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

Phase 2

Completed

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Idebenone

• Registration Number: NCT00758225
• Lead Sponsor: Santhera Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-09
• Study First Submitted: 2008-09-23
• Study First Submitted QC: 2008-09-23
• Study First Posted: 2008-09-25
• Primary Completion: 2011-01
• Study Completion: 2011-01
• Status Verified: 2011-05
• Last Update Submitted: 2011-05-30
• Last Update Posted: 2011-06-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 21

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
only one arm	Idebenone	-

Primary Outcome Measures

Name	Time	Method
Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs	Month 0, 3, 6, 12, 18, 24, FU

Secondary Outcome Measures

Name	Time	Method
Measures of efficacy of idebenone: - Respiratory Function Testing - Motor Function Measure - Quantitative Muscle Testing - Hand-Held Myometry - Echocardiography and Color Doppler Myocardial Imaging - Cardiac biomarkers	Month 0, 6, 12, 18, 24

Trial Locations

Locations (1)

University Hospitals Leuven; 🇧🇪 Leuven, Belgium