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

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: NCT04227106
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : January 13, 2020
Information provided by (Responsible Party):
Abeona Therapeutics, Inc

Tracking Information
First Submitted Date  ICMJE January 10, 2020
First Posted Date  ICMJE January 13, 2020
Last Update Posted Date January 13, 2020
Actual Study Start Date  ICMJE January 10, 2020
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 10, 2020)
Wound Healing [ Time Frame: 12 weeks post-treatment ]
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated intra-participant wound sites
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Official Title  ICMJE VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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).
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 approximately 30 autologous, gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 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. Treatment effect will be measured as wound healing at 3 months, with follow up to 6 months post treatment, comparing treated versus matched untreated wound sites. Wound healing will be adjudicated by an independent committee not involved in the study conduct. 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 12. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 24 post-treatment.

Upon completion of the 6-month study period, patients will be monitored annually as per standard of care for up to 15 years.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Epidermolysis Bullosa
  • Recessive Dystrophic Epidermolysis Bullosa
Intervention  ICMJE 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]
Study Arms  ICMJE Experimental: EB-101
One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds
Intervention: Biological: EB-101
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 10, 2020)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2021
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Abeona Clinical Operations +1-646-813-4701
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04227106
Other Study ID Numbers  ICMJE EB-101-CL-301
Has Data Monitoring Committee Yes
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
Responsible Party Abeona Therapeutics, Inc
Study Sponsor  ICMJE Abeona Therapeutics, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jean Tang, MD Stanford University
PRS Account Abeona Therapeutics, Inc
Verification Date December 2019

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