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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new method for treating spina bifida in unborn babies using a special material called NEOX Cord 1K, a biological membrane. The treatment covers the baby's spine to help prevent problems after birth. Pregnant women carrying a baby with spina bifida, who are between 19 and 25 weeks along, may qualify for this trial. The minimally invasive procedure may offer an alternative to open surgery. As an unphased trial, this study provides a unique opportunity to explore innovative treatment options for 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. However, if you have poorly controlled diabetes or certain infections, you may not be eligible to participate.

What prior data suggests that this fetoscopic procedure is safe for spina bifida repair?

Research has shown that using cryopreserved human umbilical cord patches, such as NEOX Cord 1K®, for spina bifida repair is promising in terms of safety. Studies have found that these patches effectively cover the spinal cord, improving surgical outcomes by creating a watertight seal.

Patients in previous studies demonstrated that these patches can enhance spinal cord protection during surgery, potentially reducing the need for additional treatments after birth. While specific side effects are not detailed, human umbilical cord tissue has generally been well-tolerated in similar procedures.

Overall, evidence suggests that NEOX Cord 1K® is a promising option for spina bifida repair. However, as with any medical treatment, discussing potential risks with a healthcare provider is important. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard of care for spina bifida, which typically involves open fetal surgery, NEOX Cord 1K offers a minimally invasive approach. This treatment is applied fetoscopically, meaning it uses a small incision and special instruments to access the fetus, potentially reducing the risks associated with more invasive surgeries. NEOX Cord 1K acts as a spinal cord cover, aiming to close the developmental defect effectively. Researchers are excited about this treatment because it could lead to fewer complications for both the mother and the fetus, while still providing effective protection and support to the developing spinal cord.

What evidence suggests that NEOX Cord 1K is effective for spina bifida?

Research has shown that NEOX Cord 1K, a patch made from human umbilical cord tissue, holds promise for treating spina bifida. In animal studies, it improved movement and sensation after surgery. In human studies, this patch effectively closed spinal defects and created a tight seal, reducing scarring and aiding healing. In this trial, all eligible fetuses will receive NEOX Cord 1K applied fetoscopically as a spinal cord cover to close the developmental defect. Overall, early results suggest it could be a strong alternative to traditional surgery for spina bifida repair. ^{1 6 7 8 9}

Who Is on the Research Team?

Ramesha Papanna, MD, MPH

Principal Investigator

The University of Texas Health Science Center, Houston

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11900223/

Fetoscopic Myelomeningocele (MMC) Repair: Evolution of ... An animal study with an ovine model showed improved sensorimotor function in sheep repaired with the human umbilical cord patch [52,56]. This is ...

2. med.uth.edu med.uth.edu/neurosciences/pediatric-neuroscience-journal-fall-2023/uthealth-houston-team-completes-feasibility-study-for-minimally-invasive-myelomeningocele-repair-in-25-patients/

UTHealth Houston Team Completes Feasibility Study for ... We concluded that fetoscopic repair using the HUC as a meningeal patch is feasible, with comparable short-term outcomes compared to open in-utero repair.

3. memorialhermann.org memorialhermann.org/services/treatments/spina-bifida-treatment/fetoscopic-spina-bifida-repair

Fetoscopic Spina Bifida Repair Using a Human Umbilical ... NEOX Cord 1K has demonstrated consistently high closure rates in real-world experiences. The study, the first to use a meningeal patch to cover the spina ...

4. eurekalert.org eurekalert.org/news-releases/646107

Researchers assess regenerative patch for spina bifida ... Our preclinical studies have shown the umbilical cord patch to be effective in watertight closure, reducing scarring and regenerating meningeal ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6900178/

An open‐label trial of cryopreserved human umbilical cord in ... In the present study, 60% of wounds achieved complete wound closure in 12.8 ± 4.3 weeks with TTAX01, a result that is compatible with the healing rates observed ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT04243889/fetoscopic-neox-cord-1kr-spina-bifida-repair

Fetoscopic NEOX Cord 1K® Spina Bifida Repair Spina bifida defect between T1 to S1 vertebral levels. Chiari II malformation. No evidence of kyphosis (curved spine). No major life-threatening ...

7. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/uog.27579

Fetal cardiovascular changes during open and fetoscopic in ... Nonetheless, open repair for spina bifida is associated with adverse outcomes, including maternal morbidity, preterm birth and uterine ...

8. withpower.com withpower.com/trial/phase-spinal-dysraphism-8-2023-1a1c6

Umbilical Cord Patch for Spina Bifida (HUC-FICS Trial) The study found that using cryopreserved human umbilical cord (HUC) patches for in utero repair of spina bifida resulted in significantly better spinal cord ...

9. memorialhermann.org memorialhermann.org/-/media/memorial-hermann/org/files/specialties/childrens/fetal-center-journal/childrens-fetal-center-journal-spring-2022.pdf

The Fetal Center “Our primary outcome measure for the fetoscopic patch study is successful surgical closure of the spina bifida defect with a watertight patch that regenerates ...