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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT05157958
Recruitment Status : Not yet recruiting
First Posted : December 15, 2021
Last Update Posted : March 3, 2022
Information provided by (Responsible Party):
Anterogen Co., Ltd.

Brief Summary:
After confirming eligibility, a single subject with two selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, one target lesion for each IP (one target lesion for ALLO-ASC-SHEET and the other target for Vehicle control), and which lesion to apply which IP will be determined randomly at the time of enrollment using IWRS.

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 at 5 weeks after up to 12th IP applications.

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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
Estimated Study Start Date : March 1, 2022
Estimated Primary Completion Date : April 1, 2023
Estimated Study Completion Date : February 29, 2024

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

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 at 5 weeks after the completion of treatment period

Information from the National Library of Medicine

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

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.
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Responsible Party: Anterogen Co., Ltd. Identifier: NCT05157958    
Other Study ID Numbers: ALLO-ASC-DEB-201
First Posted: December 15, 2021    Key Record Dates
Last Update Posted: March 3, 2022
Last Verified: December 2021
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