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| Name | Class |
|---|---|
| BPGbio | INDUSTRY |
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This is a single-center study to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa.
The trial will be conducted in patients with any form of Epidermolysis Bullosa (EB) with at least 1 active EB wound between 2.5 and 50 cm2 in size or up to 10% of the Body Surface Area. The investigators will identify an "index lesion" and other lesions for treatment An area of un-involved skin will also be treated with the study cream. This are will be used for testing by producing tiny blisters th so that the underlying tissue can be examined using a special microscope.
Patients will apply the study cream
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Topical BPM31510 3.0% Cream | Experimental | Patients/ caregiver will apply topical BPM31510 3.0% cream from every other day to twice per week to wounded skin, and every day to a section of intact skin for up to 12 weeks.The area to be covered may not exceed 10% BSA inclusive of intact skin area, and other lesions. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| topical BPM31510 3.0% Cream | Drug | Application of drug to lesions of epidermolysis bullosa |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment | Analysis of laboratory values and/or Adverse events | 16 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Peak Plasma Concentration of BPM31510 | Blood will be drawn pre-study drug application on Day 1 and post study drug application on Day 3 and Week 8 | Day 1, Day 3, week 8. |
| Decrease in VAS Pain Scale questionnaire |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Miami Department of Dermatology | Miami | Florida | 33136 | United States |
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| 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 |
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Subject completed Questionnaire will assess the .pain at each visit.
| Baseline, 16 weeks |
| Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) | The (EBDASI) be utilized by investigators to quantify wound healing and scarring at each visit. | Baseline, 16 weeks |
| Change in the Lansky Performance Scale and the Children's Dermatology Life Quality Index | Subject completed questionnaires will be completed at each visit. | Baseline, 16 weeks |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |