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

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00758225

Enrollment

Registered

2008-09-25

Start date

2008-09-30

Completion date

2011-01-31

Last updated

2011-06-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy, DMD, Duchenne, Idebenone

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.

Interventions

DRUGIdebenone

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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

Countries

Belgium

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026