DM1 Heart Registry - DM1 Respiratory Registry

Completed

• Conditions: Myotonic Dystrophy

• Registration Number: NCT01136330
• Lead Sponsor: Institut de Myologie, France

Brief Summary

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.

Detailed Description

1. 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.

2. Baseline medical and genetic information will be entered in a dedicated database, including cardiac and respiratory investigations.

3. The occurence of severe cardiac and respiratory adverse events will also be collected.

4. Statistical analysis will be performed to look for correlations between baseline patient characteristics and cardiac or respiratory adverse events during follow up.

Study Timeline

• Study Start: 2010-05
• Study First Submitted: 2010-06-02
• Study First Submitted QC: 2010-06-02
• Study First Posted: 2010-06-03
• Primary Completion: 2011-09
• Study Completion: 2011-09
• Status Verified: 2012-02
• Last Update Submitted: 2012-02-17
• Last Update Posted: 2012-02-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 914

Inclusion Criteria

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

Exclusion Criteria

• Patient refusal

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Trial Locations

Locations (1)

Pitié Salpêtrière Hospital; 🇫🇷 Paris, Ile de France, France