Table 1.
Clinical trials in Friedreich's ataxia
| Agent | Therapeutic mechanism | Current level of investigation |
|---|---|---|
| Idebenone | Antioxidant | Phase III randomised placebo-controlled trial |
| CoQ10/Vitamin E | Antioxidant | Phase III randomised two-dose-arm trial |
| Carnitine/Creatine | Antioxidant | Phase II randomised placebo-controlled trial |
| Deferiprone | Iron chelator | Phase II randomised placebo-controlled trial |
| Deferiprone/Idebenone | Iron chelator/antioxidant | Phase II open-label trial |
| Deferiprone/Idebenone/Riboflavin | Iron chelator/antioxidants | Phase II open-label trial |
| EPO | Increase frataxin | Phase II randomised placebo-controlled trial |
| Carbamylated EPO | Increase frataxin | Phase II randomised placebo-controlled trial |
| A0001 | Antioxidant | Phase II placebo-controlled trial |
| Nicotinamide | Increase frataxin | Phase II open-label dose-escalation trial |
| RG2833 | Increase frataxin | Phase I crossover dose-escalation trial |
| Interferon gamma | Increase frataxin | Phase II dose-escalation trial |
| Resveratrol | Antioxidant | Phase II open-label non-randomised trial |