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

NCT05157958 | Recruiting Phase 2 FDA Drug

• 1 other identifier: ALLO-ASC-DEB-201
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Dystrophic Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events

  Incidence, severity, relationship of adverse event

  First application to week 37

Secondary Outcomes (1)

• Proportion of complete wound closure

  First application to week 17

Study Arms (2)

ALLO-ASC-SHEET

EXPERIMENTAL

Allogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound

Biological: ALLO-ASC-SHEET

Conventional Therapy

ACTIVE COMPARATOR

Hydrogel Sheet Matching control

Other: Vehicle Control

Interventions

ALLO-ASC-SHEET BIOLOGICAL

Weekly administration

ALLO-ASC-SHEET

Vehicle Control OTHER

Weekly administration

Conventional Therapy

Eligibility Criteria

• Age: 4 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Anterogen Co., Ltd.: lead

Study Sites (1)

University of Miami Dermatology Clinical Trials Unit

Miami, Florida, 33125, United States

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Central Study Contacts

Fernanda Bellodi Schmidt

CONTACT

82-2-2014-0391; antstudy@anterogen.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2021
• First Posted: December 15, 2021
• Study Start: May 1, 2023
• Primary Completion: October 1, 2025
• Study Completion: April 28, 2026
• Last Updated: April 22, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)