Umbilical Cord Patch for Spina Bifida

(HUC-FICS Trial)

Overseen By Stephanie W Conaway, BSN

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: The University of Texas Health Science Center, Houston

Disqualifiers: Diabetes, Hypertension, Obesity, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1 week

100 Participants Needed

Trial Summary

What is the purpose of this trial?

The objective is to test the efficacy of a laparotomy-assisted fetoscopic surgical approach to cover spina bifida spinal cord developmental defects using cryopreserved human umbilical cords (NEOX Cord 1K®) as a meningeal and skin patch.

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 for spina bifida?

Research in animal models suggests that using a cryopreserved human umbilical cord (HUC) patch for spina bifida repair may improve neurological outcomes and reduce inflammation and scarring compared to other materials. This could potentially lead to better spinal cord function and fewer complications like tethered cord syndrome. ^{1 2 3 4 5}

Is the umbilical cord patch safe for use in humans?

The studies primarily focus on animal models, but they suggest that the cryopreserved human umbilical cord patch has regenerative, anti-inflammatory, and anti-scarring properties, which are generally positive indicators for safety. ^{1 2 3 4 5}

How is the umbilical cord patch treatment for spina bifida different from other treatments?

The umbilical cord patch treatment for spina bifida is unique because it uses a cryopreserved human umbilical cord, which has regenerative, anti-inflammatory, and anti-scarring properties, providing a watertight repair and potentially improving neurological outcomes compared to other materials like acellular dermal matrix. ^{1 2 3 4 6}

Research Team

Ramesha Papanna, MD, MPH

Principal Investigator

The University of Texas Health Science Center, Houston

Eligibility Criteria

This trial is for pregnant women with a single fetus diagnosed with spina bifida, between 19 and 25+ weeks gestation. Women must be over 18, have a BMI ≤45 kg/m2 without preterm birth risks or previous uterine incisions in the active segment. The fetus should have the defect between T1 to S1 vertebral levels without severe kyphosis or other life-threatening anomalies.

Inclusion Criteria

Fetal Inclusion Criteria: No evidence of kyphosis (curved spine)

Fetal Inclusion Criteria: No major life-threatening fetal anomaly unrelated to spina bifida

Maternal Inclusion Criteria: No preterm birth risk factors (short cervix <20 mm or a history of previous preterm delivery)

Exclusion Criteria

Maternal Exclusion Criteria: Maternal hypertension

Maternal Exclusion Criteria: Patient does not have a support person

Maternal Exclusion Criteria: Multifetal pregnancy

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo a laparotomy-assisted fetoscopic surgical approach using cryopreserved human umbilical cords as a meningeal and skin patch for spina bifida repair

1 week

Follow-up

Participants are monitored for safety and effectiveness after the surgical procedure

4 weeks

Treatment Details

Interventions

• Cryopreserved Human Umbilical Cord

Trial Overview The study tests using cryopreserved human umbilical cords (NEOX Cord 1K®) as patches during fetoscopic surgery to repair spinal defects caused by spina bifida. It aims to see if this method is effective when applied via laparotomy-assisted fetoscopy.

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 Pediatric 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.

Cryopreserved Human Umbilical Cord is already approved in United States for the following indications:

Approved in United States as NEOX Cord 1K for:

• Spina bifida repair under Investigational Device Exemption

Who Is Running the Clinical Trial?

The University of Texas Health Science Center, Houston

Lead Sponsor

Trials

974

Recruited

361,000+

Findings from Research

The study found that using cryopreserved human umbilical cord (HUC) patches for in utero repair of spina bifida resulted in significantly better spinal cord function in lambs compared to acellular dermal matrix (ADM) patches, as measured by the Texas Spinal Cord Injury Scale.

Histological analysis showed that the HUC group had lower levels of inflammation and fewer activated astrocytes compared to the ADM group, indicating that HUC patches may provide a more effective and safer option for reducing inflammation and preserving neurological function during spina bifida repair.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Allografts for Skin Closure during In Utero Spina Bifida Repair in a Sheep Model. Mann, LK., Won, JH., Patel, R., et al.[2021]

In a study involving pregnant ewes, using a cryopreserved human umbilical cord patch to repair surgically created spina bifida significantly improved neurological outcomes compared to unrepaired cases, as indicated by higher Texas Spinal Cord Injury Scale scores and partial bladder control.

Lambs that received the HUC patch showed complete healing of skin defects and better overall survival rates, suggesting that this intervention could be a promising approach for treating spina bifida in utero.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neurological Outcomes after Human Umbilical Cord Patch for In Utero Spina Bifida Repair in a Sheep Model. Papanna, R., Mann, LK., Snowise, S., et al.[2022]

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 comparing cryopreserved human umbilical cord (HUC) patches to acellular dermal matrix (ADM) patches for repairing in utero spina bifida defects in rats, the HUC patches resulted in lower inflammation and apoptosis, indicating a safer and more effective option for repair.

The HUC patches also promoted better cellular organization and growth, suggesting they could improve spinal cord function and reduce complications like tethering compared to ADM patches, making them a promising material for future spinal surgeries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cryopreserved human umbilical cord versus acellular dermal matrix patches for in utero fetal spina bifida repair in a pregnant rat model. Mann, LK., Won, JH., Trenton, NJ., et al.[2020]

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]

This case series involved 4 patients who underwent in utero repair of spinal dysraphisms using a novel patch made from cryopreserved human umbilical cord, providing new insights into this treatment approach.

Imaging results from prenatal and postnatal MRI and ultrasound revealed issues such as cord tethering and syrinx progression in all cases, highlighting potential complications associated with this novel patch material.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prenatal and Postnatal Imaging Findings After Fetal Repair of Spinal Dysraphisms Using Cryopreserved Human Umbilical Cord Patch: A Case Series of 4 Patients. Calle, S., Papanna, R., Hughes, K., et al.[2021]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Allografts for Skin Closure during In Utero Spina Bifida Repair in a Sheep Model. [2021]

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

Neurological Outcomes after Human Umbilical Cord Patch for In Utero Spina Bifida Repair in a Sheep Model. [2022]

3. 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]

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

Cryopreserved human umbilical cord versus acellular dermal matrix patches for in utero fetal spina bifida repair in a pregnant rat model. [2020]

5. 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]

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

Prenatal and Postnatal Imaging Findings After Fetal Repair of Spinal Dysraphisms Using Cryopreserved Human Umbilical Cord Patch: A Case Series of 4 Patients. [2021]