A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.
- Conditions
- Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Duchenne Muscular Dystrophy (DMD)MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2017-004279-30-FR
- Lead Sponsor
- Santhera Pharmaceuticals (Switzerland) Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- 266
Completion of the SIDEROS study at Visit 8/ Week 78.
Signed and dated Informed Consent Form.
Are the trial subjects under 18? yes
Number of subjects for this age range: 266
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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.
Enrolment in SIDEROS-E of siblings of randomized SIDEROS patients is allowed if they meet all the inclusion and none of the exclusion criteria above.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.;Secondary Objective: To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study, classified by background factors including, but not limited to age, DMD history (e.g. time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study.<br><br>;Primary end point(s): Primary:<br>Standard safety assessments, including number of premature discontinuations of study treatment due to adverse events, incidence and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG). <br><br>;Timepoint(s) of evaluation of this end point: Electrocardiogram: baseline<br>Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, 82 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary:<br>Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p). <br>;Timepoint(s) of evaluation of this end point: Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF)Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks<br><br><br><br>