Topical 5% Imiquimod Cream for Vulvar Paget's Disease

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy, safety and immunological response of topical 5% imiquimod cream for non-invasive vulvar Paget's disease.

Detailed Description
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Vulvar Paget's disease is a rare skin tumour and generally seen in postmenopausal, Caucasian women as an erythematous, eczematous, scaling or ulcerating plaque. Initial symptoms are irritation, burning, pain and a skin lesion. The origin of vulvar Paget's disease is not yet entirely understood. The treatment of choice is surgical excision, despite high recurrence rates. The mutilating consequences of surgery can lead to impressive morbidity to address this, several series report the use of topical 5% imiquimod cream for vulvar Paget's disease. The objective of this study is to assess the efficacy, evaluate the safety, immunological effect of topical 5% imiquimod cream for vulvar Paget's disease and the quality of life during treatment.

Official Title
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Topical 5% Imiquimod Cream for Vulvar Paget's Disease: Clinical Efficacy, Safety and Immunological Response

Conditions
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Paget Disease, Extramammary

Intervention / Treatment
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* Drug: Imiquimod
* Drug: paracetamol
* Drug: lidocaine in Vaseline ointment


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Non-invasive vulvar Paget's disease, primary or recurrence after earlier surgery; Willing and able to comply with the protocol and to provide informed consent in accordance with institutional and regulatory guidelines. Exclusion Criteria: Invasive vulvar Paget's disease; Underlying adenocarcinoma; Treatment of the vulva with topical 5% imiquimod cream during the last 6 months; Participation in a study with another investigational product within 30 days prior to enrolment in this study; Hypersensitivity to any component of topical 5% imiquimod cream (methylhydroxybenzoate (E218), propylhydroxybenzoate (E216), cetylalcohol, atearylalcohol); Patients with autoimmune disorders; Immune compromised patients (e.g. HIV patients, patients with a history of transplantation); Insufficient understanding of the Dutch or English language; Pregnant women; Lactating women.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Imiquimod<br>Topical 5% imiquimod cream will be applicated to the vulvar skin lesion 3 times a week during 16 weeks. | Drug: Imiquimod<br>* topical 5% imiquimod cream 3 times a week for 16 weeks.<br>* Other names: Aldara;Drug: paracetamol<br> <br> Drug: lidocaine in Vaseline ointment<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical response | Clinical response will be assessed by vulvar examination and measurement and is defined as complete remission, partial remission (decrease by ≥50% of total lesion size) or no remission. | 12 weeks after the end of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Tolerability questionnaire | Tolerability will be assessed in a descriptive manner with a tolerability questionnaire with the following questions: Have you used paracetamol during treatment? How many days? What dosage? Was the use sufficient? Have you used lidocaine in Vaseline ointment during treatment? How many days? What dosage? Was the use sufficient? Have you stopped the treatment with imiquimod because of side effects/pain? | 4, 10 and 16 weeks after the start of treatment | 
| Adverse events | Most common adverse events will be evaluated with the following questions: Have you been nauseous? No / a little (less than 3 days per week) / severe (more than 3 days a week) / Yes, daily. Have you had a head ache? No / a little (less than 3 days per week) / severe (more than 3 days a week) / Yes, daily. Have you felt more tired than you normally are? No / a little (less than 3 days per week) / severe (more than 3 days a week) / Yes, daily. | 4, 10 and 16 weeks after the start of treatment | 
| Immunological response | Vulvar skin biopsies will be taken at baseline, 4 and 28 weeks after the start of | Baseline, 4 and 28 weeks after start of treatment | 
| Quality of life: general. | The questionnaire EQ5D (EuroQOL 5 Dimensions questionnaire) will be used. | Baseline, 4 and 28 weeks after start of treatment. | 
| Quality of life: dermatology specific. | The questionnaire DLQI (Dermatology Life Quality Index) will be used. | Baseline, 4 and 28 weeks after start of treatment. | 
| Quality of life: sexuality. | The questionnaire FSDS (Female Sexual Distress Scale) will be used. | Baseline, 4 and 28 weeks after start of treatment. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Vulva, Extramammary Paget's disease, Genital, female, imiquimod