Efficacy and safety of oral ritlecitinib for the treatment of active nonsegmental vitiligo: A randomized phase 2b clinical trial

• Published in: Journal of the American Academy of Dermatology 2023-02, Vol.88 (2), p.395-403
• Main Authors: Ezzedine, Khaled, Peeva, Elena, Yamaguchi, Yuji, Cox, Lori Ann, Banerjee, Anindita, Han, George, Hamzavi, Iltefat, Ganesan, Anand K., Picardo, Mauro, Thaçi, Diamant, Harris, John E., Bae, Jung Min, Tsukamoto, Katsuhiko, Sinclair, Rodney, Pandya, Amit G., Sloan, Abigail, Yu, Dahong, Gandhi, Kavita, Vincent, Michael S., King, Brett
• Format: Article
• Language: English
• Subjects: Chronic Disease; Dermatology; Double-Blind Method; Human health and pathology; Humans; JAK inhibitor; JAK/STAT signaling; Janus Kinases; Life Sciences; Protein Kinase Inhibitors - adverse effects; randomized clinical trial; ritlecitinib; Skin - pathology; skin depigmentation; TEC inhibitor; Treatment Outcome; VASI; vitiligo; Vitiligo - drug therapy; Vitiligo - pathology
• ISSN: 0190-9622; 1097-6787
• DOI: 10.1016/j.jaad.2022.11.005

Vitiligo is a chronic autoimmune disorder characterized by depigmented patches of the skin. To evaluate the efficacy and safety of ritlecitinib, an oral JAK3 (Janus kinase)/TEC (tyrosine kinase expressed in hepatocelluar carcinoma) inhibitor, in patients with active nonsegmental vitiligo in a phase 2b trial (NCT03715829). Patients were randomized to once-daily oral ritlecitinib ± 4-week loading dose (200/50 mg, 100/50 mg, 30 mg, or 10 mg) or placebo for 24 weeks (dose-ranging period). Patients subsequently received ritlecitinib 200/50 mg daily in a 24-week extension period. The primary efficacy endpoint was percent change from baseline in Facial-Vitiligo Area Scoring Index at week 24. A total of 364 patients were treated in the dose-ranging period. Significant differences from placebo in percent change from baseline in Facial-Vitiligo Area Scoring Index were observed for the ritlecitinib 50 mg groups with (−21.2 vs 2.1; P