Imiquimod in Children With Plaque Morphea

Study Overview
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Brief Summary
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Morphea is very hard to treat. In a small number of adult patients, Imiquimod has proven to be beneficial in decreasing the thickness of the morphea plaques , while improving their appearance. There are no studies to date proving its safety and efficacy in children with this disease. We propose to conduct a pilot study to assess to potential efficacy and relative safety of Imiquimod in children with plaque morphea.

Detailed Description
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Design: Prospective, open label, pilot study Settings: The Hospital for Sick Children, Specialized Morphea Clinic Study population: Children 6-18 years of age Plaque morphea measuring less than 10 cm2 in diameter ( for children with multiple lesions, only one will be treated) Intervention: Topical imiquimod applied 3-5 times a week for 6 months Outcome measures: Decrease in the thickness of the skin by clinical scores and ultrasonography Duration of the study: 12 months ( 2 baseline visits, 1 intervention visit, 5 follow-up visits)

Official Title
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Evaluation of the Efficacy and Safety of the Imiquimod 5% Topical Cream in Plaque Morphea: A Prospective, Multiple Baseline, Open Label Pilot Study

Conditions
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Scleroderma, Localized

Intervention / Treatment
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* Drug: Imiquimod 5% cream


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age at diagnosis 6 to 18 years of age Morphea plaques Female subjects of childbearing potential must have a negative urine pregnancy test Signed consent/assent form Exclusion criteria: Children who received topical corticosteroids, tacrolimus, vitamin D derivatives (calcipotriol, calcipotriol-betamethasone dipropionate) to the affected area in the previous four weeks Children who were previously treated with Imiquimod on the affected areas Children with no demonstrable ultrasonographic changes at the baseline evaluation Children with evidence of skin breakdown on the proposed area to be treated at the time of enrollment due to potential increased absorption of the medication through impaired skin barrier Female subjects of childbearing potential who do not agree to practice effective birth control methods for the duration of the study Children who are/were (in the past 6 months) treated with systemic medications such as methotrexate and/or systemic corticosteroids Co-morbidities: systemic sclerosis, juvenile rheumatoid arthritis, other systemic diseases

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: Imiquimod 5% cream<br>* Treatment will last for 36 weeks. Patients will be instructed to apply Imiquimod three times per week for 4 weeks. If no local side effects are noted at the 4 week- follow-up visit, the frequency of the application will be increased to 5 weekly applications.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent improvement in the thickness of the skin |  | 4 weeks, 12 weeks, 24 weeks, 36 weeks, and 48 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of side-effects |  | 48 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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localized scleroderma, morphea, imiquimod, pediatrics