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

Dystrophic Epidermolysis Bullosa Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05157958
• Other study ID #: ALLO-ASC-DEB-201
• Status: Recruiting
• Phase: Phase 2
• Start date: May 1, 2023
• Est. completion date: April 28, 2026

Study information

• Verified date: April 2024
• Source: Anterogen Co., Ltd.
• Contact: Fernanda Bellodi Schmidt
• Phone: 82-2-2014-0391
• Email: antstudy[@]anterogen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 6
• Est. completion date: April 28, 2026
• Est. primary completion date: October 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 60 Years

Eligibility

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 a6ß4, 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.

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

Intervention

Biological:
• ALLO-ASC-SHEET
Weekly administration

Other:
• Vehicle Control
Weekly administration

Locations

Country	Name	City	State
United States	University of Miami Dermatology Clinical Trials Unit	Miami	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Anterogen Co., Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events	Incidence, severity, relationship of adverse event	First application to week 37
Secondary	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