| Methods |
Randomised, placebo‐controlled, double‐blind, dose‐response pilot trial |
| Participants |
16 participants (9 female, 7 male) with genetically confirmed Friedreich ataxia, aged 18 to 40 years. 11 received recombinant human erythropoietin (rhuEPO) and 5 received placebo |
| Interventions |
Participants received either rhuEPO or placebo over a period of 24 weeks. Dosage was 20,000 IU every 3 weeks for 9 weeks (visits 1 to 3), 40,000 IU every 3 weeks for 9 weeks (visits 4 to 6) and 40,000 IU every 2 weeks for 6 weeks (visits 7 to 9) |
| Outcomes |
Primary outcome measures were of safety and tolerability of rhuEPO and efficacy of rhuEPO in increasing frataxin in peripheral lymphocytes. Secondary outcome measures included the SARA and SF‐36 |
| Notes |
The study was funded by the Italian Agency for Pharmaceutics (Agenzia Italiana del Farmaco; AIFA grant FARM6H95MJ; to F.T.) EPREX, Janssen‐Cilag, Cologno Monzese, Milan, Italy provided RhuEPO |
| Risk of bias |
| Bias |
Authors' judgement |
Support for judgement |
| Random sequence generation (selection bias) |
Unclear risk |
Participants were randomly assigned to rhuEPO or placebo at a ratio of 2:1. The method of randomisation is unclear given the similarity of the genetic and clinical characteristics of the participants in each group |
| Allocation concealment (selection bias) |
Unclear risk |
Method of allocation concealment is not clear |
| Blinding of participants and personnel (performance bias)
All outcomes |
Unclear risk |
Participants reportedly blinded to treatment conditions, however the method used is not described |
| Blinding of outcome assessment (detection bias)
All outcomes |
Unclear risk |
Assessor reportedly blinded to treatment conditions, however the method used is not described |
| Selective reporting (reporting bias) |
Low risk |
All planned outcomes reported |
| Incomplete outcome data (attrition bias)
All outcomes |
Low risk |
All enrolled participants completed the protocol |
| Other bias |
Low risk |
None identified |
