TABLE 1 Clinical studies of idebenone in Friedreich ataxia carried out before the NIH phase I and II trials.
From the following article:
Drug Insight: antioxidant therapy in inherited ataxias
Massimo Pandolfo
BACK TO ARTICLE| Study | Design | n | Idebenone dose (mg/kg/day) | Treatment period | Findings |
|---|---|---|---|---|---|
Abbreviations: CAG, Cooperative Ataxia Group; ICARS, International Cooperative Ataxia Rating Scale. | |||||
| Schulz et al. (2000)56 | Open-label | 8 | 5 | 8 weeks | Urinary 8-hydroxy-2'-deoxyguanosine decreased by  20% (P <0.05) |
| Artuch et al. (2002)57 | Open-label | 9 | 5 | 1 year | Participants aged 11–19 years showed an improved ICARS score after 3, 6 and 12 months |
| Hausse et al. (2002)58 | Open-label | 38 | 5 | 6 months | Left ventricular mass index decreased by 20% in half of the patients (P <0.001); ataxia not quantified |
| Rustin et al. (2002)59 | Open-label | 40 | 5 | 6 months | Left ventricular mass index decreased by 20% in half of the patients (P <0.001) |
| Buyse et al. (2003)60 | Open-label | 8 | 5 | 1 year | Left ventricular mass index decreased by 20% (P <0.0001); increased in ataxia (CAG scale) |
| Mariotti et al. (2003)39 | Double-blind and placebo-controlled | 29 | 5 | 1 year | Left ventricular mass index decreased by 6% vs 11% increase in the placebo group (P <0.01); no neurological differences (ICARS) |