Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: idebenone 150 mg film-coated tablets


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 11 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-Label Extension Study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: idebenone 150 mg film-coated tablets<br>900 mg idebenone/day (2 tablets to be taken 3 times a day with meal) | Drug: idebenone 150 mg film-coated tablets<br>* 900 mg idebenone/day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 4 weeks after discontinuation of treatment | 
| Number of patients with abnormal ECG. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | From baseline until visit 4 (week 78) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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respiratory function in DMD