6/30/23, 2:21 AM TRuE-V1 Pathway Feedback Search Clinical Topics Home Studies TRuE V1 TRuE V1 Disease Vitiligo Trial question What is the role of ruxolitinib cream in patients with vitiligo? Study design Multi-center Double blinded RCT Population Characteristics of study participants 56.0% female N = 330 44.0% male 330 patients (186 female, 144 male) Inclusion criteria: patients 12 years of age who had non-segmental vitiligo with depigmentation covering 10% of total body-surface area Key exclusion criteria: complete leukotrichia within any facial lesions; any biologic or experimental therapy within 12 weeks; phototherapy within 8 weeks; immunomodulating treatments within 4 weeks, or topical treatments within 1 week Interventions N=221 ruxolitinib (application of ruxolitinib 1.5% cream BID for 24 weeks with treatment extension through week 52) N=109 placebo (vehicle cream applied BID for 24 weeks with treatment extension and use of 1.5% ruxolitinib cream through week 52) https://web.pathway.md/studies/recXFPJkVedhQpMS4 1/2 6/30/23, 2:21 AM TRuE-V1 Pathway Primary outcome Percentage of patients achieving depigmentation of 75% in facial vitiligo area scoring index at week 24 29.8 % 29.8 22.4 % 14.9 % Significant increase 7.5 % 7.4 NNT = 4 0.0 % Ruxolitinib Placebo Significant increase in the percentage of patients achieving depigmentation of 75% in the facial vitiligo area scoring index at week 24 (29.8% vs. 7.4%; RR 4, 95% CI 1.9 to 8.4) Secondary outcomes Significant increase in the percentage of patients achieving depigmentation of 50% in the facial vitiligo area scoring index at week 24 (51.2% vs. 16.9%; RR 3, 95% CI 1.9 to 4.8) Significant increase in the percentage of patients achieving depigmentation of 90% in the facial vitiligo area scoring index at week 24 (15.3% vs. 2.2%; RR 7.3, 95% CI 1.8 to 29.5) Significant increase in the percentage of patients achieving depigmentation of 50% in the total body vitiligo area scoring index at week 24 (20.6% vs. 5.1%; RR 4.1, 95% CI 1.6 to 10.5) Safety outcomes No significant difference in any adverse event. Conclusion In patients 12 years of age who had non-segmental vitiligo with depigmentation covering 10% of total body-surface area, ruxolitinib was superior to placebo with respect to the percentage of patients achieving depigmentation of 75% in the facial vitiligo area scoring index at week 24. Reference David Rosmarin, Thierry Passeron, Amit G Pandya et al. Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo. N Engl J Med. 2022 Oct 20;387(16):1445-1455. Open reference URL https://web.pathway.md/studies/recXFPJkVedhQpMS4 2/2