Dystrophic Epidermolysis Bullosa
Conditions
Brief summary
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
Primary efficacy endpoint will be assessed during and after 12th IP applications.
Interventions
BIOLOGICALALLO-ASC-SHEET
Weekly administration
OTHERVehicle Control
Weekly administration
Sponsors
Anterogen Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
4 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Subject diagnosed as dystrophic epidermolysis bullosa confirmed by clinical criteria and one of the following: 1. 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. 2. COL7A1 mutational analysis: confirmation of COL7A1 genetic mutation. 2. 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): 1. 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). 2. Two skin ulcer lesions meeting criteria stated in 2a) should be sized 5-20 cm2 (inclusive) 3. 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) 4. 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
1. 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). 2. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | First application to week 37 | Incidence, severity, relationship of adverse event |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of complete wound closure | First application to week 17 | Proportion of subject with target skin ulcer area meeting the definition of wound closure during and at the completion of 12 weeks treatment |
Countries
United States
Contacts
Primary ContactFernanda Bellodi Schmidt
Outcome results
None listed