The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
The study is an open-label, single-group, multi-center extension study in patients with DMD receiving glucocorticoid steroids who participated in the SIDEROS study and who meet all the inclusion criteria and none of the exclusion criteria for this extension study. The study consists of 4 study visits scheduled every 6 months (Visit 1/Baseline, Visit 2/Week 26, Visit 3/ Week 52 and Visit 4/ Week 78), and a follow-up visit 4 weeks after treatment discontinuation. Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
161
900 mg idebenone/day
Incidence and severity of adverse events, as per ICH Topic E2A
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Incidence and severity of adverse events, as per ICH Topic E2A
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: 4 weeks after discontinuation of treatment
Number of patients with premature discontinuations of study treatment due to adverse events.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Number of patients with abnormal safety laboratory parameters.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Number of patients with abnormal safety laboratory parameters.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: 4 weeks after discontinuation of treatment
Number of patients with abnormal vital signs.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Number of patients with abnormal vital signs.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: 4 weeks after discontinuation of treatment
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Number of patients with abnormal ECG.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)
Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time frame: From baseline until visit 4 (week 78)