Epidermolysis Bullosa
Conditions
Keywords
Epidermolysis bullosa
Brief summary
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
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.
Interventions
DRUGAlwextin cream
Alwextin cream contains active ingredient, allantoin 3%. Use 1 application daily for 3 month duration.
Sponsors
Ann & Robert H Lurie Children's Hospital of Chicago
Northwestern University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
Inclusion criteria
* history of epidermolysis bullosa
Exclusion criteria
* use of any skin product containing allantoin for 30 days prior to enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | baseline and then every 4 weeks for a total of 12 weeks | 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 | baseline and then every 4 weeks for a total of 12 weeks | 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) | baseline and then every 4 weeks for a total of 12 weeks | 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 | baseline and at 12 weeks | 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 |
Countries
United States
Participant flow
Recruitment details
Subjects were identified at regularly scheduled clinic visits with the Principal Investigator at Children's Memorial Hospital.
Pre-assignment details
Subjects were instructed to discontinue use of allantoin 1.5% cream (Alwyn) for at least 30 days prior to initiating the study medication, allantoin 3% cream (Alwextin).
Participants by arm
| Arm | Count |
|---|---|
| Allantoin 3% Cream This is an open-label study in which all enrolled subjects were assigned to receive the study medication, allantoin 3% cream. Subjects were required to have a diagnosis of Epidermolysis Bullosa (EB). The diagnosis of EB was based on diagnostic immunomapping, electron microscopy, or the principal investigator's judgment based on clear clinical characteristics. Subjects were instructed to apply allantoin 3% cream once daily to the entire body. | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Allantoin 3% Cream |
|---|---|
| Age, Categorical <=18 years | 8 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 3.3 years STANDARD_DEVIATION 2.7 |
| Region of Enrollment United States | 8 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 8 |
| serious Total, serious adverse events | 0 / 8 |
Outcome results
Primary
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.
Time frame: baseline and then every 4 weeks for a total of 12 weeks
Population: Subjects who used allantoin 3% cream for an entire month prior to the BSA monthly assessment were included in the analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Allantoin 3% Cream | Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | Baseline assessment | 57.25 percentage of BSA involvement | Standard Deviation 19.1 |
| Allantoin 3% Cream | Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | Week 4 assessment | 39.40 percentage of BSA involvement | Standard Deviation 24.1 |
| Allantoin 3% Cream | Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | Week 8 assessment | 27.25 percentage of BSA involvement | Standard Deviation 17.9 |
| Allantoin 3% Cream | Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage | Week 12 assessment | 25.45 percentage of BSA involvement | Standard Deviation 21.1 |
Primary
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
Time frame: baseline and at 12 weeks
Population: Subjects who used cream on entire body for the entire month was used in the analysis.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Baseline | Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia- Month 3 | Almost Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Almost Clear | 3 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Month 3 | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema-Baseline | Severe | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Mild | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Moderate | 3 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema-Baseline | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema-Baseline | Mild | 4 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Almost Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Mild | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema-Baseline | Almost Clear | 3 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Moderate | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia- Month 3 | Clear | 3 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema-Baseline | Clear | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritis-Baseline | Clear | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritis-Baseline | Almost Clear | 5 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Baseline | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia-Baseline | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritis-Baseline | Mild | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritis-Baseline | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Baseline | Almost Clear | 4 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia-Baseline | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritis-Baseline | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia- Month 3 | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritus-Month 3 | Clear | 3 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Baseline | Severe | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia-Baseline | Mild | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritus-Month 3 | Almost Clear | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritus-Month 3 | Mild | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia-Baseline | Almost Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritus-Month 3 | Moderate | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pruritus-Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Month 3 | Clear | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia-Baseline | Clear | 7 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Almost Clear | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema- Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Mild | 4 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia- Month 3 | Mild | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Moderate | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Month 3 | Almost Clear | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema- Month 3 | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Clear | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Milia- Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema- Month 3 | Mild | 1 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Almost Clear | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Mild | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Month 3 | Mild | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema- Month 3 | Almost Clear | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Moderate | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Severe | 0 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Pain-Baseline | Mild | 2 Participants |
| Allantoin 3% Cream | Physician Assessment of Individual Signs | Erythema- Month 3 | Clear | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Clear | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Severe | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Moderate | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Almost Clear | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Almost Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Moderate | 4 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Almost Clear | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Mild | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Moderate | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Moderate | 3 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Severe | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Moderate | 5 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Almost Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Almost Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Severe | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Clear | 3 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Mild | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Almost Clear | 4 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Almost Clear | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Moderate | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Almost Clear | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Almost Clear | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Moderate | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Clear | 8 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Severe | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Moderate | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Almost Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Mild | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Moderate | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Almost Clear | 5 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Clear | 2 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Severe | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Mild | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Moderate | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Moderate | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Almost Clear | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Moderate | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Clear | 0 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Clear | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Severe | 3 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Mild | 1 Participants |
