Comprehensive Phenotypic and Genetic Assessment of Tracheal and Esophageal Birth Defects in Patients

Status: Recruiting
Location: See location...
Study Type: Observational
SUMMARY

The investigators propose a preliminary study performing exome sequencing on samples from patients and their biologically related family members with tracheal and esophageal birth defects (TED). The purpose of this study is to determine if patients diagnosed with TED and similar disorders carry distinct mutations that lead to predisposition. The investigators will use advanced, non-invasive magnetic resonance imaging (MRI) techniques to assess tracheal esophageal, lung, and cardiac morphology and function in Neonatal Intensive Care Unit (NICU) patients. MRI techniques is done exclusively if patient is clinically treated at primary study location and if patient has not yet had their initial esophageal repair.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Patient that has been diagnosed by clinical team with a congenital TED OR family member to the TED diagnosed patient.

• Willingness to donate biological specimens.

• Ability to consent/assent as appropriate.

• Infant born between 24 and 42 weeks PMA.

• TED diagnosed by clinical team.

• Inpatient in the Neonatal Intensive Care Unit (NICU) OR family member to the inpatient in the NICU.

• Willingness to donate biological specimens.

• Ability to consent/assent as appropriate.

• Infant born between 24 and 42 weeks PMA.

• TED diagnosed by clinical team.

• Inpatient in the CCHMC (Cincinnati Children's Hospital Medical Center) NICU.

• Clinically stable and adequate temperature control to tolerate MRI as determined by the primary clinical team.

• Infant and biological parents are participating in the NICU TED cohort.

• Ability to consent/assent as appropriate.

• Infant born between 24 and 42 weeks post menstrual age (PMA).

• No tracheal or esophageal defects.

• Inpatient in the CCHMC NICU.

• Clinically stable and adequate temperature control to tolerate MRI as determined by the primary clinical team.

Locations
United States
Ohio
Cincinnati Children's Hospital
RECRUITING
Cincinnati
Contact Information
Primary
Paul Kingma, MD, PhD
paul.kingma@cchmc.org
(513)636-2995
Time Frame
Start Date: 2018-03-28
Estimated Completion Date: 2026-01
Participants
Target number of participants: 360
Treatments
NICU TED Genetic Cohort
This study involves one inpatient biofluid collection encounter from the subject, one biofluid collection encounter from each biological parent, and an optional biofluid collection encounter from other biological family members.
NICU TED MRI Cohort
This study involves up to three inpatient NICU MRI encounters. The first MRI may be done before surgical repair if the clinical team feels the infant is clinically stable. The second MRI may be completed post-surgical repair of TED. An additional 3rd MRI may be done prior to the time of discharge from the NICU. The pre repair, post-surgical, and pre discharge MRIs will provide valuable data for the understanding of tracheal esophageal malformation disorders and may provide clinical guidance for the participant's care.
TED Genetic Cohort
This study involves one biofluid collection encounter from the subject, one biofluid collection encounter from each biological parent, and an optional biofluid collection encounter from other biological family members.
NICU Control MRI Cohort
This study involves two inpatient NICU MRI encounters. The first MRI will occur within the first month of life, and the second MRI will occur prior to discharge.
Sponsors
Leads: Children's Hospital Medical Center, Cincinnati
Collaborators: Columbia University, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

This content was sourced from clinicaltrials.gov