DM1 Heart Registry - DM1 Respiratory Registry (DM1-Heart-R)

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Karim WAHBI, Institut de Myologie, France

ClinicalTrials.gov Identifier:

NCT01136330

First received: June 2, 2010

Last updated: February 17, 2012

Last verified: February 2012

History of Changes

Purpose

Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications.

Our primary objective was to identify cardiac and respiratory prognostic factors in DM1.

Condition
Myotonic Dystrophy


Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Retrospective
Official Title:	Cardiac and Respiratory Prognostic Factors in Patients With Myotonic Dystrophy Type 1

Resource links provided by NLM:

Genetics Home Reference related topics: myotonic dystrophy potassium-aggravated myotonia

Genetic and Rare Diseases Information Center resources: Myotonic Dystrophy Myotonia Atrophica Myotonic Dystrophy Type 1 Muscular Dystrophy

U.S. FDA Resources

Further study details as provided by Institut de Myologie, France:


Enrollment:	914
Study Start Date:	May 2010
Study Completion Date:	September 2011
Primary Completion Date:	September 2011 (Final data collection date for primary outcome measure)

Detailed Description:

Patients with genetically proven DM1 who were admitted in Pitié Salpêtrière Hospital from 2000 and 2010 will be identified. These patients systematically underwent neurological, cardiac and respiratory investigations.
Baseline medical and genetic information will be entered in a dedicated database, including cardiac and respiratory investigations.
The occurence of severe cardiac and respiratory adverse events will also be collected.
Statistical analysis will be performed to look for correlations between baseline patient characteristics and cardiac or respiratory adverse events during follow up.

Eligibility


Ages Eligible for Study:	18 Years and older (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Myotonic dystrophy type 1

Criteria

Inclusion Criteria:

DM1 mutation (>50 CTG repeats)
Age > 18 years

Exclusion Criteria:

Patient refusal

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: NCT01136330

Locations


France
Pitié Salpêtrière Hospital
Paris, Ile de France, France, 75013

Sponsors and Collaborators

Institut de Myologie, France

Investigators


Study Director:	Denis Duboc, MD,PhD	APHP

More Information

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

Wahbi K, Algalarrondo V, Bécane HM, Fressart V, Beldjord C, Azibi K, Lazarus A, Berber N, Radvanyi-Hoffman H, Stojkovic T, Béhin A, Laforêt P, Eymard B, Hatem S, Duboc D. Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1. Arch Cardiovasc Dis. 2013 Dec;106(12):635-43. doi: 10.1016/j.acvd.2013.08.003. Epub 2013 Oct 17.

Wahbi K, Meune C, Porcher R, Bécane HM, Lazarus A, Laforêt P, Stojkovic T, Béhin A, Radvanyi-Hoffmann H, Eymard B, Duboc D. Electrophysiological study with prophylactic pacing and survival in adults with myotonic dystrophy and conduction system disease. JAMA. 2012 Mar 28;307(12):1292-301. doi: 10.1001/jama.2012.346. Erratum in: JAMA. 2012 Jul 25;308(4):342.


Responsible Party:	Karim WAHBI, Karim WAHBI MD,PHD., Institut de Myologie, France
ClinicalTrials.gov Identifier:	NCT01136330 History of Changes
Other Study ID Numbers:	AFM-13286
Study First Received:	June 2, 2010
Last Updated:	February 17, 2012
Health Authority:	France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Institut de Myologie, France:


Myotonic dystrophy Prognosis Sudden death Respiratory failure

Additional relevant MeSH terms:


Myotonic Dystrophy Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Myotonic Disorders	Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 30, 2016

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