Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
Study Details
Study Description
Brief Summary
After confirming eligibility, a single subject with two selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, one target lesion for each IP (one target lesion for ALLO-ASC-SHEET and the other target for Vehicle control), and which lesion to apply which IP will be determined randomly at the time of enrollment using IWRS.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
Primary efficacy endpoint will be assessed at 5 weeks after up to 12th IP applications.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ALLO-ASC-SHEET Allogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound |
Biological: ALLO-ASC-SHEET
Weekly administration
|
| Active Comparator: Conventional Therapy Hydrogel Sheet Matching control |
Other: Vehicle Control
Weekly administration
|
Outcome Measures
Primary Outcome Measures
- Incidence of Treatment-Emergent Adverse Events [First application to week 37]
Incidence, severity, relationship of adverse event
Secondary Outcome Measures
- Proportion of complete wound closure [First application to week 17]
Proportion of subject with target skin ulcer area meeting the definition of wound closure at 5 weeks after the completion of treatment period
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Anterogen Co., Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ALLO-ASC-DEB-201