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Hypertrophic Myopathy in Children

NCT ID: NCT00267462

Last Updated: 2007-05-07

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

TERMINATED

Total Enrollment

75 participants

Study Classification

OBSERVATIONAL

Study Start Date

1996-01-31

Brief Summary

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The purpose of this study is to identify the patients seen in our practice who are seen with Hypertrophic Myopathy diagnosis in order to better understand the presenting characteristics of their disease, the diagnostic testing to determine the diagnosis, the methods used to follow the disease progression and management practices used in caring for these patients. The objectives of this data review will be an analysis to determine if there is a methodology that will foster improved diagnostic speed and accuracy, and determine the best management practices based on outcomes in these patients.

Detailed Description

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The charts of children followed at Sibley Heart Center of Children's Healthcare of Atlanta will be identified using the Mysis system and screening for the appropriate ICD-9 codes for the types of Hypertrophic Myopathy. These codes include: 425.1, 425.4, 425.7, 425.8, and 425.9. Screening will also be done for known anomalies that are associated with hypertrophic myopathy as seen in some syndromes and metabolic disorders.

Data Collection:

Age at presentation (DOB) History - Prenatal, Family, Current Surgical Procedures Physical Exam results EKG interpretation CXR results Echocardiography results MRI Results Holter monitors results Exercise testing results Radionuclear study results Spiral CT Study results Genetics Testing results Medication usage Enzyme replacement therapy usage Diagnostic laboratory results

Statistics:

A generalized linear model will be performed. Significance will be defined at P≤ 0.05.

Interim monitoring of accumulated data will be performed. One interim analysis will be performed after 3 months of accrual with additional analyses being performed at the end of data collection.

Demographic data will be summarized for all subjects. For each patient summary statistics will include the mean, standard deviation, frequency distribution, minimum, maximum and range.

Conditions

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Congenital Disorders

Study Design

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Observational Model Type

DEFINED_POPULATION

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* seen at Sibley Heart Center, Children's Healthcare of Atlanta
* diagnosed with Hypertrophic Myopathy
Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Children's Healthcare of Atlanta

OTHER

Sponsor Role lead

Principal Investigators

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Kenneth J. Dooley, MD

Role: PRINCIPAL_INVESTIGATOR

Sibley Heart Center Cardiology at Children's Healthcare of Atlanta

Locations

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Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Countries

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United States

Other Identifiers

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05-034

Identifier Type: -

Identifier Source: org_study_id