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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00758225
• Other study ID #: SN T-II-001-E
• Status: Completed
• Phase: Phase 2
• First received: September 23, 2008
• Last updated: May 30, 2011
• Start date: September 2008
• Est. completion date: January 2011

Study information

• Verified date: May 2011
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belgium: Federal Agency for Medicinal Products and Health Products
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: January 2011
• Est. primary completion date: January 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Idebenone
Patients = 45 kg: 450 mg/day (1 tablet 3 times a day) Patients > 45 kg: 900 mg/day (2 tablets 3 times a day)

Locations

Country	Name	City	State
Belgium	University Hospitals Leuven	Leuven

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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	No