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

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2005-002520-33-BE
• Date of registration: 04/10/2005
• Prospective Registration: No
• Primary sponsor: Santhera Pharmaceuticals LLC
• Public title: 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
• Scientific title: 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
• Date of first enrolment: 06/09/2005
• Target sample size: 21
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-002520-33
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: Single blind: Double blind: yes Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Belgium

Key inclusion & exclusion criteria

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



Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Product Name: idebenone
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To determine whether treatment with idebenone improves or slows decline in cardiac function in patients with cardiac dysfunction associated with DMD

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).

Secondary Objective: To determine whether treatment with idebenone improves or slows decline in muscle strength and/or respiratory function in patients with DMD
To evaluate the safety and tolerability of idebenone in patients with DMD
To determine whether treatment with idebenone changes biochemical markers reflecting cardiac overload or cardiac degeneration

Secondary ID(s)

SNT-II-1