Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001

The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD. Furthermore, the long-term effect on respiratory, cardiac and motor functions, and skeletal muscle strength/function will be assessed.

Inclusion Criteria:

• Completion of study SNT-II-001
• Body weight ≥ 25 kg
• 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

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