A Prospective Multicenter Randomized Placebo-Controlled Study of Upadacitinib in Adults with Erosive Mucosal Lichen Planus and Lichen Planopilaris

The primary objective is to evaluate the clinical efficacy of upadacitinib 30 mg daily compared to placebo in adult patients diagnosed with erosive mucosal lichen planus or lichen planopilaris after 16 weeks of treatment. The secondary objectives include:

• Assessment of efficacy at 32 weeks.
• Evaluation of quality of life at 16 and 32 weeks.
• Description of the patient global impression of severity and patient global impression of change at 16 and 32 weeks.
• Assessment of safety and tolerability.
• Evaluation of efficacy in mucosal lichen planus using the Reticular Erythematous Ulcerative score, Oral Lichen Planus Symptoms Severity Measure, and numeric rating scale for pain at 16 and 32 weeks.
• Evaluation of efficacy in lichen planopilaris using the LPP Activity Index and SCALPDEX Questionnaire at 16 and 32 weeks.

The sponsor did not provide the total number of participants. The study population consists of male and female patients between the ages of 18 and 65 years. Participants must have biopsy-confirmed mucosal lichen planus or active lichen planopilaris. Eligible individuals must demonstrate a moderate to severe disease state, characterized by an Investigator Global Assessment score of 3 or higher, and have shown an inadequate response to high- or ultra-high potency topical corticosteroids. Female subjects of childbearing potential are required to provide negative pregnancy tests.

This is a prospective, multicenter, randomized, placebo-controlled, phase 4 study designed to evaluate the clinical efficacy of upadacitinib 30 mg daily in adult patients with erosive mucosal lichen planus or lichen planopilaris. The research methodology involves comparing the test product, RINVOQ 30 mg prolonged-release tablets, against a placebo. The study includes a screening visit to confirm eligibility through biopsy and laboratory assessments, including pregnancy testing for females of childbearing potential. Eligible participants must have an Investigator Global Assessment (IGA) score of ≥ 3 and have shown an inadequate response to high-potency topical corticosteroids. Following baseline, the treatment period lasts for 16 weeks to assess the primary endpoint of IGA response (absolute IGA score ≤2). Subsequent follow-up visits occur at week 32 to evaluate secondary endpoints, including improvements in the Dermatology Life Quality Index (DLQI), Patient Global Impression of Severity (PGIS), and Patient Global Impression of Change (PGIC), as well as changes in various clinical scores and laboratory values. The total duration of participant involvement is determined by the assessment of outcomes through the end-of-study visit.

The experimental treatment consists of upadacitinib administered as 30 mg prolonged-release tablets. This medication is intended for oral administration on a once-daily (QD) dosing schedule.

The control group receives a placebo that is identical in composition to the active medication but lacks the pharmacological substance. This comparator is intended to maintain the blinding of the study.

The clinical efficacy of upadacitinib will be evaluated in adult patients with erosive mucosal lichen planus and lichen planopilaris. The primary endpoint is the achievement of an Investigator Global Assessment (IGA) response, defined as an absolute IGA score of ≤2, at Week 16.

Secondary efficacy parameters include:

• Achievement of a 2-point improvement in the IGA at Week 32.
• Achievement of a Dermatology Life Quality Index (DLQI) score of 0/1 at Week 16 and Week 32.
• Patient Global Impression of Severity (PGIS) at Week 16 and Week 32.
• Patient Global Impression of Change (PGIC) at Week 16 and Week 32.
• Absolute and relative changes in Revised Erosive Ulceration (REU), Oral Lichen Planus Severity Score Modified (OLPSSM), and Numeric Rating Scale (NRS) scores at Week 16 and Week 32.
• Absolute and relative changes in Lichen Planus Peroral Activity Index (LPPAI) and SCALPDEX Questionnaire scores at Week 16 and Week 32.