Study of Alwextin® Cream in Treating Epidermolysis Bullosa
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Open-label, Pilot Study to Investigate the Safety and Tolerability of Alwextin 3.0% Cream in the Treatment of Epidermolysis Bullosa|
- Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage [ Time Frame: baseline and then every 4 weeks for a total of 12 weeks ] [ Designated as safety issue: No ]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.
- Target Wound Size Reduction or Closure [ Time Frame: baseline and then every 4 weeks for a total of 12 weeks ] [ Designated as safety issue: No ]
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.
- Physician Global Assessment of Severity (PGAS) [ Time Frame: baseline and then every 4 weeks for a total of 12 weeks ] [ Designated as safety issue: No ]
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)
- Physician Assessment of Individual Signs [ Time Frame: baseline and then every 4 weeks for a total of 12 weeks ] [ Designated as safety issue: No ]
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
|Study Start Date:||February 2009|
|Study Completion Date:||November 2010|
|Primary Completion Date:||November 2010 (Final data collection date for primary outcome measure)|
Experimental: Alwextin cream
8 subjects enrolled in this single study arm. All 8 subjects completed the study.
Drug: Alwextin cream
Alwextin cream contains active ingredient, allantoin 3%. Use 1 application daily for 3 month duration.
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.
|United States, Illinois|
|Robert and Ann Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||Amy S Paller, MD||Northwestern University|