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Completed

NCT Number: NCT00758225

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

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

University Hospitals Leuven

Leuven, 3000, Belgium

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Idebenone

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)

Primary outcomes

  1. 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

  1. 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

    Time frame: Month 0, 6, 12, 18, 24

Sponsors and collaborators

Lead sponsor

Santhera Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2008
Primary completion
2011
Study completion
2011
First posted
Sep 25, 2008
Registry last updated
Jun 1, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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