Clinical trial NCT04243889: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Spina Bifida
Myelomeningocele
Myeloschisis
Keywords: Neox Cord 1K
Fetoscopic repair
Cryopreserved human umbilical cord
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
First posted: 2020-01-28
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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 Chromosomal microarray analysis (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 A2 diabetes mellitus (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

Primary outcomes:
measure: Number of patients with successful fetoscopic repair of the defect using NEOX Cord 1K®
timeFrame: Immediately after repair procedure
Secondary outcomes:
measure: Number of patients with intact repair of the defect, defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.
timeFrame: Birth
measure: Number of patients with intact repair of the defect as defined as no cerebrospinal fluid leakage and no dehiscence at the repair site.
timeFrame: 12 months ± 2 months
Other outcomes:
measure: Assessment of Arnold-Chiari malformation II
timeFrame: birth discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
measure: Number of patients with absence of brain stem kinking as assessed by MRI evaluation.
timeFrame: birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
measure: Number of patients with lower extremity motor and sensory levels exceeding the anatomical level of the lesion by ≥ 2 segments.
timeFrame: 2 ± 2 months, 30-36 months and 60-66 months post birth
measure: Number of patients who can ambulate with or without the use of orthotics or devices.
timeFrame: 12 ± 2 months, 30-36 months and 60-66 months post birth
measure: Number of patients with no spinal cord tethering as assessed by a lumbar spine MRI.
timeFrame: birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
measure: Number of patients with absence of syringomyelia as assessed by an MRI.
timeFrame: 12 ± 2 months post birth
measure: Number of patient with the absence of an epidermoid cyst at the repair site, determined by MRI.
timeFrame: birth-discharge or 1 month + 30 days, 12 ± 2 months, and 60-66 months post birth
measure: Number of patients needing de-tethering surgery before the 12 month ± 2 month visit as clinically indicated.
timeFrame: 12 ± 2 months
measure: Developmental motor scales as assessed by the Bayley IV test.
timeFrame: 30-36 months follow up post birth
measure: Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales III
timeFrame: 30-36 months and 60-66 months post delivery
measure: Achievement as measured by the Woodcock-Johnson IV Test of Achievement test
timeFrame: 60-66 months post birth
measure: Assess brain stem function, as measured by the child's swallowing profile
timeFrame: 12 ± 2 months; 30-36 months and 60-66 months post birth
measure: Urodynamic function assessments
timeFrame: 12 ± 2 months, 30-36 months and 60-66 months post birth
measure: Bowel function assessments bowel movements.
timeFrame: 12 ± 2 months, 30-36 months and 60-66 months post birth
measure: Verbal and non-verbal intelligence as measured by the Kaufman Brief Intelligence Test 2 (KBIT-2)
timeFrame: 60-66 months post delivery
measure: Manual dexterity and bimanual coordination as determined by the Purdue Pegboard test
timeFrame: 60-66 months post delivery
measure: Executive function testing as measured by the Behavior Rating Inventory of Executive Function 2 (BRIEF2) test
timeFrame: 60-66 months post delivery
measure: Visual motor integration as assessed by the Beery Visual-Motor Integration test
timeFrame: 60-66 months post delivery
measure: Quality of Life as assessed by the Parkin Spina Bifida Health-Related Quality of Life questionnaire
timeFrame: 60-66 months post delivery
Locations:
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
Study officials:
affiliation: The University of Texas Health Science Center, Houston
name: Ramesha Papanna, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: