The Cardiovascular Genetic and Therapeutic Implications of Muscular Dystrophy

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2007 by Baylor College of Medicine.
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by:
Baylor College of Medicine
ClinicalTrials.gov Identifier:
NCT00518817
First received: August 17, 2007
Last updated: August 20, 2007
Last verified: August 2007
  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

Resource links provided by NLM:


Further study details as provided by Baylor College of Medicine:

Estimated Enrollment: 60
Study Start Date: August 2007
Estimated Study Completion Date: August 2009
Detailed Description:

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
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00518817

Contacts
Contact: Andres Menesses-Diaz, MD 832-826-5600 diegom@bcm.tmc.edu

Locations
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         
Sponsors and Collaborators
Baylor College of Medicine
Investigators
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 September 14, 2014