NCT06029192

Brief Summary

Myotonic dystrophy type 1 (DM1) is a neuromuscular disorder in relation with an unstable expansion of CTG repeat. Patients with DM1 are at risk of arrhythmia and conduction disorders. Mortality are mainly related to respiratory failure and sudden death. Patients with DM1 may suffer from obesity, arterial hypertension, diabetes mellitus and sleep apnea. These comorbidities are classically associated with left ventricular diastolic dysfunction (DD) . The investigators aim to assess the prevalence of left ventricular diastolic dysfunction in patients with myotonic dystrophy type 1 , the distribution of DD grading as well as the long-term prognosis of DM1 patients with a left ventricular diastolic dysfunction.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
500

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2023

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 2, 2023

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

July 21, 2023

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 8, 2023

Completed
24 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 2, 2023

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2023

Completed
Last Updated

September 8, 2023

Status Verified

September 1, 2023

Enrollment Period

4 months

First QC Date

July 21, 2023

Last Update Submit

September 7, 2023

Conditions

Diastolic Dysfunction

Outcome Measures

Primary Outcomes (1)

  • mortality

    7 years

Secondary Outcomes (2)

  • Incidence of arrhythmia

    7 years

  • acute heart failure

    7 years

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

patients with myotonic dystrophy type 1 followed in a tertiary reference center.

You may qualify if:

  • patients with genetic proven myotonic dystrophy type 1
  • who experienced a Doppler- Echocardiography including an assessment of the left ventricular diastolic function

You may not qualify if:

  • wall motion basal abnormalities
  • significant valvular disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital Raymond Poincare

Garches, 92380, France

RECRUITING

Study Officials

  • Abdallah FAYSSOIL, MD PhD

    CHU Raymond Poincare

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Abdallah FAYSSOIL, MD PhD

CONTACT

+33147107778abdallah.fayssoil@aphp.fr

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

July 21, 2023

First Posted

September 8, 2023

Study Start

June 2, 2023

Primary Completion

October 2, 2023

Study Completion

November 1, 2023

Last Updated

September 8, 2023

Record last verified: 2023-09

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)