Clinical trial NCT00622453: Arrhythmias in Myotonic Muscular Dystrophy
Official title: A Registry of Arrhythmias in Myotonic Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Muscular Dystrophy
Arrhythmia
Sudden Cardiac Death
Lead sponsor: Indiana University
Sponsor class: OTHER
Collaborators: name: Muscular Dystrophy Association
First posted: 2008-02-25
Enrollment: 448
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient Recruitment: Will take place at the 230 hospital-affiliated MDA neuromuscular clinics. Individuals with myotonic dystrophy will be identified in the MDA clinics these will be asked to participate in the study.

Brief summary: Adult myotonic muscular dystrophy (Steinert's disease) is the most common inherited neuromuscular disorder. Cardiac rhythm disturbances occur frequently in this disease state and may be responsible for up to one-third of deaths. In this study, we intend to evaluate the utility of non-invasive electrocardiographic screening methods and history in predicting serious arrhythmic events.

Detailed description: The long term objectives of this population study is a more defined natural history, optimal diagnostic testing methodology, and methods of therapy for arrhythmias in individuals with myotonic muscular dystrophy. The goal is a more adequate definition of appropriate diagnosis and therapy for arrhythmias in order to decrease the likelihood of cardiac morbidity and mortality in this disorder.

The specific aims of the study involve an initial survey of individuals with myotonic muscular dystrophy detailing multiple factors. Non-invasive electrocardiographic testing will be done. Using this initial data and subsequent follow-up data collected yearly the cohort of patients will be followed as to arrhythmia development over a minimum of five years and likely longer with a long-term registry and evaluation of National Death Records and Ancestry.com. This project is unique in that it characterizes a non-neurologic abnormality associated with a neuromuscular disease, myotonic muscular dystrophy.

Eligibility criteria: Inclusion Criteria:

1. Age 18 and over
2. Willing to sign informed consent
3. Have a previous diagnosis of myotonic muscular dystrophy

Exclusion Criteria:

1. Under age 18.
2. Unwilling to sign consent.
3. Unwilling to commit to long-term follow-up.

Primary outcomes:
measure: Evaluate incidence of arrhythmias in myotonic muscular dystrophy
timeFrame: 3 years
Secondary outcomes:
measure: Evaluate with diagnostic non-invasive electrocardiogram (ECG)
timeFrame: 3 Years
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Krannert Institute of Cardiology
state: Indiana
Study officials:
affiliation: Indiana School of Medicine
name: William Groh, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Groh WJ, Groh MR, Saha C, Kincaid JC, Simmons Z, Ciafaloni E, Pourmand R, Otten RF, Bhakta D, Nair GV, Marashdeh MM, Zipes DP, Pascuzzi RM. Electrocardiographic abnormalities and sudden death in myotonic dystrophy type 1. N Engl J Med. 2008 Jun 19;358(25):2688-97. doi: 10.1056/NEJMoa062800.
citation: Bhakta D, Lowe MR, Groh WJ. Prevalence of structural cardiac abnormalities in patients with myotonic dystrophy type I. Am Heart J. 2004 Feb;147(2):224-7. doi: 10.1016/j.ahj.2003.08.008.
citation: Hardin BA, Lowe MR, Bhakta D, Groh WJ. Heart rate variability declines with increasing age and CTG repeat length in patients with myotonic dystrophy type 1. Ann Noninvasive Electrocardiol. 2003 Jul;8(3):227-32. doi: 10.1046/j.1542-474x.2003.08310.x.
citation: Groh WJ, Lowe MR, Zipes DP. Severity of cardiac conduction involvement and arrhythmias in myotonic dystrophy type 1 correlates with age and CTG repeat length. J Cardiovasc Electrophysiol. 2002 May;13(5):444-8. doi: 10.1046/j.1540-8167.2002.00444.x.
citation: Bhakta D, Shen C, Kron J, Epstein AE, Pascuzzi RM, Groh WJ. Pacemaker and implantable cardioverter-defibrillator use in a US myotonic dystrophy type 1 population. J Cardiovasc Electrophysiol. 2011 Dec;22(12):1369-75. doi: 10.1111/j.1540-8167.2011.02200.x. Epub 2011 Oct 28.
citation: Bhakta D, Groh MR, Shen C, Pascuzzi RM, Groh WJ. Increased mortality with left ventricular systolic dysfunction and heart failure in adults with myotonic dystrophy type 1. Am Heart J. 2010 Dec;160(6):1137-41, 1141.e1. doi: 10.1016/j.ahj.2010.07.032.