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Fetoscopic NEOX Cord 1K® Spina Bifida Repair

Primary Purpose

Spina Bifida, Myelomeningocele, Myeloschisis

Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
NEOX Cord 1K applied fetoscopically
Sponsored by
The University of Texas Health Science Center, Houston
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spina Bifida focused on measuring Neox Cord 1K, Fetoscopic repair, Cryopreserved human umbilical cord

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Maternal inclusion criteria:

  • Singleton pregnancy
  • Gestational age at screening is 19 to 25 5/7 weeks and gestational age at surgery is 22 to 25 6/7 weeks
  • Maternal age: 18 years and older
  • Body mass index < 40 kg/m2
  • No preterm birth risk factors (short cervix, history of previous preterm delivery)
  • No previous uterine incision in the active uterine segment
  • Willing to undergo an open fetal repair if the fetoscopic approach is unsuccessful

Fetal inclusion criteria:

  • Spina bifida defect between T1 to S1 vertebral levels
  • Chiari II malformation
  • No evidence of kyphosis (curved spine)
  • No major life-threatening fetal anomaly unrelated to spina bifida
  • Normal karyotype, or normal CMA, or a CMA with variants of unknown significance

Exclusion Criteria:

Maternal exclusion criteria:

  • Non-resident of the United States
  • Multifetal pregnancy
  • Poorly controlled insulin-dependent pregestational diabetes
  • Poorly controlled A2DM insulin-dependent diabetes
  • Current or planned cerclage or documented history of an incompetent cervix
  • Placenta previa or placental abruption
  • Short cervix of < 20 mm
  • Obesity as defined by a body mass index of > 40 kg/m2
  • Previous spontaneous singleton delivery prior to 37 weeks
  • Maternal-fetal Rh isoimmunization, Kell sensitization, or a history of neonatal alloimmune thrombocytopenia
  • HIV or Hepatitis-B positive status
  • Known Hepatitis-C positivity
  • Uterine anomaly such as large or multiple fibroids or a mullerian duct abnormality that is diagnosed via imaging prior to surgery, which that are unavoidable during surgery
  • Other medical conditions which are contraindication to surgery or general anesthesia
  • Patient does not have a support person
  • Inability to comply with the travel and follow-up requirements of the trial
  • Patient does not meet psychosocial standardized assessment criteria
  • Participation in this or another intervention study that influences maternal and fetal morbidity and mortality
  • Maternal hypertension
  • Zika virus positivity
  • Allergy/history of drug reaction to Amphotericin B

Fetal exclusion criteria:

  • Major fetal anomaly not related to spina bifida
  • Kyphosis in the fetus of 30 degrees or more
  • Ventriculomegaly greater than 15 mm and no movements noted at hip and knee joints

Sites / Locations

  • The University of Texas Health Science Center at HoustonRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NEOX Cord 1K applied fetoscopically

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of patients with successful fetoscopic repair of the defect using NEOX Cord 1K®
A digital image of the fetal repair site will be captured during and immediately after the repair. The images will be sent to three independent blinded neurosurgeons for review of successful closure of the defect. More than best of three votes will be considered as the result.

Secondary Outcome Measures

Number of patients with intact repair of the defect, defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.
Number of patients with intact repair of the defect as defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.

Full Information

First Posted
January 21, 2020
Last Updated
June 14, 2022
Sponsor
The University of Texas Health Science Center, Houston
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1. Study Identification

Unique Protocol Identification Number
NCT04243889
Brief Title
Fetoscopic NEOX Cord 1K® Spina Bifida Repair
Official Title
Fetoscopic Spina Bifida Repair Using a Cryopreserved Human Umbilical Cord Allograft (NEOX Cord 1K®) as a Meningeal Patch
Study Type
Interventional

2. Study Status

Record Verification Date
June 2022
Overall Recruitment Status
Recruiting
Study Start Date
August 28, 2020 (Actual)
Primary Completion Date
March 1, 2026 (Anticipated)
Study Completion Date
February 28, 2031 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The University of Texas Health Science Center, Houston

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spina Bifida, Myelomeningocele, Myeloschisis
Keywords
Neox Cord 1K, Fetoscopic repair, Cryopreserved human umbilical cord

