Ignite Creation Date:
2024-05-05 @ 6:37 PM
Last Modification Date:
2024-10-26 @ 9:35 AM
Study NCT ID:
NCT00518817
Status:
UNKNOWN
Last Update Posted:
2007-08-21
First Post:
2007-08-17
Brief Title:
The Cardiovascular Genetic and Therapeutic Implications of Muscular Dystrophy
Sponsor:
Baylor College of Medicine
Organization:
Baylor College of Medicine
Study Overview
Official Title:
None
Status:
UNKNOWN
Status Verified Date:
2007-08
Last Known Status:
NOT_YET_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will have significant impact on muscular dystrophy patients as it promotes early screening for heart disease With early identification beneficial medical therapy can be started sooner resulting in restoring and maintaining normal heart function This is critical to the survival of these patients We have reported previously that heart failure in all patients may have common mechanisms the final common pathway Heart failure is a significant health problem with 5 million people in the US carrying the diagnosis and accounting for 12-15 million office visits and 65 million hospital days per year The number of deaths from heart failure continues to increase The data from this study could impact patients worldwide with heart failure by offering new insight into an ever-growing disease population and lead to significant changes in how they are currently treated
Detailed Description:
Objectives and Hypothesises The objectives to be evaluated are as follows
Specific Hypothesis 1 Heart disease specifically dilated cardiomyopathy can be identified early in patients with muscular dystrophy and allow for earlier institution of medical therapies
Specific Hypothesis 2 Non-invasive testing via magnetic resonance imaging MRI and echocardiography can identify early systolic and diastolic dysfunction in patients with muscular dystrophy as well as document structural changes Reverse remodeling following institution of medical therapy
Specific Hypothesis 3 Serologic testing can identify early cardiac dysfunction prior to changes on magnetic resonance imaging or echocardiogram that can predict disease onset risk stratify future cardiac morbidity and mortality and response to medical therapy
Specific Hypothesis 4 Specific dystrophin mutations can be identified that predict the onset or protection against dilated cardiomyopathy
Specific Hypothesis 5 Construction and maintenance of a database of patients with muscular dystrophy can be established that will allow for future research in patients with muscular dystrophy specifically in the area of gene therapy
Specific Hypothesis 6 Quality of life in patients with cardiac disease can be assessed and used to modulate therapy and also allow for noncardiac directed therapies that will improve overall well-being
Specific Hypothesis 7 Further understanding of neurohormonal profiles responses to medical therapy and dystrophin mediated cardiomyopathy will impact all patients with heart failure world-wide
Specific Hypothesis 1 Heart disease specifically dilated cardiomyopathy can be identified early in patients with muscular dystrophy and allow for earlier institution of medical therapies
Specific Hypothesis 2 Non-invasive testing via magnetic resonance imaging MRI and echocardiography can identify early systolic and diastolic dysfunction in patients with muscular dystrophy as well as document structural changes Reverse remodeling following institution of medical therapy
Specific Hypothesis 3 Serologic testing can identify early cardiac dysfunction prior to changes on magnetic resonance imaging or echocardiogram that can predict disease onset risk stratify future cardiac morbidity and mortality and response to medical therapy
Specific Hypothesis 4 Specific dystrophin mutations can be identified that predict the onset or protection against dilated cardiomyopathy
Specific Hypothesis 5 Construction and maintenance of a database of patients with muscular dystrophy can be established that will allow for future research in patients with muscular dystrophy specifically in the area of gene therapy
Specific Hypothesis 6 Quality of life in patients with cardiac disease can be assessed and used to modulate therapy and also allow for noncardiac directed therapies that will improve overall well-being
Specific Hypothesis 7 Further understanding of neurohormonal profiles responses to medical therapy and dystrophin mediated cardiomyopathy will impact all patients with heart failure world-wide
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None