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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Dystrophic Epidermolysis Bullosa
• Intervention / Treatment: Biological: ALLO-ASC-SHEET; Other: Vehicle Control

Detailed Description

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

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Fernanda Bellodi Schmidt; Phone Number: 82-2-2014-0391; Email: antstudy@anterogen.com

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33125
      • Recruiting
      • University of Miami Dermatology Clinical Trials Unit

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 58 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / 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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Anterogen Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2023
• Primary Completion (Estimated): October 1, 2025
• Study Completion (Estimated): April 28, 2026

Study Registration Dates

• First Submitted: December 2, 2021
• First Submitted That Met QC Criteria: December 2, 2021
• First Posted (Actual): December 15, 2021

Study Record Updates

• Last Update Posted (Actual): April 22, 2024
• Last Update Submitted That Met QC Criteria: April 19, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Collagen Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica
• Other Study ID Numbers: ALLO-ASC-DEB-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No