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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: Idebenone


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: only one arm<br> | Drug: Idebenone<br>* Patients ≤ 45 kg: 450 mg/day (1 tablet 3 times a day) Patients > 45 kg: 900 mg/day (2 tablets 3 times a day)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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DMD, Duchenne Muscular Dystrophy, Duchenne, Idebenone