Table 3.

Details of the clinical trial(s) on the use of omaveloxolone for Friedreich’s Ataxia

Clinical trial	Study Design	Study duration/follow-up	Comparator	Sample size	Endpoint	Referencess
MOXIe trial (NCT02255435)	Part 1: Phase 2; double blind; randomized, PBOa-controlled, dose-range, international, multicentric study	12 weeks	PBO	Nd = 69 PBO = 17 Omavb = 52	Safety of various doses of Omav in patients with FA	[10, 22]
	Part 2: Phase 2; double-blind, randomized, PBO controlled, registrational, international, multicentric, parallel-group trial	52 Weeks (48 weeks treatment + 4 weeks follow-up)	PBO	n = 103 PBO = 52 Omav = 51	Safety and efficacy of 150 mg Omav once/day in patients with FA	[22, 23]
	Part 3: Blinded; open-label extension study	196 weeks	Baseline at week 48	n = 82 PBO-Omav = 42 Omav-Omav = 40	Long-term safety and efficacy of Omav patients with FAc following completion of Part 1 or Part 2	[22, 24]

^aPlacebo

^bOmaveloxolone treatment

^cFriedreich’s Ataxia

^dnumber of patients enrolled