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
NEOX Cord 1K applied fetoscopically
Arm Type
Experimental
Arm Description
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.
Intervention Type
Device
Intervention Name(s)
NEOX Cord 1K applied fetoscopically
Intervention Description
Under general anesthesia and tocolysis, in-utero repair begins with a laparotomy that is followed by exteriorization of the uterus. The fetus is then positioned by external cephalic version. Uterine entry will be accessed using 3 cannulas, followed by heated-humidified carbon dioxide insufflation for visualization. Then, fetoscopically the placode will be examined and dissected. The lesion will be repaired using NEOX Cord 1K® (HUC) for closure of the first layer over the neural placode. Primary closure of the skin will then occur, or NEOX Cord 1K® (HUC) will be used for skin closure at the discretion of the neurosurgeon. Finally, the laparotomy site will be sutured in multiple layers.
Primary Outcome Measure Information:
Title
Number of patients with successful fetoscopic repair of the defect using NEOX Cord 1K®
Description
A digital image of the fetal repair site will be captured during and immediately after the repair. The images will be sent to three independent blinded neurosurgeons for review of successful closure of the defect. More than best of three votes will be considered as the result.
Time Frame
Immediately after repair procedure
Secondary Outcome Measure Information:
Title
Number of patients with intact repair of the defect, defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.
Time Frame
Birth
Title
Number of patients with intact repair of the defect as defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.
Time Frame
12 months ± 2 months
Other Pre-specified Outcome Measures:
Title
Assessment of Arnold-Chiari malformation II
Description
Assessment of Arnold-Chiari malformation II as measured by MRI of head
Time Frame
birth discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
Title
Number of patients with absence of brain stem kinking as assessed by MRI evaluation.
Time Frame
birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
Title
Number of patients with lower extremity motor and sensory levels exceeding the anatomical level of the lesion by ≥ 2 segments.
Time Frame
2 ± 2 months, 30-36 months and 60-66 months post birth
Title
Number of patients who can ambulate with or without the use of orthotics or devices.
Time Frame
12 ± 2 months, 30-36 months and 60-66 months post birth
Title
Number of patients with no spinal cord tethering as assessed by a lumbar spine MRI.
Time Frame
birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
Title
Number of patients with absence of syringomyelia as assessed by an MRI.
Time Frame
12 ± 2 months post birth
Title
Number of patient with the absence of an epidermoid cyst at the repair site, determined by MRI.
Time Frame
birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
Title
Number of patients needing de-tethering surgery before the 12 month ± 2 month visit as clinically indicated.
Time Frame
12 ± 2 months
Title
Developmental motor scales as assessed by the Bayley IV test.
Time Frame
30-36 months follow up post birth
Title
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales III
Time Frame
30-36 months and 60-66 months post delivery
Title
Achievement as measured by the Woodcock-Johnson IV Test of Achievement test
Time Frame
60-66 months post birth
Title
Assess brain stem function, as measured by the child's swallowing profile
Time Frame
12 ± 2 months; 30-36 months and 60-66 months post birth
Title
Urodynamic function assessments
Description
Objective measures of the function of the lower urinary tract by evaluating post-void residual urine
Time Frame
12 ± 2 months, 30-36 months and 60-66 months post birth
Title
Bowel function assessments bowel movements.
Description
Bowel function as assessed by observation of neurogenic bowel incontinence or constipation
Time Frame
12 ± 2 months, 30-36 months and 60-66 months post birth
Title
Verbal and non-verbal intelligence as measured by the Kaufman Brief Intelligence Test 2 (KBIT-2)
Time Frame
60-66 months post delivery
Title
Manual dexterity and bimanual coordination as determined by the Purdue Pegboard test
Time Frame
60-66 months post delivery
Title
Executive function testing as measured by the Behavior Rating Inventory of Executive Function 2 (BRIEF2) test
Time Frame
60-66 months post delivery
Title
Visual motor integration as assessed by the Beery Visual-Motor Integration test
Time Frame
60-66 months post delivery
Title
Quality of Life as assessed by the Parkin Spina Bifida Health-Related Quality of Life questionnaire
Time Frame
60-66 months post delivery

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Maternal inclusion criteria: Singleton pregnancy Gestational age at screening is 19 to 25 5/7 weeks and gestational age at surgery is 22 to 25 6/7 weeks Maternal age: 18 years and older Body mass index < 40 kg/m2 No preterm birth risk factors (short cervix, history of previous preterm delivery) No previous uterine incision in the active uterine segment Willing to undergo an open fetal repair if the fetoscopic approach is unsuccessful Fetal inclusion criteria: Spina bifida defect between T1 to S1 vertebral levels Chiari II malformation No evidence of kyphosis (curved spine) No major life-threatening fetal anomaly unrelated to spina bifida Normal karyotype, or normal CMA, or a CMA with variants of unknown significance Exclusion Criteria: Maternal exclusion criteria: Non-resident of the United States Multifetal pregnancy Poorly controlled insulin-dependent pregestational diabetes Poorly controlled A2DM insulin-dependent diabetes Current or planned cerclage or documented history of an incompetent cervix Placenta previa or placental abruption Short cervix of < 20 mm Obesity as defined by a body mass index of > 40 kg/m2 Previous spontaneous singleton delivery prior to 37 weeks Maternal-fetal Rh isoimmunization, Kell sensitization, or a history of neonatal alloimmune thrombocytopenia HIV or Hepatitis-B positive status Known Hepatitis-C positivity Uterine anomaly such as large or multiple fibroids or a mullerian duct abnormality that is diagnosed via imaging prior to surgery, which that are unavoidable during surgery Other medical conditions which are contraindication to surgery or general anesthesia Patient does not have a support person Inability to comply with the travel and follow-up requirements of the trial Patient does not meet psychosocial standardized assessment criteria Participation in this or another intervention study that influences maternal and fetal morbidity and mortality Maternal hypertension Zika virus positivity Allergy/history of drug reaction to Amphotericin B Fetal exclusion criteria: Major fetal anomaly not related to spina bifida Kyphosis in the fetus of 30 degrees or more Ventriculomegaly greater than 15 mm and no movements noted at hip and knee joints
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ramesha Papanna, MD, MPH
Phone
(713) 500-6423
Email
Ramesha.Papanna@uth.tmc.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Jeannine Garnett, PhD
Phone
(713) 486-6557
Email
Jeannine.Garnett@uth.tmc.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ramesha Papanna, MD, MPH
Organizational Affiliation
The University of Texas Health Science Center, Houston
Official's Role
Principal Investigator
Facility Information:
Facility Name
The University of Texas Health Science Center at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ramesha Papanna, MD, MPH
Phone
713-500-6423
Email
Ramesha.Papanna@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Jeannine Garnett, PhD
Phone
(713) 486-6557
Email
Jeannine.Garnett@uth.tmc.edu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Fetoscopic NEOX Cord 1K® Spina Bifida Repair

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