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

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Brief Title

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

Official Title

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 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.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Wound Healing


Condition

Epidermolysis Bullosa

Intervention

EB-101

Study Arms / Comparison Groups

 EB-101
Description:  One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

15

Start Date

January 10, 2020

Completion Date

September 2022

Primary Completion Date

September 2022

Eligibility Criteria

        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.
      

Gender

All

Ages

6 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Jean Tang, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04227106

Organization ID

EB-101-CL-301


Responsible Party

Sponsor

Study Sponsor

Abeona Therapeutics, Inc


Study Sponsor

Jean Tang, MD, Principal Investigator, Stanford University


Verification Date

April 2022