A Phase III double blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of Friedreich's Ataxia patients.
- Conditions
- Friedreich's Ataxia
- Registration Number
- EUCTR2005-004083-22-FR
- Lead Sponsor
- Santhera Pharmaceuticals (Switzerland) Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 204
· Documented diagnosis of FRDA with confirmed FRDA mutations
· Patients 8 years of age or older at baseline
· Patients with body weight = 25kg
· Hypertrophic cardiomyopathy as indicated by:
- maximum LV wall thickness, on echocardiographic or MRI assessment, greater than or equal to 13 mm or greater than the upper normal limit corrected for age and body surface area calculated using the formulae by Henry et al
and
- abnormal ECG
· Patients who in the opinion of the investigator are able to comply with the requirements of the study
· Negative urine pregnancy test at screening and at Visit 1/Day 0/Baseline (women of childbearing potential)
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
· Treatment with Idebenone or Coenzyme Q 10 within the past 3 months
· Pregnancy or breast-feeding
· Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of SGOT, SGPT or creatinine
· Past or present history of abuse of drugs or alcohol
· Any contraindication for MRI
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method