Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Santhera Pharmaceuticals
The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
Age
8–17
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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)
You may not be if
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
Clareo Health | Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
