Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

• Status: Completed
• Trial end date: 2009-04-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.

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

Details

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

Criteria

Inclusion criteria:

• Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations

• Patients 8 - 17 years of age at baseline

• Patients with a body weight greater than 25 kg/55 lbs at baseline

• Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline

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

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

Exclusion criteria:

• Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month

• Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening

• Pregnancy and/or breast-feeding

• Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine

• History of abuse of drugs or alcohol

• Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH)

• Participation in a trial of another investigational drug within the last 3 months