Genetic Analysis of Congenital Diaphragmatic Disorders

NCT ID: NCT01243229

Last Updated: 2023-04-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

305 participants

Study Classification

OBSERVATIONAL

Study Start Date

2010-10-31

Study Completion Date

2021-06-01

Brief Summary

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The purpose of this study is to understand the genetic causes of congenital diaphragmatic disorders (CDD), namely congenital diaphragmatic hernia (CDH), eventration and hiatal hernia.

Specifically, the investigators plan to:

1. Ascertain informative families and sporadic cases with congenital diaphragmatic disorders and obtain appropriate phenotypic data and genetic material (peripheral blood and/or diaphragm tissue sample).
2. Localize the gene(s) for CDD to specific chromosomal segments using linkage analysis, and determine the role of somatic mutations in CDD.
3. Isolate and characterize genes involved in the pathogenesis of CDD.
4. Develop molecular markers that will facilitate accurate diagnosis (including prenatal diagnosis) and permit correlation of phenotypic variation with specific mutations.
5. Compare RNA-sequencing from tissue samples of children without CDH to those children with CDH.

Detailed Description

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Conditions

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Congenital Diaphragmatic Hernia Congenital Diaphragmatic Eventration Congenital Hiatal Hernia Congenital Diaphragmatic Disorders

Study Design

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Observational Model Type

OTHER

Study Time Perspective

OTHER

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with a congenital diaphragmatic disorder

Exclusion Criteria

* none
Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of Utah

OTHER

Sponsor Role lead

Responsible Party

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Luca Brunelli

M.D.

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Luca Brunelli, MD

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

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Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

University Hospital

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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35848

Identifier Type: -

Identifier Source: org_study_id

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