In-Utero Surgery Techniques for Spina Bifida

Recruiting in Palo Alto (17 mi)

+2 other locations

Overseen by Ramen Chmait, MD

Age: 18 - 65

Sex: Female

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: University of Southern California

Disqualifiers: Multiple gestation, Diabetes, Obesity, Hypertension, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

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 data supports the effectiveness of this treatment for spina bifida?

Research shows that prenatal myelomeningocele repair, whether through open or endoscopic surgery, can improve neurological outcomes for children. The endoscopic approach may reduce risks for the mother and improve pregnancy outcomes. ^{1 2 3 4 5}

Is in-utero surgery for spina bifida generally safe for humans?

In-utero surgery for spina bifida, especially using the endoscopic approach, is considered feasible and tends to have lower risks for mothers compared to open surgery. However, both open and endoscopic surgeries have similar safety profiles in hospitals, and there are still some maternal and fetal risks involved. ^{1 2 3 4 6}

How does the in utero endoscopic correction of myelomeningocele treatment differ from other treatments for spina bifida?

This treatment is unique because it uses minimally invasive fetoscopic techniques to repair spina bifida before birth, aiming to improve neurological outcomes while reducing risks to the mother compared to traditional open fetal surgery. ^{1 2 4 6 7}

Research Team

Ruben Quintero, MD

Principal Investigator

US Fetus

Ramen Chmait, MD

Principal Investigator

University of Southern California

Eligibility Criteria

This trial is for pregnant women over 18 with a fetus diagnosed with spina bifida (T1-S1 level) and hindbrain herniation, between 19-27 weeks' gestation. Candidates must have normal genetic test results, be able to consent and follow the study plan, and have support systems in place. Exclusions include multiple pregnancies, certain maternal health issues like diabetes or obesity (BMI ≥40), previous early deliveries, positive HIV/Hepatitis-B status or other conditions that risk surgery/anesthesia.

Inclusion Criteria

I am 18 years old or older.

Gestational age of 19 to 27 6/7 weeks' gestation as determined by clinical information and evaluation of first ultrasound

Willing to return for follow-up evaluations at specified intervals

Exclusion Criteria

You are extremely overweight, with a body mass index (BMI) of 40 or higher.

You have placenta previa or placental abruption.

If you have high blood pressure during pregnancy that could lead to complications like preeclampsia or having a baby too early, you may not be able to participate.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgery

Fetoscopic surgical correction of fetal spina bifida using either the percutaneous or laparotomy/uterine exteriorization technique

At time of surgery

Follow-up

Participants are monitored for safety and effectiveness after the surgical procedure

Until delivery

Treatment Details

Interventions

• In Utero Endoscopic Correction of Myelomeningocele IDE - Laparotomy/Uterine Exteriorization Technique (Procedure)
• In Utero Endoscopic Correction of Myelomeningocele IDE - Percutaneous Technique (Procedure)

Trial Overview The trial tests two surgical methods to correct fetal spina bifida before birth: one through small incisions using cameras (percutaneous technique) and another by opening the abdomen to access the uterus directly (laparotomy/uterine exteriorization). The goal is to see which method might be safer or more effective.

Participant Groups

Group I: Percutaneous Technique Experimental Treatment 1 Intervention

The percutaneous technique uses endoscopic scopes through the maternal skin and uterus to perform the surgery. A variation of the percutaneous technique is the mini-laparotomy technique, which uses an endoscopic scope through an incision on the abdomen, smaller than a laparotomy ("mini-laparotomy"), and smaller endoscopic scopes through the maternal skin and uterus to perform the surgery.

Group II: Laparotomy/Uterine Exteriorization Technique Experimental Treatment 1 Intervention

The laparotomy/uterine exteriorization technique consists of performing a laparotomy (incision into the abdominal cavity), exteriorizing the uterus, and using endoscopic scopes through the uterus to perform the correction.

Who Is Running the Clinical Trial?

University of Southern California

Lead Sponsor

Trials

956

Recruited

1,609,000+

Dr. Samir A.

University of Southern California

Chief Executive Officer since 2024

PhD in Molecular Biology from the University of Southern California

Dr. Chung

University of Southern California

Chief Medical Officer since 2016

MD from UC San Diego

USFetus

Collaborator

Trials

2

Recruited

120+

Findings from Research

In-utero surgical repair of fetal myelomeningocele can improve outcomes for affected infants, but it carries significant risks, as demonstrated by a case where the mother experienced complications like pulmonary edema and preterm labor after the procedure.

The infant delivered at 33 weeks after the surgery ultimately died from respiratory distress, highlighting the need for further well-controlled clinical trials to assess the safety and efficacy of this investigational technique.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lethal pulmonary hypoplasia after in-utero myelomeningocele repair. Lu, GC., Steinhauer, J., Ramsey, PS., et al.[2019]

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 378 pregnant patients undergoing fetal myelomeningocele repair, both open and endoscopic approaches showed similar rates of fetal complications, such as intrauterine fetal demise and preterm delivery, indicating comparable safety profiles for both methods.

However, the endoscopic approach was associated with a higher rate of intrauterine infections (4.2%) compared to the open approach (0%), suggesting a potential risk that needs to be considered when choosing the surgical method.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Open and endoscopic fetal myelomeningocele surgeries display similar in-hospital safety profiles in a large, multi-institutional database. Mikulski, MF., Well, A., Beckerman, Z., et al.[2023]

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]

Intrauterine surgery for repairing fetal myelomeningocele has been a practice since 1994, with open repair techniques being used since 1997, indicating a long history of this intervention.

Despite extensive research on various aspects of this procedure, there are still unanswered questions regarding its implications for both mothers and babies that need to be addressed in public discussions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intrauterine surgery in myelomeningocele. Bruner, JP.[2007]

Prenatal repair of myelomeningocele significantly reduced the need for cerebrospinal fluid shunts in infants, with only 40% requiring shunts compared to 82% in the postnatal repair group, indicating a relative risk reduction of 52%.

At 30 months, children who underwent prenatal surgery showed better mental development and motor function scores, although this approach also carried increased risks of preterm delivery and uterine complications for the mother.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized trial of prenatal versus postnatal repair of myelomeningocele. Adzick, NS., Thom, EA., Spong, CY., et al.[2022]

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]

References

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

Lethal pulmonary hypoplasia after in-utero myelomeningocele repair. [2019]

2. Brazil pubmed.ncbi.nlm.nih.gov

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

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

Open and endoscopic fetal myelomeningocele surgeries display similar in-hospital safety profiles in a large, multi-institutional database. [2023]

4. 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]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Intrauterine surgery in myelomeningocele. [2007]

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

A randomized trial of prenatal versus postnatal repair of myelomeningocele. [2022]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Implementation Process and Evolution of a Laparotomy-Assisted 2-Port Fetoscopic Spina Bifida Closure Program. [2021]