient care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Peri- and postoperative management for minimally invasive fetoscopic surgery of spina bifida]. Degenhardt, J., Axt-Fliedner, R., Enzensberger, C., et al.[2014]

A minimally invasive fetoscopic approach for closing spina bifida aperta in two fetuses resulted in successful neural cord coverage and avoided the need for postnatal neurosurgery, demonstrating its efficacy.

Both neonates showed significant improvements, including reversal of hindbrain herniation and satisfactory leg, bladder, and bowel function, indicating the potential for this technique to enhance outcomes compared to traditional methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Percutaneous fetoscopic patch closure of human spina bifida aperta: advances in fetal surgical techniques may obviate the need for early postnatal neurosurgical intervention. Kohl, T., Tchatcheva, K., Merz, W., et al.[2018]

Prenatal repair of open spina bifida through fetoscopic techniques shows promise in reducing shunt rates and improving postnatal neurological outcomes, comparable to traditional open surgery.

The laparotomy-based fetoscopic approach has been associated with fewer complications and higher gestational ages at delivery compared to the percutaneous method, which is still being refined to improve outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Endoscopic fetal surgery for neural tube defects. Lapa, DA.[2020]

In a study involving 7 women with spina bifida aperta, laparotomy-assisted fetoscopic repair was performed successfully without fetal demise, demonstrating its potential as a safe prenatal surgical option.

The procedure resulted in watertight repairs at birth for most cases, with some needing postnatal surgery; however, 50% of the infants required shunting within 6 months, indicating the need for careful monitoring post-surgery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Laparotomy-Assisted 2-Port Fetoscopic Repair of Spina Bifida Aperta: Report of a Single-Center Experience in Paris, France. Arthuis, C., James, S., Bussieres, L., et al.[2023]

The laparotomy-assisted 2-port fetoscopic technique for prenatal closure of spina bifida was successfully completed in 84% of cases, indicating its feasibility and effectiveness in a cohort of 19 women.

Postoperative improvements were observed in 86% of undelivered patients regarding hindbrain herniation, suggesting that this technique can lead to significant fetal health benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Implementation Process and Evolution of a Laparotomy-Assisted 2-Port Fetoscopic Spina Bifida Closure Program. Miller, JL., Groves, ML., Ahn, ES., et al.[2021]

The first five percutaneous fetoscopic in-utero spina bifida repair procedures in the UK highlighted the critical importance of managing anesthesia and analgesia to ensure the safety of both the mother and fetus during surgery.

Key strategies were implemented to minimize risks such as fetal acidosis and maternal fluid overload, emphasizing the need for careful monitoring and optimization of conditions to support fetal well-being during these complex procedures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Peri-operative management of percutaneous fetoscopic spina-bifida repair: a descriptive review of five cases from the United Kingdom, with focus on anaesthetic implications. Goonasekera, CD., Skelton, VA., Zebian, B., et al.[2021]

Prenatal repair of open spina bifida through fetoscopic techniques can improve neurological outcomes for infants, reducing the need for shunting and enhancing motor function after birth.

The newly described percutaneous/mini-laparotomy fetoscopy combines the advantages of both laparotomy-assisted and percutaneous methods, potentially offering a safer option for mothers while maintaining effective surgical results for the fetus.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Percutaneous/mini-laparotomy fetoscopic repair of open spina bifida: a novel surgical technique. Chmait, RH., Monson, MA., Pham, HQ., et al.[2022]

The fetoscopic surgical approach for repairing myelomeningocele resulted in significantly better maternal and neonatal outcomes compared to the open approach, including fewer cases of preterm birth, neonatal death, and neurodevelopmental disabilities in both the index and subsequent pregnancies.

Although the fetoscopic technique was more expensive, it was deemed cost-effective, with an incremental cost-effectiveness ratio of $1029 per quality-adjusted life year, indicating that the improved outcomes justify the higher costs in a theoretical cohort of 500 women.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetoscopic compared with open repair of myelomeningocele: a 2-delivery cost-effectiveness analysis. Packer, CH., Hersh, AR., Caughey, AB.[2021]

Percutaneous fetoscopic patch coverage for spina bifida aperta in human fetuses is technically feasible and results in less maternal trauma compared to traditional open fetal repair methods.

Out of three cases, two children survived after the procedure, indicating potential for this approach, but further clinical experience is needed to fully assess its efficacy in protecting neural tissue and improving outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Percutaneous fetoscopic patch coverage of spina bifida aperta in the human--early clinical experience and potential. Kohl, T., Hering, R., Heep, A., et al.[2006]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8360048/

Fetoscopic myelomeningocoele closure: Is the scientific ... Likewise, fetal and neonatal deaths (4/131, 3%; two fetal and two neonatal deaths) were similar to what is reported for open spina bifida repair (2/91, 2%; one ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03090633

Fetoscopic Repair of Isolated Fetal Spina Bifida The purpose of this investigation is to evaluate maternal and fetal outcomes following fetoscopic repair of fetal spina bifida at the Johns Hopkins Hospital ...

3. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/uog.29289

Comparison of open fetal, fetoscopic and postnatal surgical ... Open fetal and fetoscopic surgery yielded the greatest number of QALYs due to a substantial reduction in the incidence of wheelchair use and the ...

4. ajogmfm.org ajogmfm.org/article/S2589-9333(21)00129-4/abstract

Fetoscopic compared with open repair of myelomeningocele Fetoscopic technique resulted in 130 more cases of functional level >2 levels better than the anatomic level, 35 fewer cases of functional level ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03562286?term=spina%20bifida&viewType=Table&rank=6

NCT03562286 | Open Spina Bifida Fetoscopic Repair Project The aim of the study is to assess a new fetal surgery approach to repair open spina bifida . The fetal group hypothesis is to perform a minimally invasive ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5113790/

Fetoscopic repair of spina bifida: safer and better? - PMC The primary composite outcome (fetal or neonatal death, or need for VP shunting before 1 year of age) occurred in 68% of the prenatal repair ...

7. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/full/10.1002/uog.13430

Percutaneous minimally invasive fetoscopic surgery for spina ... This paper provides an overview of its development and presents technical data as well as perioperative outcomes from 51 cases that underwent ...

8. ajogmfm.org ajogmfm.org/article/S2589-9333(23)00036-8/fulltext

Fetoscopic repair of spina bifida: continued refinement ... They found that there was no difference between approaches in the rates of surgical complications, obstetrical complications, intrauterine fetal ...

9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9293198/

Percutaneous fetoscopic spina bifida repair A trial comparing prenatal with postnatal open spina bifida (OSB) repair established that prenatal surgery was associated with better postnatal outcome.