Lynch 2012.
Methods | Double‐blind, placebo‐controlled, randomised trial involving 3 treatment arms (placebo, low‐dose α‐tocopheryl quinone (510 mg/day) or high‐dose α‐tocopheryl quinone (EPI‐A0001) (750 mg/day)) | |
Participants | 31 participants with genetically confirmed Friedreich ataxia aged between 18 and 60 years. 10 participants were randomised into the placebo arm, 11 into the low‐dose α‐tocopheryl quinone arm and 10 into the high‐dose α‐tocopheryl quinone arm | |
Interventions | Participants received capsules containing either 250 mg or 170 mg of α‐tocopheryl quinone in olive oil 3 times daily with meals. The placebo group received identical capsules containing 0.01% beta‐carotene in olive oil. Treatment was provided for 28 days | |
Outcomes | Outcomes were measured at baseline, at 14 days of treatment and at 28 days of treatment. The primary study outcome was a measure of diabetic tendency. Secondary outcome measures included the FARS and SF‐36 | |
Notes | The Friedreich Ataxia Research Alliance and Penwest Pharmaceutical provided funding Authors Lynch and Willi received grant support from Penwest Pharmaceutical to undertake the study. Authors Hawi and Sciascia were employed by Penwest during the study. Authors Miller and Shrader held stock in Edison Pharmaceuticals (owner of EPI‐A0001). Author Miller was an employee and received 100% of his compensation from Edison Pharmaceuticals. Author Shrader was a compensated consultant for Edison Pharmaceuticals. Author Rioux had been previously employed by Edison Pharmaceuticals. Provider of the drug for this study is not stated |
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Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Random sequence generation (selection bias) | High risk | The method of randomisation is not clear and did not completely match the 3 groups. The placebo group had higher clinical severity scores |
Allocation concealment (selection bias) | Unclear risk | The method of allocation concealment is not clear |
Blinding of participants and personnel (performance bias) All outcomes | Low risk | Participants were blinded to the treatment by the use of identical capsules for α‐tocopheryl quinone and placebo |
Blinding of outcome assessment (detection bias) All outcomes | Unclear risk | It is not clear how examiners were blinded to the treatments |
Selective reporting (reporting bias) | Low risk | All planned outcomes were reported |
Incomplete outcome data (attrition bias) All outcomes | Low risk | One participant from the high‐dose α‐tocopheryl quinone arm discontinued treatment due to a major protocol violation |
Other bias | Low risk | None identified |