DM1 Heart Registry - DM1 Respiratory Registry

Myotonic Dystrophy Clinical Trial

— DM1-Heart-R  

Official title:

Cardiac and Respiratory Prognostic Factors in Patients With Myotonic Dystrophy Type 1

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01136330
• Other study ID #: AFM-13286
• Status: Completed
• Phase: N/A
• First received: June 2, 2010
• Last updated: February 17, 2012
• Start date: May 2010
• Est. completion date: September 2011

Study information

• Verified date: February 2012
• Source: Institut de Myologie, France
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 914
• Est. completion date: September 2011
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• DM1 mutation (>50 CTG repeats)

• Age > 18 years

Exclusion Criteria:

• Patient refusal

Study Design

Observational Model: Cohort, Time Perspective: Retrospective

Related Conditions & MeSH terms

• Myotonic Dystrophy

Locations

Country	Name	City	State
France	Pitié Salpêtrière Hospital	Paris	Ile de France

Sponsors (1)

Lead Sponsor	Collaborator
Institut de Myologie, France

Country where clinical trial is conducted

France,