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

Phase 2

Recruiting

• Conditions: Dystrophic Epidermolysis Bullosa
• Interventions: Biological: ALLO-ASC-SHEET; Other: Vehicle Control

• Registration Number: NCT05157958
• Lead Sponsor: Anterogen Co., Ltd.

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.

Study Timeline

• Study First Submitted: 2021-12-02
• Study First Submitted QC: 2021-12-02
• Study First Posted: 2021-12-15
• Study Start: 2023-05-01
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-19
• Last Update Posted: 2024-04-22
• Primary Completion: 2025-10-01
• Study Completion: 2026-04-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ALLO-ASC-SHEET	ALLO-ASC-SHEET	Allogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound
Conventional Therapy	Vehicle Control	Hydrogel Sheet Matching control

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events	First application to week 37	Incidence, severity, relationship of adverse event

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

University of Miami Dermatology Clinical Trials Unit; 🇺🇸 Miami, Florida, United States