Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Registration Number
- NCT03603288
- Lead Sponsor
- Santhera Pharmaceuticals
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 161
- Completion of the SIDEROS study at Visit 8/ Week 78
- Signed and dated Informed Consent Form for SIDEROS-E
- 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
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description idebenone 150 mg film-coated tablets idebenone 150 mg film-coated tablets 900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
- Primary Outcome Measures
Name Time Method 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 vital signs. 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. From baseline until visit 4 (week 78) 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 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. 4 weeks after discontinuation of treatment To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
- Secondary Outcome Measures
Name Time Method 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.
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.
Trial Locations
- Locations (39)
University of Alabama - Birmingham, Child Health Research
🇺🇸Birmingham, Alabama, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
UC Davis Department of Physical Medicine and Rehabilitation
🇺🇸Sacramento, California, United States
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
University of Iowa, Department of Pediatrics
🇺🇸Iowa City, Iowa, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Children's Hospital Boston, Harvard Medical School, Department of Neurology
🇺🇸Boston, Massachusetts, United States
Gillette Children's Specialty Healthcare
🇺🇸Saint Paul, Minnesota, United States
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
🇺🇸Charlotte, North Carolina, United States
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