Fetoscopic Umbilical Cord Repair for Spina Bifida

Overseen By Jeannine Garnett, PhD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: The University of Texas Health Science Center, Houston

Must not be taking: Amphotericin B

Disqualifiers: Diabetes, Hypertension, Obesity, others

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

Compensation: Varies

Number of Visits: 1

Duration: Immediate procedure

50 Participants Needed

Trial Summary

What is the purpose of this trial?

To fetoscopically use cryopreserved human umbilical cord allografts, named NEOX Cord 1K®, as a spinal cord cover of spina bifida defects. This procedure will be performed to create a watertight seal covering over the spinal cord in order to decrease the incidence rates of postnatal morbidities. For larger skin defects, NEOX Cord 1K® may be used as a skin cover.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you have poorly controlled diabetes or certain infections, you may not be eligible to participate.

What data supports the effectiveness of the treatment NEOX Cord 1K for spina bifida?

The study on cryopreserved human umbilical cord (HUC) as a meningeal patch suggests that using HUC can reduce scarring and improve function in spina bifida repair, which may indicate potential benefits of NEOX Cord 1K, as it involves similar materials. ^{1 2 3 4 5}

Is fetoscopic umbilical cord repair for spina bifida safe for humans?

There is no direct safety data available for fetoscopic umbilical cord repair in humans, but related fetal surgery techniques for spina bifida have been studied in animal models, showing that they can be safe and effective, though invasive. ^{5 6 7 8 9}

How is the treatment NEOX Cord 1K for spina bifida different from other treatments?

NEOX Cord 1K is used in a fetoscopic umbilical cord repair for spina bifida, which is a minimally invasive approach that aims to reduce maternal trauma compared to traditional open fetal surgery. This method may also lower the risk of maternal complications and improve postnatal neurological outcomes for the fetus. ^{10 11 12 13 14}

Research Team

Ramesha Papanna, MD, MPH

Principal Investigator

The University of Texas Health Science Center, Houston

Eligibility Criteria

This trial is for pregnant women over 18 with a single pregnancy, carrying fetuses with spina bifida but no other major unrelated anomalies or kyphosis. The fetus must have a normal karyotype and the defect between T1 to S1 vertebral levels. Women must be within a specific gestational age range, have no significant health risks or previous preterm deliveries, and BMI under 40 kg/m2.

Inclusion Criteria

You do not have any factors that could increase the risk of having a baby born too early.

The baby does not have a curved spine.

Maternal: No previous uterine incision in the active uterine segment

Exclusion Criteria

I am HIV or Hepatitis-B positive.

The placenta is in the wrong place or there is bleeding behind the placenta during pregnancy.

If you are pregnant, your cervix is shorter than 20 millimeters.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Fetoscopic application of NEOX Cord 1K® as a spinal cord cover for spina bifida defects

Immediate procedure

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of Arnold-Chiari malformation II, urodynamic function, and bowel function

60-66 months

Treatment Details

Interventions

• NEOX Cord 1K

Trial Overview The trial tests the use of NEOX Cord 1K®, cryopreserved human umbilical cord allografts, applied via fetoscopy as a cover for spina bifida defects in fetuses. It aims to create a watertight seal on the spinal cord to reduce postnatal complications and may also serve as skin cover for larger defects.

Participant Groups

Group I: NEOX Cord 1K applied fetoscopically Experimental Treatment 1 Intervention

Patients intending to undergo open in-utero spina bifida repair, will be offered to be screened for an alternative minimally invasive approach. All eligible pregnant mothers' fetuses within the trial will receive NEOX Cord 1K® as a spinal cord cover to close the developmental defect. In some cases, at the discretion of the Neurosurgeon, NEOX Cord 1K® may be required to cover the skin. All eligible subjects meeting all inclusion criteria but none of the exclusion criteria may be enrolled.

Who Is Running the Clinical Trial?

The University of Texas Health Science Center, Houston

Lead Sponsor

Trials

974

Recruited

361,000+

Findings from Research

Using cryopreserved human umbilical cord (HUC) as a meningeal patch in a modified spina bifida repair model significantly improved spinal cord function compared to conventional repair methods, as shown by higher Texas Spinal Cord Injury Scale scores.

The HUC patch reduced spinal cord tethering and promoted better regeneration of the arachnoid layer, indicating its potential as a safer and more effective option for in utero spina bifida repairs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cryopreserved human umbilical cord as a meningeal patch during in utero spina bifida repair in a modified ovine model. Mann, LK., Won, JH., Garnett, J., et al.[2023]

In a study involving 20 pregnant Lewis dams and their fetuses with induced spina bifida, intra-amniotic injection of amniotic-derived neural stem cells (aNSCs) resulted in successful localization of donor cells in 93.3% of the affected fetuses.

The injected aNSCs were found to cluster in the neural placode and retained an undifferentiated state, suggesting that intra-amniotic delivery could be a viable method for enhancing spinal cord repair in spina bifida.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intra-amniotic delivery of amniotic-derived neural stem cells in a syngeneic model of spina bifida. Turner, CG., Pennington, EC., Gray, FL., et al.[2013]

Induced pluripotent stem cells (iPSCs) and neural stem/progenitor cells (NSPCs) were successfully derived from skin fibroblasts of three newborns with spina bifida aperta (SBa), indicating a potential source for future therapeutic applications.

The generated iPSCs exhibited characteristics of human pluripotent stem cells and were differentiated into NSPCs, which could be expanded for potential use in treating neurological and urological defects associated with SBa.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Generation of Induced Pluripotent Stem Cells and Neural Stem/Progenitor Cells from Newborns with Spina Bifida Aperta. Bamba, Y., Nonaka, M., Sasaki, N., et al.[2022]

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]

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]

References

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

The International Tethered Cord Partnership: Beginnings, process, and status. [2021]

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

Cryopreserved human umbilical cord as a meningeal patch during in utero spina bifida repair in a modified ovine model. [2023]

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

Effect of preoperative low-normal cervical length on perinatal outcome after laparotomy-assisted fetoscopic spina bifida repair. [2023]

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

Fetal surgery for myelomeningocele and the incidence of shunt-dependent hydrocephalus. [2022]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Intra-amniotic delivery of amniotic-derived neural stem cells in a syngeneic model of spina bifida. [2013]

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

Safety and efficacy of fetal surgery techniques to close a spina bifida defect in the fetal lamb model: A systematic review. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Validation of the Fetal Lamb Model of Spina Bifida. [2021]

8. Korea (South) pubmed.ncbi.nlm.nih.gov

Generation of Induced Pluripotent Stem Cells and Neural Stem/Progenitor Cells from Newborns with Spina Bifida Aperta. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Prognostication of the neurological outcome of tethered cord based on intraoperative neuromonitoring findings: how close can we get? [2022]

10. Germany pubmed.ncbi.nlm.nih.gov

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

11. Switzerland pubmed.ncbi.nlm.nih.gov

Fetoscopic Myelomeningocele Repair with Complete Release of the Tethered Spinal Cord Using a Three-Port Technique: Twelve-Month Follow-Up-A Case Report. [2022]

12. 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]

13. 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]

14. England pubmed.ncbi.nlm.nih.gov

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