A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: EUCTR2005-002520-33-BE
• Lead Sponsor: Santhera Pharmaceuticals LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2005-10-04
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 21

Inclusion Criteria

•Patients 10 – 16 years of age at time of enrolment
•Male
•Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in LV inferolateral wall
•Confirmed diagnosis of DMD (out of frame dystrophin gene deletion or absent/< 5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD)
•If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease): dosage must be stable (unchanged) 6 months prior to inclusion
•If on chronic medication for DMD associated cardiomyopathy (ß-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion
•Ability to provide reproducible repeat QMT upper limb score within 15% of first assessment score (at Visit 1/Day 1 vs. screening)

Are the trial subjects under 18? yes
Number of subjects for this age range:
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

Exclusion Criteria

•Symptomatic cardiomyopathy or heart failure
•Asymptomatic but severe cardiac dysfunction as Screening Visit (( evaluation): FS < 20% and/or EF < 40%
•Use of ACE inhibitors
•Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening
•Previous (6 months or less) participation in any other therapeutic trial for DMD
•Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months
•History of significant concomitant illness or significant impairment of renal or hepatic function (serum creatinine and GGT greater than 1.5 times upper limit for age and gender)
•Known individual hypersensitivity to idebenone

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine whether treatment with idebenone improves or slows decline in cardiac function in patients with cardiac dysfunction associated with DMD;Secondary Objective: To determine whether treatment with idebenone improves or slows decline in muscle strength and/or respiratory function in patients with DMD<br>To evaluate the safety and tolerability of idebenone in patients with DMD<br>To determine whether treatment with idebenone changes biochemical markers reflecting cardiac overload or cardiac degeneration<br>;Primary end point(s): The relative change from Baseline (at Screening) to Week 52 in peak systolic radial strain of left ventricle (LV) inferolateral wall, assessed by Color Doppler Myocardial imaging (CDMI).