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
Trial Snapshot
- Phase
- Phase 3
- Status
- Terminated
- Sponsor
- Santhera Pharmaceuticals
- Enrollment
- 161
- Locations
- 39
- Primary Endpoint
- Number of patients with premature discontinuations of study treatment due to adverse events.
Study Overview
Brief Summary
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.
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 11 Years to — (Child, Adult, Older Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Completion of the SIDEROS study at Visit 8/ Week 78
- •Signed and dated Informed Consent Form for SIDEROS-E
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
Arms & Interventions
idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
Intervention: idebenone 150 mg film-coated tablets (Drug)
Outcomes
Primary Outcomes
Number of patients with premature discontinuations of study treatment due to adverse events.
Time Frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
Time Frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Incidence and severity of adverse events, as per ICH Topic E2A
Time Frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters.
Time Frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal ECG.
Time Frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Secondary Outcomes
- Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)(From baseline until visit 4 (week 78))
- Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)(From baseline until visit 4 (week 78))
- Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).(From baseline until visit 4 (week 78))