Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
Primary Purpose
Dystrophic Epidermolysis Bullosa
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ALLO-ASC-SHEET
Vehicle Control
Sponsored by
About this trial
This is an interventional treatment trial for Dystrophic Epidermolysis Bullosa
Eligibility 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.
Sites / Locations
- University of Miami Dermatology Clinical Trials UnitRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
ALLO-ASC-SHEET
Conventional Therapy
Arm Description
Allogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound
Hydrogel Sheet Matching control
Outcomes
Primary Outcome Measures
Incidence of Treatment-Emergent Adverse Events
Incidence, severity, relationship of adverse event
Secondary Outcome Measures
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05157958
Brief Title
Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
May 1, 2023 (Actual)
Primary Completion Date
November 1, 2024 (Anticipated)
Study Completion Date
February 28, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Anterogen Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystrophic Epidermolysis Bullosa
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
6 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ALLO-ASC-SHEET
Arm Type
Experimental
Arm Description
Allogeneic mesenchymal stem cells
Dressing for Dystrophic Epidermolysis Bullosa wound
Arm Title
Conventional Therapy
Arm Type
Active Comparator
Arm Description
Hydrogel Sheet
Matching control
Intervention Type
Biological
Intervention Name(s)
ALLO-ASC-SHEET
Intervention Description
Weekly administration
Intervention Type
Other
Intervention Name(s)
Vehicle Control
Intervention Description
Weekly administration
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events
Description
Incidence, severity, relationship of adverse event
Time Frame
First application to week 37
Secondary Outcome Measure Information:
Title
Proportion of complete wound closure
Description
Proportion of subject with target skin ulcer area meeting the definition of wound closure during and at the completion of 12 weeks treatment
Time Frame
First application to week 17
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Fernanda Bellodi Schmidt
Phone
82-2-2014-0391
Email
antstudy@anterogen.com
Facility Information:
Facility Name
University of Miami Dermatology Clinical Trials Unit
City
Miami
State/Province
Florida
ZIP/Postal Code
33125
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
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