Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa

Study Overview
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Brief Summary
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After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which lesion to apply which IP treatment will be determined randomly at the time of enrollment using pre-designed block randomization scheme.

Detailed Description
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Primary efficacy endpoint will be assessed during and after 12th IP applications.

Official Title
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Double Blind, Randomized, Phase II Clinical Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET Versus Vehicle Control in Dystrophic Epidermolysis Bullosa (DEB) Patients

Conditions
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Dystrophic Epidermolysis Bullosa

Intervention / Treatment
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* Biological: ALLO-ASC-SHEET
* Other: Vehicle Control


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Subject diagnosed as dystrophic epidermolysis bullosa confirmed by clinical criteria and one of the following: Immunostaining test: patients who have reduced or no type 7 collagen in staining degree of immunofluorescence. Other antigens (laminin-332, type 17 collagen, plectin, integrin α6β4, type 5 and type 14 keratin, etc.) are normal in immune-staining. COL7A1 mutational analysis: confirmation of COL7A1 genetic mutation. Subject with skin ulcer lesions of dystrophic epidermolysis bullosa meet the following criteria, on the screening start day (Visit 1) and treatment start day (enrollment day) (Visit 3): Subject has two skin ulcer lesions judged as comparable to compare the safety and efficacy by investigator during screening period and prior to the IP application (enrollment day). Two skin ulcer lesions meeting criteria stated in 2a) should be sized 5-20 cm2 (inclusive) Subject who has two comparable target skin ulcer lesions, and each lesions with a change of size equal to or less than ±50% at treatment day (Visit 3) compared to that of screening day (Visit 1) Subject who has no clinical evidence of infection related signs/symptoms, or visible necrosis in the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied). Key Exclusion Criteria: Subject who requires antibiotics due to bacterial infection on skin of the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied). Female subjects: pregnant woman (indicated by serum hCG test result at screening), breast-feeding patient, all sexually active patient, with child bearing potential in case of female*, who is not willing to contracept** during the clinical trial.

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ALLO-ASC-SHEET<br>Allogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound | Biological: ALLO-ASC-SHEET<br>* Weekly administration<br>|
| Active Comparator: Conventional Therapy<br>Hydrogel Sheet Matching control | Other: Vehicle Control<br>* Weekly administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events | Incidence, severity, relationship of adverse event | First application to week 37 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of complete wound closure | Proportion of subject with target skin ulcer area meeting the definition of wound closure during and at the completion of 12 weeks treatment | First application to week 17 |