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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00758225
• Date of registration: 23/09/2008
• Prospective Registration: No
• Primary sponsor: Santhera Pharmaceuticals
• Public title: Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
• Scientific 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
• Date of first enrolment: September 2008
• Target sample size: 21
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00758225
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Belgium

Contacts

• Name: Prof Gunnar Buyse, MD PhD
• Affiliation: Universitaire Ziekenhuizen Leuven

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Idebenone

Primary Outcome(s)

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(s)

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]

Secondary ID(s)

SN T-II-001-E

Source(s) of Monetary Support

Please refer to primary and secondary sponsors