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

NCT ID: NCT00758225

Last Updated: 2011-06-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-09-30
• Study Completion Date: 2011-01-31

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

only one arm

Group Type: EXPERIMENTAL

Idebenone

Intervention Type: DRUG

Patients ≤ 45 kg: 450 mg/day (1 tablet 3 times a day) Patients \> 45 kg: 900 mg/day (2 tablets 3 times a day)

Interventions

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)

Intervention Type: DRUG

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

• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Santhera Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Santhera Pharmaceuticals

Principal Investigators

Prof Gunnar Buyse, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Universitaire Ziekenhuizen KU Leuven

Locations

University Hospitals Leuven

Leuven, , Belgium

Countries

Belgium

Other Identifiers

SN T-II-001-E

Identifier Type: -

Identifier Source: org_study_id