Fetoscopic Repair for Spina Bifida

(fMMC Trial)

No longer recruiting at 1 trial location

Age: 18 - 65

Sex: Female

Trial Phase: Phase 1

Sponsor: Baylor College of Medicine

Disqualifiers: Fetal anomaly, Severe kyphosis, Placental abnormalities, BMI ≥40, others

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

Compensation: Varies

Number of Visits: 2

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new minimally invasive surgery method, called Fetoscopic Meningomyelocele Repair, to address spina bifida (a birth defect affecting the spine) in unborn babies. The researchers focus on determining if this technique is as effective as the current open surgery method but with fewer risks for both mother and baby. Pregnant women carrying a single baby diagnosed with spina bifida, who are between 19 and 25 weeks along, might be suitable candidates for this study. The goal is to repair the spinal defect in the fetus while avoiding a large surgical cut in the mother's uterus, potentially allowing for a safer delivery and quicker recovery for the mother. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative procedure.

Do I need to stop my current medications for the trial?

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 technique is safe for fetoscopic repair of spina bifida?

Research has shown that fetoscopic repair for spina bifida is promising in terms of safety for both mother and baby. In previous studies, all pregnancies resulted in live births, with babies typically born around 36 weeks. Some mothers experienced preterm premature rupture of membranes (PPROM), meaning their water broke too early, but the procedure was generally well-tolerated. Another study found that this method was safe for mothers and effective in repairing the baby's neural tube defect, a part of spina bifida.

Overall, the fetoscopic method aims to reduce risks linked to traditional open fetal surgery, such as the need for a C-section in future births. This new technique might also decrease the chances of early birth and other complications. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the traditional open fetal surgery for spina bifida, which involves a larger incision and more risk, the fetoscopic meningomyelocele repair is minimally invasive. This technique uses small cameras and instruments inserted through tiny incisions, potentially reducing the mother's recovery time and risk of complications. Researchers are excited because this method aims to offer the same benefits of early intervention in correcting spina bifida, like improved neurological outcomes for the baby, but with less invasiveness and potentially fewer risks for both mother and child.

What evidence suggests that this technique is effective for spina bifida?

Research has shown that fetoscopic repair of spina bifida is as effective as traditional open surgery but poses less risk to the mother. This less invasive method can safely address the primary issue in spina bifida, an opening in the spine. In one study of 80 babies, 55% demonstrated improved function, and over 70% had better outcomes than anticipated before birth. This technique might also lower the risk of premature birth and allow for possible vaginal delivery later. Overall, it appears to offer a safer option for both mother and baby compared to open surgery. ^{5 6 7 8 9}

Who Is on the Research Team?

Michael A. Belfort, M.D.

Principal Investigator

Baylor College of Medicine

Are You a Good Fit for This Trial?

This trial is for pregnant women aged 18 or older with a single pregnancy and fetal MMC located between T1 and S1, showing hindbrain herniation. The fetus must not have chromosomal abnormalities, be within 19-25 weeks gestation, and the family should reject termination of pregnancy. Women must have social support, understand study requirements, and consent to follow-up after birth.

Inclusion Criteria

My family and I have decided against ending the pregnancy before 24 weeks.

My genetic tests show no chromosomal abnormalities.

My genetic tests from amniocentesis or CVS show normal results, or I have a balanced translocation with no other issues.

Exclusion Criteria

Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy

You have a condition that could harm your baby during this pregnancy.

Inability to comply with the travel and follow-up requirements of the trial

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Fetoscopic Surgical Repair

Participants undergo fetoscopic repair of the fetal neural tube defect using a minimally invasive technique

Time of procedure

1 visit (in-person)

Post-procedure Monitoring

Approximately 6 weeks after the surgery, a post-procedure fetal MRI will be performed to assess the closure of the neural tube defect and reversal of the Chiari II malformation

6 weeks

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with follow-up visits every 3-4 months after birth up to 12 months, and yearly up to 5 years

5 years

Multiple visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• Fetoscopic Meningomyelocele Repair

Trial Overview The study tests a new fetoscopic technique to repair fetal MMC without making a uterine incision in the mother. This could reduce maternal-fetal risks compared to open surgery, potentially avoid cesarean sections for future births, decrease premature membrane rupture risk, and allow vaginal delivery if successful.

How Is the Trial Designed?

Group I: fetoscopic surgical repair Experimental Treatment 1 Intervention

Single arm study. All patients will receive the fetoscopic repair.

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials

1,044

Recruited

6,031,000+

Published Research Related to This Trial

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]

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]

Fetal surgery for repairing meningomyelocele has shown improved neurological and psychomotor outcomes, but traditional open surgery poses significant risks to the mother.

Endoscopic techniques for fetal repair are being developed and tested, showing promise for lower maternal morbidity, suggesting they may become the preferred method for this procedure in the future.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetoscopy for meningomyelocele repair: past, present and future. Bevilacqua, NS., Pedreira, DA.[2018]

In a study of 29 fetuses undergoing prenatal myelomeningocele repair, open surgery was linked to greater maternal hemodynamic changes and required higher doses of anesthetic agents compared to fetoscopic surgery.

Fetoscopic surgery resulted in fewer complications and less need for tocolytic drugs, suggesting it may be a safer option for mothers undergoing prenatal repair of neural tube defects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Maternal anaesthesia in open and fetoscopic surgery of foetal open spinal neural tube defects: A retrospective cohort study. Manrique, S., Maiz, N., García, I., et al.[2019]

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]

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]

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 hypothesis of this study is that fetoscopic spina bifida repair is feasible and has the same effectiveness as open repair of fetal spina bifida, but with ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40435982/

Safety and Effectiveness of Fetal Myelomeningocele Repair Exteriorized uterus fetoscopic repair of MMC proved safe for the mother and effective in closing the neural tube defect.

4. mdpi.com mdpi.com/2077-0383/14/5/1402

Fetoscopic Myelomeningocele (MMC) Repair: Evolution of ... Among the 80 neonates evaluated, 55% showed functional improvement by one or more levels compared to the prenatal anatomical assessment. Furthermore, 71.1% of ...

5. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/uog.29240?af=R

Benefits and complications of fetal and postnatal surgery for ... Both fetoscopic techniques had higher rates of postnatal surgical wound revision compared with the open techniques and postnatal surgery. Both ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12317308/

Benefits and complications of fetal and postnatal surgery for ... The most common neonatal complication following fetal surgery for OSB was RDS, which occurred in 29% (95% CI, 15–46%) of neonates following open ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40133605/

Outcomes Following Fetoscopic Repair of Myelomeningocele All pregnancies resulted in live births in a median GA of 36w1d (IQR 32w2d-37w0d). Preterm premature rupture of membranes was observed (PPROM) in five patients.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT04356703

Fetoscopic Open Spina Bifida Repair Using the SAFER ... An adverse event that results in death, is life-threatening, requires inpatient hospitalization or extends a current hospital stay, results in an ongoing or ...

9. karger.com karger.com/fdt/article/52/5/521/927726/Safety-and-Effectiveness-of-Fetal-Myelomeningocele

Safety and Effectiveness of Fetal Myelomeningocele ... Conclusion: Exteriorized uterus fetoscopic repair of MMC proved safe for the mother and effective in closing the neural tube defect. It also ...