RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy

Study Description

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Brief Summary:

This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.

Condition or disease	Intervention/treatment	Phase
Myotonic Dystrophy; Sudden Cardiac Death	Procedure: Electrophysiological study; Device: pacemaker (PM) implant, internal cardiac defibrillator (ICD) implant, loop-recorder implant	Phase 3

Detailed Description:

Myotonic dystrophy type 1 (DM1, Steinert disease) is a multisystem disorder that affects, beside muscle, several other organs, including the heart.

Cardiac involvement represents a major problem in the clinical management of patients, so that cardiac complications represent one of the primary causes of premature death in DM1. In particular there is a high incidence of sudden death, ranging from 2 to 30% of cases, so far principally related to the development of conduction blocks. However, literature reports of sudden death in patients implanted with pacemakers, as well as of spontaneous ventricular tachycardia would suggest a potential etiologic role also for ventricular arrhythmias. The lack of clinical research studies conducted on a large number of patients does not make available definite data regarding the etiology and the epidemiology of arrhythmic events in DM1. For the same reasons, other considerable topics, such as prognostic stratification of the arrhythmic risk and clinical management of life-threatening arrhythmias in DM1 patients, are still undefined.

To clarify these issues, the investigators propose a clinical research study performed on a large cohort of DM1 patients enrolled through a multicenter collaboration that also involves 5 cardiological-neurological Italian centres.

Aims of this study are:

• To estimate the incidence of arrhythmias and to characterize the brady-tachyarrhythmic mechanisms underlying the occurrence of cardiac sudden death in DM1;
• To verify by statistical analysis the reliability of data obtained from both non invasive and invasive diagnostic procedures as indexes useful for estimating the arrhythmic risk in DM1;
• To identify more adequate therapeutic guidelines in order to prevent the occurrence of life-threatening arrhythmias.

The protocol of study includes:

1. Clinical-genetic evaluation;
2. Non invasive and invasive diagnostic cardiac procedures;
3. The use of devices for diagnostic and therapeutic follow-up.

Study Design

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Study Type :	Interventional (Clinical Trial)
Enrollment :	537 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Evaluation of Arrhythmic Risk in Myotonic Dystrophy Type I (DM 1)
Study Start Date :	January 2003

Arms and Interventions

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Outcome Measures

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Primary Outcome Measures :

1. Evaluate incidence of: major cardiac events (sudden death
2. resuscitated cardiac arrest
3. ventricular fibrillation
4. sustained ventricular tachycardia
5. sinoatrial and atrioventricular [AV] blocks)

Secondary Outcome Measures :

1. Evaluate with diagnostic non-invasive (standard electrocardiogram [ECG]
2. 24-hour monitoring ECG
3. signal-averaged ECG
4. echocardiography) and invasive procedures (electrophysiology study [EPS] and implantable loop recorders) the risk to develop cardiac arrhythmias in DM patients

Eligibility Criteria

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Ages Eligible for Study:	18 Years to 70 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

• Patient affected by myotonic dystrophy type I (MD1).
• Patient willing to provide a signed informed consent.

Exclusion Criteria:

• Age < 18 years old or >70 years old.
• Ischemic cardiomyopathy
• Cardiomyopathy due to chronic excess of alcohol consumption (>100 g\day)
• Congenital heart disease
• Acquired valvular heart disease
• Metabolic cardiomyopathy: thyrotoxicosis, hypothyroidism, adrenal cortical insufficiency, pheochromocytoma, acromegaly
• Familiar storage and infiltrative diseases (hemochromatosis, glycogen storage, Hurler’s syndrome, Niemann-Pick disease; primary, secondary, familial and hereditary cardiac amyloidoses)
• Systemic diseases (connective tissue disorder; sarcoidosis)
• Peripartum cardiomyopathy

Contacts and Locations

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Contacts

Contact: Fulvio Bellocci, MD	+390630154187	adellorusso@tin.it
Contact: Antonio Dello Russo, MD	+393393971873	adellorusso@tin.it

Locations

Italy
Catholic University of Sacred Heart	Recruiting
Rome, Italy, 00168
Contact: Fulvio Bellocci, MD +390630154187 adellorusso@tin.it
Principal Investigator: Fulvio Bellocci, MD

Sponsors and Collaborators

Catholic University of the Sacred Heart

Fondazione Telethon

Investigators

Principal Investigator:	Fulvio Bellocci, MD	Catholic University of Sacred Heart

More Information

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Publications:

Pelargonio G, Dello Russo A, Sanna T, De Martino G, Bellocci F. Myotonic dystrophy and the heart. Heart. 2002 Dec;88(6):665-70. Review.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Dello Russo A, Mangiola F, Della Bella P, Nigro G, Melacini P, Bongiorni MG, Tondo C, Calò L, Messano L, Pace M, Pelargonio G, Casella M, Sanna T, Silvestri G, Modoni A, Zachara E, Moltrasio M, Morandi L, Nigro G, Politano L, Palladino A, Bellocci F. Risk of arrhythmias in myotonic dystrophy: trial design of the RAMYD study. J Cardiovasc Med (Hagerstown). 2009 Jan;10(1):51-8.

ClinicalTrials.gov Identifier:	NCT00127582 History of Changes
Other Study ID Numbers:	GUP02067
First Posted:	August 8, 2005
Last Update Posted:	August 25, 2005
Last Verified:	May 2005

Keywords provided by Catholic University of the Sacred Heart:

Myotonic dystrophy type 1; Sudden cardiac death; Ventricular tachyarrhythmias; Bradyarrhythmias

Additional relevant MeSH terms:

Myotonic Disorders; Death; Death, Sudden, Cardiac; Myotonic Dystrophy; Pathologic Processes; Heart Arrest; Heart Diseases; Cardiovascular Diseases; Death, Sudden	Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn