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

This study has been completed.
Information provided by:
Santhera Pharmaceuticals Identifier:
First received: September 23, 2008
Last updated: May 30, 2011
Last verified: May 2011
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.

Condition Intervention Phase
Duchenne Muscular Dystrophy Drug: Idebenone Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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 ]

Estimated Enrollment: 21
Study Start Date: September 2008
Study Completion Date: January 2011
Primary Completion Date: January 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: only one arm 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)


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00758225

University Hospitals Leuven
Leuven, Belgium, 3000
Sponsors and Collaborators
Santhera Pharmaceuticals
Principal Investigator: Prof Gunnar Buyse, MD PhD Universitaire Ziekenhuizen Leuven
  More Information

Responsible Party: Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals Identifier: NCT00758225     History of Changes
Other Study ID Numbers: SN T-II-001-E
Study First Received: September 23, 2008
Last Updated: May 30, 2011

Keywords provided by Santhera Pharmaceuticals:
Duchenne Muscular Dystrophy

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Growth Substances processed this record on August 16, 2017