Scleroderma, Localized
Conditions
Keywords
localized scleroderma, morphea, imiquimod, pediatrics
Brief summary
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
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)
Interventions
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.
Sponsors
The Hospital for Sick Children
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent improvement in the thickness of the skin | 4 weeks, 12 weeks, 24 weeks, 36 weeks, and 48 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Frequency of side-effects | 48 weeks |
Countries
Canada
Outcome results
None listed