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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 December 2021
• Main ID: NCT03603288
• Date of registration: 31/05/2018
• Prospective Registration: Yes
• Primary sponsor: Santhera Pharmaceuticals
• Public title: Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) SIDEROS-E
• Scientific title: 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
• Date of first enrolment: July 4, 2018
• Target sample size: 161
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03603288
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Austria, Belgium, France, Germany, Italy, Spain, Switzerland, United Kingdom, United States

Key inclusion & exclusion criteria

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



Age minimum: 11 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: idebenone 150 mg film-coated tablets

Primary Outcome(s)

Number of patients with premature discontinuations of study treatment due to adverse events. [Time Frame: From baseline until visit 4 (week 78)]

Number of patients with abnormal safety laboratory parameters. [Time Frame: From baseline until visit 4 (week 78)]

Number of patients with abnormal ECG. [Time Frame: From baseline until visit 4 (week 78)]

Incidence and severity of adverse events, as per ICH Topic E2A [Time Frame: 4 weeks after discontinuation of treatment]

Incidence and severity of adverse events, as per ICH Topic E2A [Time Frame: From baseline until visit 4 (week 78)]

Number of patients with abnormal safety laboratory parameters. [Time Frame: 4 weeks after discontinuation of treatment]

Number of patients with abnormal vital signs. [Time Frame: 4 weeks after discontinuation of treatment]

Number of patients with abnormal vital signs. [Time Frame: From baseline until visit 4 (week 78)]

Secondary Outcome(s)

Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). [Time Frame: From baseline until visit 4 (week 78)]

Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p) [Time Frame: From baseline until visit 4 (week 78)]

Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) [Time Frame: From baseline until visit 4 (week 78)]

Secondary ID(s)

SNT-III-012-E

Source(s) of Monetary Support

Please refer to primary and secondary sponsors