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Study of Alwextin® Cream in Treating Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT00825565
Recruitment Status : Completed
First Posted : January 21, 2009
Results First Posted : January 1, 2013
Last Update Posted : February 28, 2018
Sponsor:
Collaborator:
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
Amy Paller, Northwestern University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Epidermolysis Bullosa
Intervention Drug: Alwextin cream
Enrollment 8
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).
Arm/Group Title Allantoin 3% Cream
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title Allantoin 3% Cream
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
<=18 years
8
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
3.3  (2.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
5
  62.5%
Male
3
  37.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
1.Primary Outcome
Title Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who used allantoin 3% cream for an entire month prior to the BSA monthly assessment were included in the analysis.
Arm/Group Title Allantoin 3% Cream
Hide Arm/Group Description:
Subjects were instructed to apply allantoin 3% cream once daily to the entire body.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: percentage of BSA involvement
Baseline assessment 57.25  (19.1)
Week 4 assessment 39.40  (24.1)
Week 8 assessment 27.25  (17.9)
Week 12 assessment 25.45  (21.1)
2.Primary Outcome
Title Target Wound Size Reduction or Closure
Hide Description

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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who used allantoin 3% cream on the target wound daily up to full closure of that wound were included in the analysis.
Arm/Group Title Allantoin 3% Cream
Hide Arm/Group Description:
Subjects were instructed to apply allantoin 3% cream once daily to the target lesion area for assessing the impact on target wound closure.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: number of unhealed target wounds
Baseline assessment 8
Week 12 assessment 1
3.Primary Outcome
Title Physician Global Assessment of Severity (PGAS)
Hide Description

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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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/Group Title Alwextin Cream
Hide Arm/Group Description:

8 subjects enrolled in this single study arm. All 8 subjects completed the study.

Alwextin cream: Alwextin cream contains active ingredient, allantoin 3%. Use 1 application daily for 3 month duration.

Overall Number of Participants Analyzed 8
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 8 participants
PGAS score of 1
0
   0.0%
PGAS score of 2
0
   0.0%
PGAS score of 3
0
   0.0%
PGAS score of 4
0
   0.0%
PGAS score of 5
0
   0.0%
PGAS score of 6
0
   0.0%
PGAS score of 7
1
  12.5%
PGAS score of 8
2
  25.0%
PGAS score of 9
3
  37.5%
PGAS score of 10
2
  25.0%
Month 1 Number Analyzed 7 participants
PGAS score of 1
0
   0.0%
PGAS score of 2
0
   0.0%
PGAS score of 3
0
   0.0%
PGAS score of 4
0
   0.0%
PGAS score of 5
2
  28.6%
PGAS score of 6
1
  14.3%
PGAS score of 7
2
  28.6%
PGAS score of 8
1
  14.3%
PGAS score of 9
0
   0.0%
PGAS score of 10
1
  14.3%
Month 2 Number Analyzed 6 participants
PGAS score of 1
0
   0.0%
PGAS score of 2
0
   0.0%
PGAS score of 3
0
   0.0%
PGAS score of 4
1
  16.7%
PGAS score of 5
2
  33.3%
PGAS score of 6
1
  16.7%
PGAS score of 7
1
  16.7%
PGAS score of 8
0
   0.0%
PGAS score of 9
1
  16.7%
PGAS score of 10
0
   0.0%
Month 3 Number Analyzed 4 participants
PGAS score of 1
0
   0.0%
PGAS score of 2
0
   0.0%
PGAS score of 3
0
   0.0%
PGAS score of 4
2
  50.0%
PGAS score of 5
0
   0.0%
PGAS score of 6
0
   0.0%
PGAS score of 7
1
  25.0%
PGAS score of 8
0
   0.0%
PGAS score of 9
1
  25.0%
PGAS score of 10
0
   0.0%
4.Primary Outcome
Title Physician Assessment of Individual Signs
Hide Description

