The Cardiovascular Genetic and Therapeutic Implications of Muscular Dystrophy
Recruitment status was Not yet recruiting
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Purpose
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 6.5 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.
| Condition |
|---|
|
Muscular Dystrophy Dilated Cardiomyopathy Heart Failure |
| Study Type: | Observational |
| Study Design: | Observational Model: Defined Population Primary Purpose: Screening Time Perspective: Longitudinal Time Perspective: Prospective |
| Estimated Enrollment: | 60 |
| Study Start Date: | August 2007 |
| Estimated Study Completion Date: | August 2009 |
Objective(s) and Hypothesis(es): 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
Eligibility| Ages Eligible for Study: | 1 Month to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- All patients with the diagnosis of muscular dystrophy.
Exclusion Criteria:
- Patients that do not carry the diagnosis of muscular dystrophy.
Contacts and Locations| Contact: Andres Menesses-Diaz, MD | 832-826-5600 | diegom@bcm.tmc.edu |
| United States, Texas | |
| Texas Children's Hospital | Not yet recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Andres Menesses-Diaz, M.D. 832-826-5600 diegom@bcm.tmc.edu | |
| Principal Investigator: John L Jefferies, MD, MPH | |
| Principal Investigator: | John L Jefferies, MD | Baylor College of Medicine |
| Study Director: | Jeffrey A Towbin, MD | Baylor College of Medicine |
More Information
No publications provided
| ClinicalTrials.gov Identifier: | NCT00518817 History of Changes |
| Other Study ID Numbers: | Thrasher |
| Study First Received: | August 17, 2007 |
| Last Updated: | August 20, 2007 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Baylor College of Medicine:
|
Dystrophy Cardiomyopathy |
Additional relevant MeSH terms:
|
Cardiomyopathy, Dilated Heart Failure Muscular Dystrophies Cardiomyopathies Cardiomegaly Heart Diseases Cardiovascular Diseases |
Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 22, 2013