Duchennes Muscular Dystrophy MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Conditions
Sponsors
Santhera Pharmaceuticals (Switzerland) Ltd
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Completion of study SNT-II-001 • Body weight = 25kg • Glucocorticosteroids and ACE-inhibitors are allowed, if on stable dosage within 2 months prior to inclusion • Eligibility to participate in the present extension study as confirmed by the investigator Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Safety or tolerability issues arising during the course of SNT-II-001 which in the opinion of the investigator preclude further treatment with idebenone • Clinically significant abnormalities of haematology or biochemistry • Abuse of drugs or alcohol • Use of coenzyme Q10 or idebenone within 30 days prior to inclusion • Intake of any investigational drug within 30 days prior to inclusion • Symptomatic heart failure • Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Baseline • Known individual hypersensitivity to idebenone or to any of the excipients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To gather long-term data on the safety and tolerability of idebenone in DMD patients;Secondary Objective: To explore the effect of idebenone after longer term administration on respiratory, cardiac and motor functions, and skeletal muscle strength/function;Primary end point(s): • Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs | — |
Countries
Belgium
Outcome results
None listed