Duchenne muscular dystrophy-related cardiomyopathy Nervous System Diseases
Conditions
Interventions
Almost all boys with Duchenne muscular dystrophy (DMD) develop progressive cardiomyopathy. The prior multicentre, randomized, placebo-controlled, Heart-Protection Study ['A double-blind randomized, mu
Sponsors
Duchenne UK
Eligibility
Sex/Gender
Male
Inclusion criteria
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
Exclusion criteria: 1. Did not participate in the original DMD Heart Protection study 2. Refusal of re-consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in echocardiogram-measured left ventricular ejection fraction measured using electronic medical records from initial recruitment to study end | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change in left ventricular fractional shortening and left ventricular chamber dimensions measured using electronic medical records from initial recruitment to study end 2. Sub-group analysis may be measured using electronic medical records from initial recruitment to study end. Pre-specified sub-group analyses may include: 2.1. Steroid use versus steroid naive patients 2.2. Actual therapy received since original study exit 2.3. Participant age at onset of detectable cardiomyopathy 2.4 Use of other DMD-modifying therapies (ie: exon skipping; ataluren; adenovirus gene therapy or similar). | — |
Countries
England, United Kingdom
Contacts
Public ContactAlexis Burn
Outcome results
None listed