Natural History of Asphyxiating Thoracic Dystrophy (DTJ)

NCT ID: NCT00948376

Last Updated: 2011-12-13

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

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2007-11-30

Study Completion Date

2011-07-31

Brief Summary

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The aim of the project is to prospectively analyze at a clinical and molecular level a series of 50 asphyxiating thoracic dysplasia (ATD) patients and 20 fetuses to further define the natural history of the disorder and to contribute to the identification of its molecular basis.

Detailed Description

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Asphyxiating thoracic dysplasia (ATD, MIM 208500) belongs to the short rib polydactyly group and is characterized by short ribs often responsible for an early death due to respiratory distress, trident acetabular roof and short long bones.

In the course of the disease, renal, liver and eye complications may occur. However, their frequencies are unknown.

ATD is transmitted with an autosomal mode of inheritance and up till now only one gene has been identified, namely IFT80, which accounts only for a small part of ATD.

The aim of our project is to prospectively analyze at a clinical and molecular level a series of 50 ATD patients and 20 fetuses to further define the natural history of the disorder and to contribute to the identification of its molecular basis.

Conditions

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Asphyxiating Thoracic Dystrophy

Keywords

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Natural history IFT80 short ribs multivisceral involvement

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients

All ages

IFT80 screening

Intervention Type GENETIC

Natural History

Fetuses

IFT80 screening

Intervention Type GENETIC

Natural History

Interventions

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IFT80 screening

Natural History

Intervention Type GENETIC

Other Intervention Names

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Natural History

Eligibility Criteria

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Inclusion Criteria

* Short ribs with narrow thorax
* Trident acetabular roof
* Short hands
* All ages
* Informed consent signed

Exclusion Criteria

* Other disease
* Ellis van creveld syndrome
* No social security
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Valérie Cormier-Daire, PUPH

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique - Hôpitaux de Paris

Locations

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Hopital Necker

Paris, , France

Site Status

Countries

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France

Other Identifiers

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P060223

Identifier Type: -

Identifier Source: org_study_id