Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
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
1 other identifier
interventional
21
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Sep 2008
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2008
CompletedFirst Submitted
Initial submission to the registry
September 23, 2008
CompletedFirst Posted
Study publicly available on registry
September 25, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2011
CompletedJune 1, 2011
May 1, 2011
2.3 years
September 23, 2008
May 30, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (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
Month 0, 3, 6, 12, 18, 24, FU
Secondary Outcomes (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
Month 0, 6, 12, 18, 24
Study Arms (1)
only one arm
EXPERIMENTALInterventions
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 Criteria
You may qualify if:
- Completion of study SNT-II-001
- Body weight ≥ 25 kg
- Eligibility to participate in the present extension study as confirmed by the investigator
You may not qualify if:
- 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
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University Hospitals Leuven
Leuven, 3000, Belgium
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Prof Gunnar Buyse, MD PhD
Universitaire Ziekenhuizen KU Leuven
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
September 23, 2008
First Posted
September 25, 2008
Study Start
September 1, 2008
Primary Completion
January 1, 2011
Study Completion
January 1, 2011
Last Updated
June 1, 2011
Record last verified: 2011-05