Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa

Study Overview
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Brief Summary
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This is a phase I/II open-label study to evaluate the efficacy and safety of ALLO-ASC-DFU in patients with Dystrophic Epidermolysis Bullosa.

Detailed Description
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ALLO-ASC-DFU is a hydrogel sheet containing allogenic adipose-derived mesenchymal stem cells. Adipose-derived stem cells have anti-inflammatory effect and release growth factors such as vascular endothelial growth factor (VEGF) and hepatocyte growth factor (HGF), which can enhance wound healing and regeneration of new tissue, finally may provide an new option in treating a Dystrophic Epidermolysis Bullosa.

Official Title
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A Phase 1/2 Clinical Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa

Conditions
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Dystrophic Epidermolysis Bullosa

Intervention / Treatment
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* Other: Allogeneic mesenchymal stem cells
* Device: Polyurethene Film


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age : 10 60 Diagnosed with Dystrophic Epidermolysis Bullosa based on immunofluorescence test. Bullous skin lesion sized over 10 cm^2 Test negative for Serum β-HCG pregnancy test on screening, if the subject is fertile A subject who is willing to follow the protocol and provide a informed consent on screening, given that the information with respect to the clinical trial is provided. Exclusion Criteria: A subject with history of epidermoid carcinoma within a year from screening. A subject who requires antibiotics due to bacterial infection on skin. A subject who was dosed with oral steroid, over 0.5mg/kg a day for subjects under 18, or over 20mg in 2 weeks for subjects over 18, within 30 days prior to screening. A subject treated with radiotherapy or immunosuppressants, within 30 days prior to screening. A subject treated with steroids locally, within 30 days prior to screening. A subject with 2-times the maximum-standardized value of ALT, AST, ALP, bilirubin, total protein A subject with 2-times the maximum-standardized value of BUN, Creatinine A subject with Albumin below 2.0 g/dL. A subject with Hemoglobin below 6 g/dL (anemic). A subject with allergic response to bovine derived protein and fibrin glue. A subject administered with biologic agents or cell therapy, within 30 days prior to screening. A subject administered with stem cell treatment by IV or subcutaneously to the target wound, prior to the trial A subject who enrolled into another clinical trial, within 30 days prior to screening A subject with serious disease that can affect on clinical trial. A pregnant or breast-feeding subject. A subject with history of drug abuse within 1 year of clinical significance A subject who cannot proceed according to the protocol.

Ages Eligible for Study
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Minimum Age: 10 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: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ALLO-ASC-DFU<br> | Other: Allogeneic mesenchymal stem cells<br>* Dressing for Dystrophic Epidermolysis Bullosa wound.<br>* Other names: ALLO-ASC-DFU;|
| Active Comparator: Conventional Therapy<br> | Device: Polyurethene Film<br>* Dressing for Dystrophic Epidermolysis Bullosa wound.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area of re-epithelization |  | Follow up to 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of a target wound's re-epithelialization |  | Every time of visit for follow up to 8 weeks | 
| Time taken to re-epithelization |  | Over 8 weeks | 
| Safety assessed by clinically measured abnormality of laboratory tests and adverse events |  | Over 8 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Epidermolysis Bullosa, stem cell