A Phase III Open Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study
Phase 3
Completed
- Conditions
- muscular dystrophyNeuromuscular disease10028302
- Registration Number
- NL-OMON48973
- Lead Sponsor
- Santhera Pharmaceuticals (Switzerland),
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 11
Inclusion Criteria
Completion of the SIDEROS study at Visit 8/ Week 78.
Signed and dated Informed Consent Form.
Exclusion Criteria
Patients who discontinued SIDEROS study prematurely (i.e. did not attend all
visits from V1 to V8).
Safety, tolerability or other issues arising during the course of the SIDEROS
study which in the opinion of the Investigator may put the patient at
significant risk or may interfere significantly with the patient's
participation in the SIDEROS-E study.
Use of any investigational drug other than the study medication.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary:<br /><br>* Standard safety assessments, including number of premature discontinuations<br /><br>of study treatment due to adverse events, incidence and severity of adverse<br /><br>events, actual values and changes from baseline in safety laboratory<br /><br>parameters, vital signs and electrocardiogram (ECG).</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary:<br /><br>* Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted<br /><br>(FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced<br /><br>Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p).</p><br>