Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Santhera Pharmaceuticals (Switzerland) Limited
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Completion of the SIDEROS study at Visit 8/ Week 78. 2. Signed and dated Informed Consent Form. Inclusion criteria for the optional continued treatment with idebenone beyond Visit 4 of SIDEROS-E study: 1. Completion of Visit 4/ Week 78 of SIDEROS-E study 2. Signed and dated Informed Consent Form for continued treatment Are the trial subjects under 18? yes Number of subjects for this age range: 266 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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. Enrolment in SIDEROS-E of siblings of randomized SIDEROS patients is allowed if they meet all the inclusion and none of the exclusion criteria above. Exclusion criteria for the optional continued treatment with idebenone beyond Visit 4/ Week 78 of SIDEROS-E study: 1. Premature withdrawal from SIDEROS-E study before Visit 4/ Week 78 2. Any conditions, which, in the opinion of the Investigator might trigger a negative risk-benefit assessment for the patient 3. Use of any Investigational drug other than the study medication
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. (up to Visit 4/Week 78);Secondary Objective: To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study, classified by background factors including, but not limited to age, DMD history (e.g. time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study. (up to Visit 4/Week 78);Primary end point(s): Standard safety assessments, including number of premature discontinuations of study treatment due to adverse events, incidence and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG). ;Timepoint(s) of evaluation of this end point: Electrocardiogram: baseline Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, post-completion follow-up visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p). Feasibility of continued idebenone treatment, measured with number of discontinuations of study treatment due to adverse events, will be the only applicable endpoint for the optional continued treatment period (beyond Visit 4).;Timepoint(s) of evaluation of this end point: Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF)Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks.FVC and PEF in optional continued period : 130 weeks, 182 weeks. Adverse events for the optional continued treatment period : 104 weeks, 130 weeks, 156 weeks, 182 weeks | — |
Countries
Austria, Belgium, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States
Contacts
Public ContactQuentin Desvigne
Santhera Pharmaceuticals (Switzerland) Limited
Outcome results
None listed