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

Primary Purpose

Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
EB-101
Sponsored by
Abeona Therapeutics, Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring chronic wounds

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Stanford University
  • University of Massachusetts Medical School

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EB-101

Arm Description

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

Outcomes

Primary Outcome Measures

Wound Healing
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
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

Secondary Outcome Measures

Full Information

First Posted
January 10, 2020
Last Updated
December 2, 2022
Sponsor
Abeona Therapeutics, Inc
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1. Study Identification

Unique Protocol Identification Number
NCT04227106
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)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2022
Overall Recruitment Status
Completed
Study Start Date
January 10, 2020 (Actual)
Primary Completion Date
October 18, 2022 (Actual)
Study Completion Date
October 18, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Abeona Therapeutics, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Keywords
chronic wounds

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
EB-101
Arm Type
Experimental
Arm Description
One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds
Intervention Type
Biological
Intervention Name(s)
EB-101
Other Intervention Name(s)
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]
Intervention Description
autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7
Primary Outcome Measure Information:
Title
Wound Healing
Description
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
Time Frame
24 weeks post-treatment
Title
Pain Reduction
Description
Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
Time Frame
24 weeks post-treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jean Tang, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
University of Massachusetts Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01605
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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