Back to resultsLong-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
Primary Purpose
Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 2
Locations
Belgium
Study Type
Interventional
Intervention
Idebenone
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, DMD, Duchenne, Idebenone
Eligibility 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
Sites / Locations
- University Hospitals Leuven
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
only one arm
Arm Description
Outcomes
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
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
Full Information
NCT ID
NCT00758225
First Posted
September 23, 2008
Last Updated
May 30, 2011
Sponsor
Santhera Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00758225
Brief Title
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
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 Type
Interventional
2. Study Status
Record Verification Date
May 2011
Overall Recruitment Status
Completed
Study Start Date
September 2008 (undefined)
Primary Completion Date
January 2011 (Actual)
Study Completion Date
January 2011 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Santhera Pharmaceuticals
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy, DMD, Duchenne, Idebenone
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
only one arm
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Idebenone
Intervention Description
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 Outcome Measure Information:
Title
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 Measure Information:
Title
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
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Prof Gunnar Buyse, MD PhD
Organizational Affiliation
Universitaire Ziekenhuizen KU Leuven
Official's Role
Principal Investigator
Facility Information:
Facility Name
University Hospitals Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
12. IPD Sharing Statement
Learn more about this trial
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
We'll reach out to this number within 24 hrs