A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients

• Status: Completed
• Trial end date: 2010-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Santhera Pharmaceuticals
• Treatments: Idebenone; Ubiquinone

Criteria

Inclusion Criteria:

• Documented diagnosis of FRDA with confirmed FRDA mutations

• Patients 8 years of age or older at baseline

• Patients with body weight ≥ 25kg

• Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the medication

• Negative urine pregnancy test at screening and at baseline (women of childbearing potential)

Exclusion Criteria:

• Treatment with idebenone or Coenzyme Q10 within the past 1 month

• Pregnancy and/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