NCT07352917 Methotrexate for Severe Vulvar Lichen Sclerosus
| NCT ID | NCT07352917 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Clinica Dermatologica Arbache ltda |
| Condition | Vulvar Lichen Sclerosus |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2026-01-21 |
| Primary Completion | 2027-01 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
Vulvar lichen sclerosus is a chronic inflammatory skin disease affecting the vulva and genital region, which may cause intense pruritus, pain, burning sensations, and progressive scarring. In severe cases, the disease may lead to significant anatomical changes, sexual dysfunction, and a reduction in quality of life. First-line treatment for vulvar lichen sclerosus consists of high-potency topical corticosteroids. Although many patients experience symptom relief, a considerable number of women continue to have persistent symptoms or progressive anatomical damage. For these patients, therapeutic options are limited. MTX is an immunomodulatory medication that has been used for several decades in the treatment of inflammatory and autoimmune diseases. Previous clinical observations suggest that MTX may be beneficial for patients with severe or treatment-resistant vulvar lichen sclerosus; however, prospective clinical studies in this population remain scarce. This prospective, open-label, phase II pilot study aims to evaluate the efficacy and safety of subcutaneous injectable MTX, administered once weekly at a standardized initial dose of 12.5 mg, in women with severe vulvar lichen sclerosus who have not responded adequately to standard topical therapy. Participants will receive treatment for 12 months, with regular clinical and laboratory monitoring. The primary objective of the study is to assess whether treatment with MTX leads to improvement in symptoms and stabilization of vulvar skin changes, using patient-reported outcome measures and standardized clinical assessments. Secondary objectives include evaluation of treatment tolerability, adherence, and the occurrence of adverse events. As a pilot study, this research is designed to generate preliminary data on feasibility, safety, and potential clinical benefit, which may guide future larger controlled studies and contribute to the development of evidence-based therapeutic strategies for women with severe vulvar lichen sclerosus.
Eligibility Criteria
Inclusion Criteria: * Clinical diagnosis of genital lichen sclerosus et atrophicus (vulvar, perineal, or perianal); * Histopathological diagnosis of genital lichen sclerosus et atrophicus; * Disease severity defined according with a score above 24 on the Vulvar Quality of Life Index (VQLI); * Failure of symptom regression or progressive genital deformity after at least 3 months of treatment with a high-potency topical corticosteroid; * Normal baseline laboratory evaluation, including complete blood count, liver and kidney function tests, and negative serologies for HIV and hepatitis B and C; * For women of childbearing potential, a negative pregnancy test at screening. Exclusion Criteria (assessed before signing the Informed Consent Form): * Lack of full mental or psychiatric capacity; * Need for assistance from a third party (legal guardian) to participate in the study; * Significant difficulty in understanding or expressing oneself in Portuguese; * Unavailability or inability to comp