Study of Alwextin® Cream in Treating Epidermolysis Bullosa

Study Overview
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Brief Summary
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The purpose of this study is to determine how safe and effective allantoin 3% cream (Alwextin) is in improving the healing of recurrent skin lesions and reducing overall blistering in people with epidermolysis bullosa (EB). Allantoin 3% cream is applied topically to the entire body once daily.

Detailed Description
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Potential subjects came to the for a screening visit. Eligible subjects had baseline assessments performed and were provided study medication, allantoin 3% cream. Subjects were instructed to apply the study medication to the entire body once daily and to keep daily record of study medication use. Subjects returned every 4 weeks for a total of 12 weeks for repeat assessments.

Official Title
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Open-label, Pilot Study to Investigate the Safety and Tolerability of Alwextin 3.0% Cream in the Treatment of Epidermolysis Bullosa

Conditions
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Epidermolysis Bullosa

Intervention / Treatment
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* Drug: Alwextin cream


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: history of epidermolysis bullosa Exclusion Criteria: use of any skin product containing allantoin for 30 days prior to enrollment

Ages Eligible for Study
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Minimum Age: 6 Months

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Alwextin cream<br>8 subjects enrolled in this single study arm. All 8 subjects completed the study. | Drug: Alwextin cream<br>* Alwextin cream contains active ingredient, allantoin 3%. Use 1 application daily for 3 month duration.<br>* Other names: allantoin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | A common measure of the degree of involvement in skin disease is the Body Surface Area Index (BSAI). This measure is also commonly used in psoriasis studies. It is a global measure of disease spread with weighting factors. | baseline and then every 4 weeks for a total of 12 weeks | 
| Target Wound Size Reduction or Closure | EB patients may have chronic wounds which are resistant to healing. Wound size may be very large and the probability of total wound closure with currently available treatments is unlikely. Reduction in the size of wounds may be clinically important to the rate of infection and pain. If a patient has a reduction in the size of wounds which are refractory to healing, this may be seen as a positive outcome. Wound size reduction is one of the primary assessments used to determine the efficacy of the study cream. Wounds which had been present for at least several weeks prior to study entry were measured by using VISITRAK Digital, a Smith and Nephew wound tracing and measurement system that will calculate the length and width of the lesion (class 1 medical device; FDA listing designation E142354FDA). Only one target lesion per patient was used for the study assessment. At each subsequent study until the final visit, the target lesion was evaluated using VISITRAK Digital. | baseline and then every 4 weeks for a total of 12 weeks | 
| Physician Global Assessment of Severity (PGAS) | The FDA has suggested that a global measure of severity might be the best way to assess EB from visit to visit. Assessment score may be influenced by other clinical observations in addition to the percentage of body affected by blistering and erosions. The assessment was intended to be a global impression. This scale produced a score with the following correlations: 0 = clear (no blistering/erosions) 1-2 = almost clear (infrequent blistering and erosions) 3-4 = mild disease (up to 15% of body affected) 5-6 = moderate disease (between 16-25% of body affected) 7-8 = severe disease (between 26-50% of body affected) 9-10 = very severe disease (greater than 50% of body affected) | baseline and then every 4 weeks for a total of 12 weeks | 
| Physician Assessment of Individual Signs | In addition to skin blistering and erosions, people with EB experience other symptoms, such as erythema on unblistered skin, wound oozing, weeping, and crusting. These symptoms may vary with area of the body evaluated. This scale evaluates the following signs: Blistering and erosions, oozing/weeping/crusting, pruritis, erythema on unblistered surrounding skin, pain, milia Each of these signs will be scored in 4 body areas: head/neck, upper limbs, trunk, lower limbs The following scale is used:0 = clear 1 = almost clear 2 = mild 3 = moderate 4 = severe | baseline and at 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Epidermolysis bullosa