Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Ann & Robert H Lurie Children's Hospital of Chicago | OTHER |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alwextin cream | Experimental | 8 subjects enrolled in this single study arm. All 8 subjects completed the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alwextin cream | Drug | Alwextin cream contains active ingredient, allantoin 3%. Use 1 application daily for 3 month duration. |
|
| 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 |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Amy S Paller, MD | Northwestern University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Robert and Ann Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
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).
Subjects were identified at regularly scheduled clinic visits with the Principal Investigator at Children's Memorial Hospital.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Subjects who used allantoin 3% cream for an entire month prior to the BSA monthly assessment were included in the analysis. | Posted | Mean | Standard Deviation | percentage of BSA involvement | baseline and then every 4 weeks for a total of 12 weeks |
|
For 12 week duration of study. Adverse events (AE) and serious adverse events (SAE_that were unresolved by study completion date were followed for an additional 1 month post-study completion.
AE is defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medical treatment or procedure. SAE meets any of these criteria: causes death, hospitalization, disability, or birth defect, is life-threatening, is an important medical event that may require intervention to prevent these outcomes.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| red, itchy skin | Skin and subcutaneous tissue disorders | Non-systematic Assessment | unlikely to be related to study medication |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Stephanie Rangel | Northwestern University | 312-503-5942 | stephanie.rangel@northwestern.edu |
Not provided
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
Not provided
Not provided
| ID | Term |
|---|---|
| D000481 | Allantoin |
| ID | Term |
|---|---|
| D014508 | Urea |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D006827 | Hydantoins |
| D048289 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units |
|---|
| Counts |
|---|
| 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. | Subjects who used allantoin 3% cream on the target wound daily up to full closure of that wound were included in the analysis. | Posted | Number | number of unhealed target wounds | baseline and then every 4 weeks for a total of 12 weeks |
|
|
|
| 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) | 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. | Posted | Count of Participants | Participants | baseline and then every 4 weeks for a total of 12 weeks |
|
|
|
| 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 | Subjects who used cream on entire body for the entire month was used in the analysis. | Posted | Count of Participants | Participants | baseline and at 12 weeks |
|
|
|
| 0 |
| 8 |
| 4 |
| 8 |
|
| cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | unlikely to be related to study medication |
|
| fever | General disorders | Systematic Assessment | unlikely to be related to study medication |
|
| red spots on right foot | Skin and subcutaneous tissue disorders | Non-systematic Assessment | unlikely to be related to study medication |
|
| red spots on left foot | Skin and subcutaneous tissue disorders | Non-systematic Assessment | unlikely to be related to study medication |
|
| diarrhea | Gastrointestinal disorders | Non-systematic Assessment | unlikely to be related to study medication |
|
| hematemesis | Gastrointestinal disorders | Non-systematic Assessment | not related to study medication |
|
Not provided
Not provided
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
| Imidazolidines |
| D007093 | Imidazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| PGAS score of 3 |
|
| PGAS score of 4 |
|
| PGAS score of 5 |
|
| PGAS score of 6 |
|
| PGAS score of 7 |
|
| PGAS score of 8 |
|
| PGAS score of 9 |
|
| PGAS score of 10 |
|
| Month 1 |
|
|
| Month 2 |
|
|
| Month 3 |
|
|
|
| Pain-Month 3 |
|
|
| Pruritis-Baseline |
|
|
| Pruritus-Month 3 |
|
|
| Blisters/Erosions-Baseline |
|
|
| Blisters/Erosions- Month 3 |
|
|
| Oozing/Weeping/Crusting-Baseline |
|
|
| Oozing/Weeping/Crusting- Month 3 |
|
|
| Erythema-Baseline |
|
|
| Erythema- Month 3 |
|
|
| Milia-Baseline |
|
|
| Milia- Month 3 |
|
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|
| Almost Clear |
|
| Mild |
|
| Moderate |
|
| Severe |
|