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This is a pilot study designed to see if HP802-247, an investigational treatment with living human skin cells, helps to heal blisters or wounds in subjects with Dystrophic Epidermolysis Bullosa (DEB). The durability of the skin in healed wounds treated with HP802-247 will also be assessed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| HP802-247 | Experimental |
| |
| Placebo (Vehicle) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HP802-247 | Biological | Wounds up to 12 cm2: 1 spray each component (0.5 x 106 cells per mL allogeneic human keratinocytes and fibroblasts) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods Larger wounds: (>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each |
| Measure | Description | Time Frame |
|---|---|---|
| Time to healing | Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed). | 4 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Wound pain as measured by faces pain scale | Change from baseline (i.e., Week 1 of each period) in wound pain for each visit (Weeks 2-5 of each treatment period)as assessed by the Faces Pain Scale (FPS) | 4 weeks for each of 3 treatment periods |
| Percent of change in wound area |
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Inclusion Criteria:
Written consent must be obtained from a parent or legally authorized representative. Assent will be obtained according to local requirements.
Subjects with a clinical diagnosis of DEB with recurring blisters, ≥ 2 years of age at Screening. Subjects may be of either sex and of any race or skin type provided that their skin color, in the opinion of the Investigator, will not interfere with the study assessments.
A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits.
A parent or legally authorized representative must be able to follow instructions.
Have an open wound for ≤ 72 hours that:
Females of childbearing potential (defined as post-menarcheal as documented in the medical history) may participate in the study if they meet all of the following conditions:
A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits (for minor subjects)
A parent or legally authorized representative must be able to follow instructions (for minor subjects)
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Herbert B Slade, MD | Healthpoint | Study Chair |
| Jaime E Dickerson, PhD | Healthpoint | Study Director |
| Herbert B Slade, MD | Healthpoint | Principal Investigator |
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| ID | Term |
|---|---|
| D016108 | Epidermolysis Bullosa Dystrophica |
| 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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|
| Placebo | Other | Wounds up to 12 cm2: 1 spray each component (thrombin solution and fibrinogen solution) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods Larger wounds: (>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each |
|
Percent of change from baseline (i.e., Week 1 of each period) in the target wound area for each visit and Weeks 2-5 of each treatment period. |
| 4 weeks for each of 3 treatment periods |
| Persistence of healing | Persistence of healing as assessed by lack of blister/wound recurrence for healed wounds of each period (up to 40 weeks following enrollment) | 4 weeks for each of 3 treatment periods |
| Burning and stinging upon application | Burning and stinging upon application of HP802-247 or its Vehicle will be assessed at each visit of each period with the exception of the end of treatment visit (Weeks 1-4 of treatment period). Burning/stinging scale is a four point scale (i.e., none, mild, moderate, severe). | 4 weeks for each of 3 treatment periods |
| D030342 | Genetic Diseases, Inborn |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D012872 | Skin Diseases, Vesiculobullous |