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

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ClinicalTrials.gov Identifier: NCT05157958
Recruitment Status : Recruiting
First Posted : December 15, 2021
Last Update Posted : April 22, 2024
Sponsor:
Information provided by (Responsible Party):
Anterogen Co., Ltd.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Dystrophic Epidermolysis Bullosa Biological: ALLO-ASC-SHEET Other: Vehicle Control Phase 2

Detailed Description:
Primary efficacy endpoint will be assessed during and after 12th IP applications.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : May 1, 2023
Estimated Primary Completion Date : October 1, 2025
Estimated Study Completion Date : April 28, 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [ Time Frame: First application to week 37 ]
    Incidence, severity, relationship of adverse event


Secondary Outcome Measures :
  1. Proportion of complete wound closure [ Time Frame: 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


Eligibility Criteria
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Ages Eligible for Study:   4 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05157958


Contacts
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Contact: Fernanda Bellodi Schmidt 82-2-2014-0391 antstudy@anterogen.com

Locations
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United States, Florida
University of Miami Dermatology Clinical Trials Unit Recruiting
Miami, Florida, United States, 33125
Sponsors and Collaborators
Anterogen Co., Ltd.
More Information
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Responsible Party: Anterogen Co., Ltd.
ClinicalTrials.gov Identifier: NCT05157958    
Other Study ID Numbers: ALLO-ASC-DEB-201
First Posted: December 15, 2021    Key Record Dates
Last Update Posted: April 22, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases