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

This study has been completed.
Sponsor:
Information provided by:
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00758225
First received: September 23, 2008
Last updated: May 30, 2011
Last verified: May 2011
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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 ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: No ]

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)

  Eligibility

Genders 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00758225

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

No publications provided

Responsible Party: Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals
ClinicalTrials.gov 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
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products

Keywords provided by Santhera Pharmaceuticals:
Duchenne Muscular Dystrophy
DMD
Duchenne
Idebenone

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 20, 2014