Duchenne Muscular Dystrophy
Conditions
Keywords
respiratory function in DMD
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.
Interventions
900 mg idebenone/day
Sponsors
Santhera Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-Label Extension Study
Eligibility
Sex/Gender
MALE
Age
11 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Completion of the SIDEROS study at Visit 8/ Week 78 2. Signed and dated Informed Consent Form for SIDEROS-E
Exclusion criteria
1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8) 2. 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 3. Use of any investigational drug other than the study medication
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events, as per ICH Topic E2A | 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 premature discontinuations of study treatment due to adverse events. | 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 safety laboratory parameters. | 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. | 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 ECG. | From baseline until visit 4 (week 78) | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). | From baseline until visit 4 (week 78) | To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. |
| Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) | From baseline until visit 4 (week 78) | To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. |
| Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p) | From baseline until visit 4 (week 78) | To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. |
Countries
Austria, Belgium, France, Germany, Italy, Spain, Switzerland, United Kingdom, United States
Outcome results
None listed