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A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia
David R Lynch1, Susan L Perlman, Thomas Meier
1Division of Neurology, Children's Hospital of Philadelphia, 502 Abramson Bldg, Philadelphia, PA 19104-4318, USA. lynch@pharm.med.upenn.edu
Insights
Idebenone did not significantly improve neurological function in pediatric patients with Friedreich ataxia over a 6-month period. Further research is needed to explore its therapeutic potential in longer studies.
Area of Science:
- Neurology
- Clinical Trials
- Pharmacology
Background:
- Friedreich ataxia is a rare inherited neurodegenerative disorder.
- Current treatment options for Friedreich ataxia are limited.
- Idebenone is a synthetic compound similar to Coenzyme Q10, investigated for its potential antioxidant properties.
Purpose of the Study:
- To evaluate the efficacy of idebenone in improving neurological function in pediatric patients with Friedreich ataxia.
- To assess the impact of idebenone on the International Cooperative Ataxia Rating Scale (ICARS) and other functional measures.
Main Methods:
- A randomized, double-blind, placebo-controlled trial was conducted.
- Seventy pediatric patients (ages 8-18) with Friedreich ataxia participated.
- Participants received either idebenone (450-2250 mg/day) or a placebo for 24 weeks.
Main Results:
- Patients receiving idebenone showed a mean improvement of 2.5 points on the ICARS score, compared to 1.3 points in the placebo group.
- Idebenone also showed a mean improvement of 1.6 points on the Friedreich Ataxia Rating Scale (FARS), while placebo declined by 0.6 points.
- The observed differences between the idebenone and placebo groups were not statistically significant for either primary or secondary endpoints.
Conclusions:
- Idebenone did not demonstrate a statistically significant effect on neurological function in pediatric patients with Friedreich ataxia within the 6-month study period.
- Larger, longer-duration studies are warranted to fully assess the therapeutic potential of idebenone and other drug candidates for Friedreich ataxia.
- The trial was registered under NCT00537680 at clinicaltrials.gov.
Objective:
To assess the efficacy of idebenone on neurological function in patients with Friedreich ataxia.
Design:
Randomized, double-blind, placebo-controlled intervention trial.
Setting:
Children's Hospital of Philadelphia and the University of California at Los Angeles.
Participants:
Seventy ambulatory pediatric patients (age, 8-18 years) with a baseline International Cooperative Ataxia Rating Scale (ICARS) score of 10 to 54.
Interventions:
Participants were randomized into 1 of 3 treatment arms: 450 or 900 mg of idebenone per day (in those with a body weight < or = or >45 kg, respectively; n = 22); 1350 or 2250 mg of idebenone per day (n = 24); or placebo (n = 24).
Main Outcome Measures:
Mean change from baseline to week 24 in ICARS score was the primary efficacy variable. Mean change in Friedreich Ataxia Rating Scale (FARS) score, performance measures, and activities of daily living were the secondary efficacy variables.
Results:
Patients who received idebenone improved by 2.5 points on mean ICARS score compared with baseline, while patients in the placebo group improved by 1.3 points. Patients who took idebenone also improved by 1.6 points on the FARS, while patients taking placebo declined by 0.6 points. For both end points, the difference between the idebenone and placebo groups was not statistically different.
Conclusions:
Idebenone did not significantly alter neurological function in Friedreich ataxia during the 6-month study. Larger studies of longer duration may be needed to assess the therapeutic potential of drug candidates on neurological function in Friedreich ataxia. Trial Registration clinicaltrials.gov Identifier: NCT00537680.
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