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Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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ClinicalTrials.gov Identifier: NCT04227106
Recruitment Status : Completed
First Posted : January 13, 2020
Last Update Posted : December 5, 2022
Information provided by (Responsible Party):
Abeona Therapeutics, Inc

Brief Summary:
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Biological: EB-101 Phase 3

Detailed Description:

Recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare, severe inherited blistering skin disease caused by the absence of a protein known as type 7 collagen (C7). There is no approved treatment for RDEB. Only supportive care is currently possible.

This open-label, controlled study will evaluate the efficacy and safety of EB-101 for the treatment of large, chronic, RDEB wounds. The study intervention consists of one-time surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 10-15 participants. A single EB-101 sheet is able to provide healing to a wound area up to approximately 40cm2. Up to 6 (six) EB-101 sheets may be applied to each patient, depending on the area of existing wounds. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). Patient-reported outcomes and safety will also be collected throughout the study.

The primary analysis for efficacy will be assessed when all patients reach Week 24. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 26 post-treatment.

Upon completion of the study period, patients will be monitored annually as per standard of care for up to 15 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Actual Study Start Date : January 10, 2020
Actual Primary Completion Date : October 18, 2022
Actual Study Completion Date : October 18, 2022

Arm Intervention/treatment
Experimental: EB-101
One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds
Biological: EB-101
autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7
Other Name: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]

Primary Outcome Measures :
  1. Wound Healing [ Time Frame: 24 weeks post-treatment ]
    Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds

  2. Pain Reduction [ Time Frame: 24 weeks post-treatment ]
    Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Clinical diagnosis of RDEB;
  • Age 6 years or older, willing and able to give consent/assent;
  • If under the age of 18, guardian(s) is/are willing and able to give consent;
  • Positive expression of the non-collagenous region 1 of the type 7 collagen protein (NC1+) in the skin;
  • Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation that parents don't have any evidence of dominant disease);
  • At least 40 cm2 areas of chronically wounded area on the trunk and/or extremities suitable for EB-101 application (open erosions);
  • Able to undergo adequate anesthesia during EB-101 application;
  • Must have at least two matched, eligible wound sites (one pair);

    • Wound sites must:

      • Have an area ≥20 cm2,
      • Present for ≥6 months, and
      • Stage 2 wound;
  • Women of childbearing potential must use a reliable birth control method throughout the duration of the study and for 6 months post treatment;
  • Negative pregnancy test;
  • Must be on stable pain medication regimen at least 30 days prior to Screening

Exclusion Criteria:

  • Medical instability limiting ability to travel to the study site;
  • The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B or hepatitis C;
  • Evidence of immune response to C7 by indirect immunofluorescence (IIF);
  • Evidence of systemic infection;
  • Current evidence or a history of squamous cell carcinoma (SCC) in the area that will undergo EB-101 application;
  • Active drug or alcohol addiction;
  • Hypersensitivity to vancomycin or amikacin;
  • Receipt of chemical or biological study product for the specific treatment of RDEB in the past 3 months;
  • Positive pregnancy test or breast-feeding;
  • Clinically significant medical or laboratory abnormalities as determined by the Principal Investigator;
  • Inability to properly follow protocol and protect keratinocyte sheet sites, as determined by the Principal Investigator;
  • Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in RDEB patients. These abnormalities will not exclude a participant; and
  • Inability to culture participant's keratinocytes.

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): NCT04227106

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United States, California
Stanford University
Redwood City, California, United States, 94063
United States, Massachusetts
University of Massachusetts Medical School
Worcester, Massachusetts, United States, 01605
Sponsors and Collaborators
Abeona Therapeutics, Inc
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Principal Investigator: Jean Tang, MD Stanford University
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Responsible Party: Abeona Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT04227106    
Other Study ID Numbers: EB-101-CL-301
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: December 5, 2022
Last Verified: April 2022
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
Keywords provided by Abeona Therapeutics, Inc:
chronic wounds
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