RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy

NCT ID: NCT00127582

Last Updated: 2005-08-25

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE3
• Total Enrollment: 537 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-01-31

Brief Summary

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

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.

Conditions

Myotonic Dystrophy; Sudden Cardiac Death

Keywords

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Electrophysiological study

Intervention Type: PROCEDURE

pacemaker (PM) implant, internal cardiac defibrillator (ICD) implant, loop-recorder implant

Intervention Type: DEVICE

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Telethon

OTHER

Sponsor Role: collaborator

Catholic University of the Sacred Heart

OTHER

Sponsor Role: lead

Principal Investigators

Fulvio Bellocci, MD

Role: PRINCIPAL_INVESTIGATOR

Catholic University of Sacred Heart

Locations

Catholic University of Sacred Heart

Rome, Rome, Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Fulvio Bellocci, MD

Role: CONTACT

Phone: +390630154187

Email: adellorusso@tin.it

Antonio Dello Russo, MD

Role: CONTACT

Phone: +393393971873

Email: adellorusso@tin.it

Facility Contacts

Fulvio Bellocci, MD

Role: primary

References

Pelargonio G, Dello Russo A, Sanna T, De Martino G, Bellocci F. Myotonic dystrophy and the heart. Heart. 2002 Dec;88(6):665-70. doi: 10.1136/heart.88.6.665. No abstract available.

Reference Type: BACKGROUND

PMID: 12433913 (View on PubMed)

Dello Russo A, Mangiola F, Della Bella P, Nigro G, Melacini P, Bongiorni MG, Tondo C, Calo 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. doi: 10.2459/jcm.0b013e328319bd2c.

Reference Type: DERIVED

PMID: 19708226 (View on PubMed)

Other Identifiers

GUP02067

Identifier Type: -

Identifier Source: org_study_id