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

NCT ID: NCT05157958

Last Updated: 2024-04-22

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-01
• Study Completion Date: 2026-04-28

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.

Conditions

Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

ALLO-ASC-SHEET

Allogeneic mesenchymal stem cells

Dressing for Dystrophic Epidermolysis Bullosa wound

Group Type: EXPERIMENTAL

ALLO-ASC-SHEET

Intervention Type: BIOLOGICAL

Weekly administration

Conventional Therapy

Hydrogel Sheet

Matching control

Group Type: ACTIVE_COMPARATOR

Vehicle Control

Intervention Type: OTHER

Weekly administration

Interventions

ALLO-ASC-SHEET

Weekly administration

Intervention Type: BIOLOGICAL

Vehicle Control

Weekly administration

Intervention Type: OTHER

Eligibility Criteria

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).

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Anterogen Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Miami Dermatology Clinical Trials Unit

Miami, Florida, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Fernanda Bellodi Schmidt

Role: CONTACT

antstudy@anterogen.com

82-2-2014-0391

Other Identifiers

ALLO-ASC-DEB-201

Identifier Type: -

Identifier Source: org_study_id