| Upper Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Mild | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Baseline | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Baseline | Almost Clear | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Baseline | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Baseline | Moderate | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Baseline | Severe | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Month 3 | Clear | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Month 3 | Almost Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Month 3 | Mild | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Month 3 | Moderate | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pain-Month 3 | Severe | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritis-Baseline | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritis-Baseline | Almost Clear | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritis-Baseline | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritis-Baseline | Moderate | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritis-Baseline | Severe | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritus-Month 3 | Clear | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritus-Month 3 | Almost Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritus-Month 3 | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritus-Month 3 | Moderate | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Pruritus-Month 3 | Severe | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Almost Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Mild | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Moderate | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Severe | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Almost Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Mild | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Moderate | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Severe | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Almost Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Mild | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Moderate | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Severe | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Almost Clear | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Moderate | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Severe | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema-Baseline | Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema-Baseline | Almost Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema-Baseline | Mild | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema-Baseline | Moderate | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema-Baseline | Severe | 4 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema- Month 3 | Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema- Month 3 | Almost Clear | 2 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema- Month 3 | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema- Month 3 | Moderate | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Erythema- Month 3 | Severe | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia-Baseline | Clear | 8 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia-Baseline | Almost Clear | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia-Baseline | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia-Baseline | Moderate | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia-Baseline | Severe | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia- Month 3 | Clear | 3 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia- Month 3 | Almost Clear | 1 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia- Month 3 | Mild | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia- Month 3 | Moderate | 0 Participants |
| Trunk | Physician Assessment of Individual Signs | Milia- Month 3 | Severe | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Severe | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Moderate | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Almost Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Mild | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Almost Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Almost Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Almost Clear | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions- Month 3 | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Severe | 5 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Almost Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Moderate | 3 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Clear | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Moderate | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Almost Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Blisters/Erosions-Baseline | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Severe | 4 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema- Month 3 | Severe | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Severe | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Moderate | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Clear | 8 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Almost Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Almost Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritus-Month 3 | Clear | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Severe | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Moderate | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Moderate | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Mild | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Baseline | Mild | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Moderate | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Almost Clear | 3 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pruritis-Baseline | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Severe | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia-Baseline | Severe | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Severe | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Moderate | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Severe | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Mild | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Almost Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Moderate | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Almost Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting- Month 3 | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Severe | 4 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Moderate | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Mild | 3 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Moderate | 3 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Mild | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Milia- Month 3 | Clear | 4 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Moderate | 2 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Almost Clear | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Oozing/Weeping/Crusting-Baseline | Clear | 0 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Pain-Month 3 | Mild | 1 Participants |
| Lower Limbs | Physician Assessment of Individual Signs | Erythema-Baseline | Severe | 3 Participants |
Primary
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)
Time frame: baseline and then every 4 weeks for a total of 12 weeks
Population: One subject discontinued after week 3. One subject discontinued at Month 2. Only data presented for subjects who used cream on entire body for the entire month was used in the analysis.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 3 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 2 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 1 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 2 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 3 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 4 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 5 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 6 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 7 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 8 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 9 | 3 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Baseline | PGAS score of 10 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 1 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 2 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 3 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 4 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 5 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 6 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 7 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 8 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 9 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 1 | PGAS score of 10 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 1 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 4 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 5 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 6 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 7 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 8 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 9 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 2 | PGAS score of 10 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 1 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 2 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 3 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 4 | 2 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 5 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 6 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 7 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 8 | 0 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 9 | 1 Participants |
| Allantoin 3% Cream | Physician Global Assessment of Severity (PGAS) | Month 3 | PGAS score of 10 | 0 Participants |
Primary
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.
Time frame: baseline and then every 4 weeks for a total of 12 weeks
Population: Subjects who used allantoin 3% cream on the target wound daily up to full closure of that wound were included in the analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Allantoin 3% Cream | Target Wound Size Reduction or Closure | Baseline assessment | 8 number of unhealed target wounds |
| Allantoin 3% Cream | Target Wound Size Reduction or Closure | Week 12 assessment | 1 number of unhealed target wounds |