The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD. Furthermore, the long-term effect on respiratory, cardiac and motor functions, and skeletal muscle strength/function will be assessed.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
Patients ≤ 45 kg: 450 mg/day (1 tablet 3 times a day) Patients \> 45 kg: 900 mg/day (2 tablets 3 times a day)
University Hospitals Leuven
Leuven, Belgium
Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
Time frame: Month 0, 3, 6, 12, 18, 24, FU
Measures of efficacy of idebenone: - Respiratory Function Testing - Motor Function Measure - Quantitative Muscle Testing - Hand-Held Myometry - Echocardiography and Color Doppler Myocardial Imaging - Cardiac biomarkers
Time frame: Month 0, 6, 12, 18, 24
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