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Methotrexate for Severe Vulvar Lichen Sclerosus

ConditionVulvar Lichen Sclerosus
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexFemale
Age18+
SponsorClinica Dermatologica Arbache ltda

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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 complete Likert-scale questionnaires;

Trial design

Design model

Single group

Treatments tested in this trial

  • Methotrexate (MTX)

    Drug

    Subcutaneous injectable methotrexate administered once weekly, starting at a standardized initial dose of 12.5 mg per administration. Dose adjustments may be performed according to clinical response and safety monitoring, based on predefined protocol criteria. Folic acid supplementation will be administered on the day following the weekly methotrexate injection.

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: Subcutaneous MethotrexateExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change in vulvar symptom severity

Change in vulvar symptom severity from baseline to 12 months, assessed using the total symptom score of the Clinical Scoring System for Vulvar Lichen Sclerosus (symptom domains only), as described by Günthert et al. (J Sex Med. 2012;9:2342-2350). The total score ranges from 0 to 30 points and is calculated by summing the items within the symptom domain, yielding a single aggregated value per assessment. Higher scores indicate greater symptom severity. Symptoms related to sexual discomfort or pain during sexual activity are not included in the primary outcome score and will be recorded separately and reported descriptively.

Time frame
12 months

Secondary outcomes

1

Change in vulvar morphology

Change in vulvar morphology score from baseline to 12 months, assessed using the total morphology score of the Clinical Scoring System for Vulvar Lichen Sclerosus as described by Günthert et al. (J Sex Med. 2012;9:2342-2350). The morphology score is a single aggregated score, ranging from 0 to 12, with higher scores indicating greater morphological severity.

Time frame
12 months
2

Change in vulvar-related quality of life

Change in vulvar-related quality of life from baseline to 12 months, assessed using the Vulvar Quality of Life Index (VQLI), as described by Saunderson et al. Australasian Journal of Dermatology 61.2 (2020): 152-157. Total VQLI scores range from 0 to 45, with higher scores indicating worse quality of life.

Time frame
12 months
3

Safety and tolerability of methotrexate

Safety outcomes will include the incidence of adverse events, laboratory abnormalities, and treatment discontinuations throughout the study period.

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Clinica Dermatologica Arbache ltda

Lead sponsor

Source ClinicalTrials.gov
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