The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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.
 
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.

Tracking Information
First Submitted Date  ICMJE December 2, 2021
First Posted Date  ICMJE December 15, 2021
Last Update Posted Date April 22, 2024
Actual Study Start Date  ICMJE May 1, 2023
Estimated Primary Completion Date October 1, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 15, 2022)		 Incidence of Treatment-Emergent Adverse Events [ Time Frame: First application to week 37 ]
Incidence, severity, relationship of adverse event
Original Primary Outcome Measures  ICMJE
 (submitted: December 2, 2021)		 Safety of ALLO-ASC-SHEET versus matching control [ Time Frame: First application to week 37 ]
Incidence, severity, relationship of adverse event
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 17, 2022)		 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
Original Secondary Outcome Measures  ICMJE
 (submitted: December 2, 2021)		 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
Official Title  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Dystrophic Epidermolysis Bullosa
Intervention  ICMJE
  • Biological: ALLO-ASC-SHEET
    Weekly administration
  • Other: Vehicle Control
    Weekly administration
Study Arms  ICMJE
  • Experimental: ALLO-ASC-SHEET

    Allogeneic mesenchymal stem cells

    Dressing for Dystrophic Epidermolysis Bullosa wound

    Intervention: Biological: ALLO-ASC-SHEET
  • Active Comparator: Conventional Therapy

    Hydrogel Sheet

    Matching control

    Intervention: Other: Vehicle Control
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 2, 2021)		 6
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 28, 2026
Estimated Primary Completion Date October 1, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Fernanda Bellodi Schmidt 82-2-2014-0391 antstudy@anterogen.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05157958
Other Study ID Numbers  ICMJE ALLO-ASC-DEB-201
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Anterogen Co., Ltd.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Anterogen Co., Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Anterogen Co., Ltd.
Verification Date April 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP