Fetoscopic Repair for Spina Bifida

(fMMC Trial)

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

60 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new minimally invasive surgery to fix a spinal defect in unborn babies. The surgery uses tiny tools and cameras inserted through small openings, aiming to reduce risks for both mother and baby. It targets pregnant women whose fetuses have been diagnosed with myelomeningocele (MMC), a severe form of spina bifida. This new treatment technique allows early closure of the spinal defect to prevent potential neurological and psychomotor complications.

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 data supports the effectiveness of the treatment Fetoscopic Meningomyelocele Repair for Spina Bifida?

Research suggests that fetoscopic surgery for spina bifida can improve neurological outcomes for babies and reduce risks for mothers compared to traditional open surgery. This approach aims to enhance the baby's health while minimizing the mother's recovery time and complications. ^{1 2 3 4 5}

Is fetoscopic repair for spina bifida generally safe for humans?

Fetoscopic repair for spina bifida is considered feasible and tends to have lower risks for mothers compared to open surgery. Studies in both animals and humans suggest it leads to fewer complications for mothers, although there are still risks like fetal acidosis (too much acid in the baby's blood) and maternal fluid overload. ^{1 6 7 8 9}

How is the Fetoscopic Meningomyelocele Repair treatment different from other treatments for spina bifida?

Fetoscopic Meningomyelocele Repair is unique because it is a minimally invasive procedure performed inside the womb using small incisions and a camera, which reduces trauma to the mother compared to traditional open fetal surgery. This approach aims to improve the baby's neurological function after birth while minimizing risks to the mother. ^{1 2 4 5 10}

Research Team

Michael A. Belfort, M.D.

Principal Investigator

Baylor College of Medicine

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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 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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Fetoscopic compared with open repair of myelomeningocele: a 2-delivery cost-effectiveness analysis. [2021]

2. Germany pubmed.ncbi.nlm.nih.gov

[Peri- and postoperative management for minimally invasive fetoscopic surgery of spina bifida]. [2014]

3. England pubmed.ncbi.nlm.nih.gov

Maternal anaesthesia in open and fetoscopic surgery of foetal open spinal neural tube defects: A retrospective cohort study. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Fetoscopic Open Neural Tube Defect Repair: Development and Refinement of a Two-Port, Carbon Dioxide Insufflation Technique. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Percutaneous minimally invasive fetoscopic surgery for spina bifida aperta. Part III: neurosurgical intervention in the first postnatal year. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Patient empowerment improves follow-up data collection after fetal surgery for spina bifida: institutional audit. [2023]

7. Netherlands 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. [2021]

8. Brazil pubmed.ncbi.nlm.nih.gov

Fetoscopy for meningomyelocele repair: past, present and future. [2018]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Laparotomy-Assisted 2-Port Fetoscopic Repair of Spina Bifida Aperta: Report of a Single-Center Experience in Paris, France. [2023]

10. England pubmed.ncbi.nlm.nih.gov

Fetoscopic single-layer repair of open spina bifida using a cellulose patch: preliminary clinical experience. [2022]