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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who used cream on entire body for the entire month was used in the analysis.
Arm/Group Title Head/Neck Upper Limbs Trunk Lower Limbs
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 8 8 8 8
Measure Type: Count of Participants
Unit of Measure: Participants
Pain-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
1
  12.5%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
4
  50.0%
4
  50.0%
3
  37.5%
1
  12.5%
Mild
2
  25.0%
1
  12.5%
0
   0.0%
1
  12.5%
Moderate
0
   0.0%
1
  12.5%
3
  37.5%
2
  25.0%
Severe
1
  12.5%
2
  25.0%
2
  25.0%
4
  50.0%
Pain-Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
2
  50.0%
2
  50.0%
2
  50.0%
2
  50.0%
Almost Clear
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Mild
2
  50.0%
1
  25.0%
1
  25.0%
1
  25.0%
Moderate
0
   0.0%
1
  25.0%
0
   0.0%
0
   0.0%
Severe
0
   0.0%
0
   0.0%
1
  25.0%
1
  25.0%
Pruritis-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
2
  25.0%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
5
  62.5%
5
  62.5%
3
  37.5%
3
  37.5%
Mild
1
  12.5%
0
   0.0%
0
   0.0%
2
  25.0%
Moderate
0
   0.0%
0
   0.0%
2
  25.0%
2
  25.0%
Severe
0
   0.0%
3
  37.5%
3
  37.5%
1
  12.5%
Pruritus-Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
3
  75.0%
2
  50.0%
2
  50.0%
2
  50.0%
Almost Clear
0
   0.0%
1
  25.0%
1
  25.0%
1
  25.0%
Mild
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Moderate
1
  25.0%
1
  25.0%
1
  25.0%
1
  25.0%
Severe
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Blisters/Erosions-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
1
  12.5%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
1
  12.5%
1
  12.5%
0
   0.0%
0
   0.0%
Mild
4
  50.0%
1
  12.5%
2
  25.0%
0
   0.0%
Moderate
2
  25.0%
4
  50.0%
3
  37.5%
3
  37.5%
Severe
0
   0.0%
2
  25.0%
3
  37.5%
5
  62.5%
Blisters/Erosions- Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
2
  50.0%
0
   0.0%
1
  25.0%
1
  25.0%
Mild
2
  50.0%
2
  50.0%
2
  50.0%
1
  25.0%
Moderate
0
   0.0%
2
  50.0%
0
   0.0%
1
  25.0%
Severe
0
   0.0%
0
   0.0%
1
  25.0%
1
  25.0%
Oozing/Weeping/Crusting-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
1
  12.5%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
3
  37.5%
2
  25.0%
1
  12.5%
1
  12.5%
Mild
1
  12.5%
0
   0.0%
1
  12.5%
0
   0.0%
Moderate
3
  37.5%
5
  62.5%
3
  37.5%
3
  37.5%
Severe
0
   0.0%
1
  12.5%
3
  37.5%
4
  50.0%
Oozing/Weeping/Crusting- Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
1
  25.0%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
1
  25.0%
1
  25.0%
2
  50.0%
1
  25.0%
Mild
0
   0.0%
1
  25.0%
1
  25.0%
1
  25.0%
Moderate
2
  50.0%
2
  50.0%
0
   0.0%
1
  25.0%
Severe
0
   0.0%
0
   0.0%
1
  25.0%
1
  25.0%
Erythema-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Almost Clear
3
  37.5%
2
  25.0%
1
  12.5%
0
   0.0%
Mild
4
  50.0%
1
  12.5%
1
  12.5%
3
  37.5%
Moderate
0
   0.0%
3
  37.5%
2
  25.0%
2
  25.0%
Severe
1
  12.5%
2
  25.0%
4
  50.0%
3
  37.5%
Erythema- Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
1
  25.0%
1
  25.0%
1
  25.0%
1
  25.0%
Almost Clear
2
  50.0%
2
  50.0%
2
  50.0%
2
  50.0%
Mild
1
  25.0%
1
  25.0%
0
   0.0%
0
   0.0%
Moderate
0
   0.0%
0
   0.0%
1
  25.0%
1
  25.0%
Severe
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Milia-Baseline Number Analyzed 8 participants 8 participants 8 participants 8 participants
Clear
7
  87.5%
8
 100.0%
8
 100.0%
8
 100.0%
Almost Clear
1
  12.5%
0
   0.0%
0
   0.0%
0
   0.0%
Mild
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Moderate
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Severe
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Milia- Month 3 Number Analyzed 4 participants 4 participants 4 participants 4 participants
Clear
3
  75.0%
3
  75.0%
3
  75.0%
4
 100.0%
Almost Clear
1
  25.0%
0
   0.0%
1
  25.0%
0
   0.0%
Mild
0
   0.0%
1
  25.0%
0
   0.0%
0
   0.0%
Moderate
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Severe
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame 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.
Adverse Event Reporting Description 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.
 
Arm/Group Title Allantoin 3% Cream
Hide Arm/Group Description 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.
All-Cause Mortality
Allantoin 3% Cream
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Allantoin 3% Cream
Affected / at Risk (%) # Events
Total   0/8 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Allantoin 3% Cream
Affected / at Risk (%) # Events
Total   4/8 (50.00%)    
Gastrointestinal disorders   
diarrhea * [1]  1/8 (12.50%)  1
hematemesis * [2]  1/8 (12.50%)  1
General disorders   
fever  [1]  2/8 (25.00%)  2
Respiratory, thoracic and mediastinal disorders   
cough  [1]  1/8 (12.50%)  1
Skin and subcutaneous tissue disorders   
red, itchy skin * [1]  1/8 (12.50%)  1
red spots on right foot * [1]  1/8 (12.50%)  1
red spots on left foot * [1]  1/8 (12.50%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
[1]
unlikely to be related to study medication
[2]
not related to study medication
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Stephanie Rangel
Organization: Northwestern University
Phone: 312-503-5942
Responsible Party: Amy Paller, Northwestern University
ClinicalTrials.gov Identifier: NCT00825565     History of Changes
Other Study ID Numbers: Alwextin 3.0%-04
First Submitted: January 19, 2009
First Posted: January 21, 2009
Results First Submitted: February 22, 2012
Results First Posted: January 1, 2013
Last Update Posted: February 28, 2018