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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00758225
Recruitment Status : Completed
First Posted : September 25, 2008
Last Update Posted : June 1, 2011
Sponsor:
Information provided by:
Santhera Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Idebenone Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : September 2008
Actual Primary Completion Date : January 2011
Actual Study Completion Date : January 2011

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Arms and Interventions
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Arm Intervention/treatment
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)


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00758225


Locations
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Belgium
University Hospitals Leuven
Leuven, Belgium, 3000
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
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Principal Investigator: Prof Gunnar Buyse, MD PhD Universitaire Ziekenhuizen KU Leuven
More Information
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Responsible Party: Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00758225    
Other Study ID Numbers: SN T-II-001-E
First Posted: September 25, 2008    Key Record Dates
Last Update Posted: June 1, 2011
Last Verified: May 2011
Keywords provided by Santhera Pharmaceuticals:
Duchenne Muscular Dystrophy
DMD
Duchenne
Idebenone
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
 Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs