About this trial
The goal of this study is to identify genes that convey susceptibility to congenital diaphragmatic hernia in humans. The identification of such genes, and examination of their structure and function, will enable a delineation of molecular pathogenesis and, ultimately, prevention or treatment of congenital diaphragmatic hernia. There are many different possible modes of inheritance for congenital anomalies, including autosomal dominant, autosomal recessive, and multifactorial. Multi-factorial inheritance is responsible for many common medical disorders, including hypertension, myocardial infarction, diabetes and cancer. This type of inheritance pattern appears to involve environmental factors as well as a combination of genetic variations that together can predispose to or produce congenital anomalies, such as congenital diaphragmatic hernia.
Our study is designed to establish a small, well-defined genetic resource consisting of 1) Nuclear families suitable for linkage analysis by parametric,non-parametric (e.g. sib pairs, TDT) and association techniques, 2) Individuals with congenital diaphragmatic hernia who can be directly screened for allelic variation in candidate genes, and 3) Individuals who can serve as controls (are unaffected by congenital diaphragmatic hernia). Neonates and their families will be collected from homogenous and heterogeneous populations. By characterizing diverse populations, it should be possible to increase the likelihood of demonstration of genetic variation in selected candidate genes that can then be used in association and linkage studies in individual subjects with congenital diaphragmatic hernia.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
All individuals affected with a congenital diaphragmatic hernia (CDH), or with a family history of a CDH
Disqualifiers
Individuals with no personal history of a CDH or family history of a family member affected with congenital diaphragmatic hernia
Trial population
Children/neonates with an unrepaired congenital diaphragmatic hernia Children/neonates with a reparied congenital diaphragmatic hernia Women who are pregnant with a fetus diagnosed with congenital diaphragmatic hernia Individuals with a family history of congenital diaphragmatic hernia
Trial design
Case-only
Prospective
Treatments tested in this trial
Not listed
Trial groups
Trial outcomes
Primary outcomes
Percentage of patients with a genetic diagnosis
DNA samples from patients will be analyzed for underlying genetic causes.
Secondary outcomes
Developmental outcomes at 2 and 5 years of age
Formal Developmental outcome measures
Percentage of patients with pulmonary hypertension
pulmonary hypertension measured by echocardiogram
Sponsors and contacts
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Columbia University
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator
National Institutes of Health (NIH)
Collaborator