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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for spina bifida, a condition where a baby's spinal cord doesn't develop properly. Researchers use a patch made from cryopreserved human umbilical cord (known as the NEOX Cord 1K or HUC patch) to cover the spinal cord defect in the womb, aiming for less invasive surgery. Pregnant women carrying a single baby diagnosed with spina bifida between certain vertebral levels, without other complicating factors, may be eligible to participate. This approach aims to provide a safer alternative to traditional open fetal repairs. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could transform spina bifida treatment.

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 surgical approach using the umbilical cord patch is safe for spina bifida repair?

Research has shown that using frozen human umbilical cords as a patch is generally safe. In a study with 32 participants who had difficult-to-heal wounds, the treatment was well-tolerated, and most participants did not experience serious side effects. This finding suggests that the umbilical cord patch could be a promising option for repairing spina bifida. Additionally, this type of treatment has succeeded in other procedures. Overall, the evidence supports the safety of using frozen human umbilical cords in treatments like those being tested. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard of care, which typically involves open fetal surgery for spina bifida, the treatment using the NEOX Cord 1K® patch is applied fetoscopically and uses cryopreserved human umbilical cord tissue. This minimally invasive approach reduces the risks associated with open surgery for both the mother and fetus. Researchers are excited because this method aims to effectively cover and protect the spinal cord defect, potentially improving outcomes with less trauma and faster recovery times for both mom and baby.

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

Research has shown that a specially preserved human umbilical cord patch, which participants in this trial will receive, can improve spina bifida surgery outcomes. Animal studies found that this patch increases survival rates and enhances nerve function. This trial will evaluate the patch applied fetoscopically, a less invasive surgery method using small cuts to repair spina bifida. This method has proven practical and offers similar short-term results to traditional open surgery. The patch acts as a protective cover for the spinal cord defect, potentially aiding in better healing and function. Overall, early findings suggest that this treatment could enhance the quality of life for individuals with spina bifida. ^{2 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 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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

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 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+

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5017885/

Neurological Outcomes after Human Umbilical Cord Patch for ... Conclusions Cryopreserved HUC patch appears to improve survival and neurological outcome in this severe form of the ovine model of spina bifida. Keywords: ...

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

Fetoscopic Spina Bifida Repair Using a Human Umbilical ... A study to determine the feasibility of fetoscopic surgery to repair spina bifida and facilitate vaginal delivery.

3. thejns.org thejns.org/view/journals/j-neurosurg/139/4/article-p1169.xml

Cryopreserved human umbilical cord as a meningeal patch ... Despite in utero spina bifida (SB) repair, more than two-thirds of patients with SB are unable to ambulate independently, and 1 in 4 children ...

4. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/full/10.1002/uog.15790

Cryopreserved human umbilical cord patch for in‐utero spina ... Cryopreserved umbilical cord graft is a promising regenerative patch for intrauterine repair of spina bifida.

5. 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.

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

An open‐label trial of cryopreserved human umbilical cord in ... An open‐label, multicenter trial of cryopreserved human umbilical cord (TTAX01) was undertaken in 32 subjects presenting with such complex wounds.

7. uth.edu uth.edu/fetal-institute/outcomes

Outcomes | UTHealth Houston Fetal Institute Read about UTHealth Houston Fetal Institute outcomes related to Fetoscopic Myelomeningocele Repair and the human umbilical cord (HUC) patch.

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

Umbilical Cord Patch for Spina Bifida (HUC-FICS Trial) Research in animal models suggests that using a cryopreserved human umbilical cord (HUC) patch for spina bifida repair may improve neurological outcomes and ...

9. utphysicians.com utphysicians.com/fetal-intervention-for-spina-bifida/

Fetal intervention for spina bifida The UTHealth Houston Fetal Center has a track record of outstanding outcomes in minimally invasive fetoscopic procedures for spina bifida.