Text Size

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
History of Changes
  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:

U.S. FDA Resources

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
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 May 22, 2013