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

Conditions: Duchennes Muscular Dystrophy
MedDRA version: 12.0Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy

Registration Number: EUCTR2007-007752-34-BE

Lead Sponsor: Santhera Pharmaceuticals (Switzerland) Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: Not specified

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

• 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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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:<br>- Nature and frequency of AEs<br>- Laboratory parameters (haematology, biochemistry and urinalysis)<br>- Physical examinations and vital signs<br>- ECGs

Secondary Outcome Measures