Effect and Safety of preventive Treatment with ACE-Inhibitor and Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy

Phase 2

• Conditions: I50; Heart failure

• Registration Number: DRKS00000115
• Lead Sponsor: Friedrich- Alexander- Universität Erlangen Nürnberg,Universitätsklinikum , Dekan

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-11-18
• Study Completion: 2015-12-11
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: Male
• Target Recruitment: 42

Inclusion Criteria

1. Male patients aged 10 - 14 years
2. Normal left ventricular function (LVSF >=30%)
3. DMD confirmed by genetic analysis (muscle biopsy facultative)
4. Normal renal function (GFR >30 ml/min/1,73 m2)
5. Informed Consent of both parents, assent of patient

Exclusion Criteria

1. Contraindications for ACE-Inhibitors/Beta-Blocker.
2. Previous therapy with ACE-Inhibitors, AT II Antagonists, Beta-Blockers in the past 3 Months.
3. Participation in another one clinical Trial parallel to this clinical trial
4.Dilated left ventricle ( > 97 percentile) or reduced LVSF < 30%

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate the effect and safety of a preventive treatment with ACE-Inhibitor and a Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy .<br>Time to first diagnosis of left ventricular dysfunction defined as fraction shortening < 28% in standard echocardiography.

Secondary Outcome Measures

Name	Time	Method
Overall survival (time from randomization to death) <br>Quality of life (noted by Kindl-questionnaire) in both groups (baseline; once a year; end of study for individual patient or end of follow-up)<br>Mean drop of frequency and heart rate variability in 24h-ECG <br>Tissue-Doppler data: systolic and diastolic strain, strain rate and wall velocities in left and right ventricle<br>Laboratory parameters (pro-BNP, Angiotensin II, Noradrenalin)<br>assessment of safety: safety -laboratory (esp.Creatinine, Electrolytes,lowest blood pressure measurements, 24h ECG (heart rate, arrhythmias), Adverse Events