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

Santhera Pharmaceuticals1 site in 1 country21 target enrollmentSeptember 2008

ConditionsDuchenne Muscular Dystrophy

InterventionsIdebenone

DrugsIdebenone

Overview

Phase: Phase 2
Intervention: Idebenone
Conditions: Duchenne Muscular Dystrophy
Sponsor: Santhera Pharmaceuticals
Enrollment: 21
Locations: 1
Primary Endpoint: Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
Status: Completed
Last Updated: 14 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

September 2008

End Date

January 2011

Last Updated

14 years ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Santhera Pharmaceuticals

Eligibility Criteria

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

Arms & Interventions

only one arm

Intervention: Idebenone

Outcomes

Primary Outcomes

Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs

Time Frame: Month 0, 3, 6, 12, 18, 24, FU

Secondary Outcomes

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)