ong-term follow-up of heart function in participants of the Duchenne Muscular Dystrophy Heart Protection study

Not Applicable

Completed

• Conditions: Duchenne muscular dystrophy-related cardiomyopathy; Nervous System Diseases

• Registration Number: ISRCTN43827539
• Lead Sponsor: Duchenne UK

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2023-07-30
• Study Start: 2023-09-11
• Last Update Posted: 16 October 2023

Recruitment & Eligibility

• Status: Completed
• Sex: Male
• Target Recruitment: 85

Inclusion Criteria

1. Participated in the original DMD Heart Protection study (https://www.isrctn.com/ISRCTN50395346)
2. Valid consent of boys/parent or carer (age dependent) to allow access to serial measures of heart function and limited other data wherever undertaken, from the time each participant exited the original study

Exclusion Criteria

1. Did not participate in the original DMD Heart Protection study
2. Refusal of re-consent

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in echocardiogram-measured left ventricular ejection fraction measured using electronic medical records from initial recruitment to study end

Secondary Outcome Measures

Name	Time	Method
1. Change in left ventricular fractional shortening and left ventricular chamber dimensions measured using electronic medical records from initial recruitment to study end<br>2. Sub-group analysis may be measured using electronic medical records from initial recruitment to study end. Pre-specified sub-group analyses may include:<br>2.1. Steroid use versus steroid naive patients<br>2.2. Actual therapy received since original study exit<br>2.3. Participant age at onset of detectable cardiomyopathy<br>2.4 Use of other DMD-modifying therapies (ie: exon skipping; ataluren; adenovirus gene therapy or similar).