Congenital DisordersNCT00267462
Hypertrophic Myopathy in Children
An observational study of Congenital Disorders, run by Children's Healthcare of Atlanta at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Congenital Disorders
- How many people
- 75 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 1996to not given
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- seen at Sibley Heart Center, Children's Healthcare of Atlanta
- diagnosed with Hypertrophic Myopathy
Who cannot join
- those who do not meet inclusion criteria
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Retrospective Review of Hypertrophic Myopathy in Children
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.
Read the detailed description
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
- Congenital Disorders
- Keywords
- pediatric, cardiac, hypertrophic Myopathy
- Medical subject headings
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2005 (estimated)
- Study startJan 1996
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Defined population
- Time perspective
- Retrospective
- Enrolment
- 75 participants
Sponsor and investigators
- Lead sponsor
- Children's Healthcare of Atlanta
- Sponsor type
- University, hospital or other
- Investigators
- Kenneth J. Dooley, MD, Principal investigator, Sibley Heart Center Cardiology at Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00267462
- Sponsor's protocol ID
- 05-034
- Status last verified
- May 2007
- First submitted
- 19 Dec 2005
- Last updated
- 7 May 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Review of Hypertrophic Myopathy in Children
- ClinicalTrials.gov ID
- NCT00267462
- Acronym / sponsor ID
- 05-034
- Conditions
- Congenital Disorders
- Keywords
- pediatric, cardiac, hypertrophic Myopathy
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 75 participants
- Lead sponsor
- Children's Healthcare of AtlantaUniversity, hospital or other
- Study start
- Jan 1996
- First posted
- 21 Dec 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Observational model
- Defined population
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- seen at Sibley Heart Center, Children's Healthcare of Atlanta
- diagnosed with Hypertrophic Myopathy
Key exclusion criteria (1 listed)
- those who do not meet inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2007.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth J. Dooley, MD principal investigator
Sibley Heart Center Cardiology at Children's Healthcare of Atlanta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Disorders, run by Children's Healthcare of Atlanta at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 May 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2007.