Table 5.

Proportion of patients with decline in FVC% p and FVC by 10% or more at any time point during the study

Patients with decline of 10% or more from Baseline to Week 52	Event type	Idebenone (N = 31)	Placebo (N = 33)	HR (95% CI) p-value
FVC% p: 10% absolute	first event	8 (25.8%)	14 (42.4%)	0.67 (0.28, 1.61) p = 0.367
	persistent	5 (16.1%)	12 (36.4%)	0.50 (0.17, 1.44) p = 0.197
FVC% p: 10% relative	first event	21 (67.7%)	27 (81.8%)	0.46 (0.26, 0.81) p = 0.008
	persistent	18 (58.1%)	25 (75.6%)	0.54 (0.30, 1.00) p = 0.048
FVC [L]: 10% relative	first event	9 (29.0%)	20 (60.6%)	0.35 (0.16, 0.77) p = 0.009
	persistent	7 (22.6%)	16 (48.5%)	0.39 (0.16, 0.95) p = 0.039

Event type: “first event” analysis allows for subsequent improvement; “persistent” defined as a decline that stays below the threshold at all subsequent assessments until the end of the study. Hazard ratio (95% CI) calculated by Cox model.