Study within children with Duchenne Muscular Dystrophy
- Conditions
- Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2009-009871-36-DE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- Not specified
Male patients with DMD (confirmed by genetic analysis or muscle biopsy) • aged 10 – 14 years • Normal left ventricular function (fraction shortening = 30%) • Normal renal function (GFR > 30 ml/min/1,73 m2) • Informed Consent of both parents, assent of patient
Are the trial subjects under 18? yes
Number of subjects for this age range: 55
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Dilated left ventricle (size > 97. Percentile) or Left Ventricular Dysfunction (fraction shortening <30%) • contraindications for ACE-Inhibitors/Beta-Blocker • previous therapy with ACE-Inhibitors, AT II Antagonists, Beta-Blockers • Other medical reasons which render participation not reasonable for the patient
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Efficacy of prophylactic anticongestive therapy: time from randomisation to left ventricular dysfunction, defined as left-ventricular shortening fraction <28%(6 monthly control visits);Secondary Objective: - Comparison of safety and tolerability<br>- Comparison of quality of life (Kindl questionnaire12-16) <br>- Comparison of diagnostics: Tissue Doppler vs 2D Echocardiography<br>- Comparison of total survival in both arms <br>- Comparison of pathophysiological changes in neuromuscular regulation (neurohumeral parameters like pro- BNP, Angiotensin II, Noradrenalin)<br>- Comparison of drop of heart frequency rate and heart frequency variability (24h ECG, Tissue Doppler)<br><br>;Primary end point(s): efficacy of prophylactic anticongestive therapy, time from randomisation to left ventricular dysfunction,defined as left-ventricular shortening fraction < 28% (6 monthly control visits);Timepoint(s) of evaluation of this end point: from baseline R4 until study end
- Secondary Outcome Measures
Name Time Method Secondary end point(s): comparison of safety and tolerability<br>comparison of quality of life (Kindl)<br>comparison of diagnostics tissue doppler vs 2 D Echo<br>comparison of pro-BNP, Angiotensin II, Noradrenalin;Timepoint(s) of evaluation of this end point: change of LVSF from baseline