Table 1.
Compound | Pre-Clinical Studies in FRDA | Clinical Trials in FRDA | ||||
---|---|---|---|---|---|---|
Model | Doses/Treatment | Ref. | Nº Subjects | Doses/Treatment | Ref. | |
Resveratrol | YG8R mouse | 200 mg/kg daily for 3 days. Subcutaneous injection. | [170] | 27 FRDA patients: 13 low dosis and 14 high dosis | 0.5 g or 2.5 g twice daily for 12 weeks. Capsules. | [171] |
Human fibroblast MSCiPSC-derived neurons |
25 µM to 125 µM once 25 µM to 125 µM once 10 µM to 50 µM once |
[172] | 40 patients (estimated) | 2 g daily for 24 weeks. Capsules. | ClinicalTrials.gov Id: NCT03933163 | |
Sulforaphane (SFN) | Mouse NSC34 motor neurons Human fibroblasts |
5 µM for 24 h 10 µM for 24 h |
[180] | |||
Neural stem cells KIKO mouse | 5 µM for 2, 6, and 24 h | [181] | ||||
Human fibroblast | 10 µM for 2, 6, and 24 h | [182] | ||||
Omaveloxolone | Cerebellar Granule Neurons KIKO and YG8R mice Human Fibroblast |
50 nM for 24 h 50 nM for 24 h |
[183] | 103 patients | 150 mg daily for 48 weeks. Capsules. |
[184] |
Melatonin | Case report: 1 FRDA patient | 5 mg and 10 mg | [188] |