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

NCT04227106 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: EB-101-CL-301
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 2

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).

Conditions

Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa

Keywords

chronic wounds

Outcome Measures

Primary Outcomes (2)

• Wound Healing

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

  24 weeks post-treatment

• 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

  24 weeks post-treatment

Study Arms (1)

EB-101

EXPERIMENTAL

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

Biological: EB-101

Interventions

EB-101 BIOLOGICAL

autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7

Also known as: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]

EB-101

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Abeona Therapeutics, Inc: lead

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01605, United States

Location

Related Publications (1)

• Tang JY, Marinkovich MP, Wiss K, McCarthy D, Truesdale A, Chiou AS, Eid E, McIntyre JK, Bailey I, Furukawa LK, Gorell ES, Harris N, Khosla RK, Peter Lorenz H, Lu Y, Nazaroff J, Grachev ID, Moore AJ. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. Lancet. 2025 Jul 12;406(10499):163-173. doi: 10.1016/S0140-6736(25)00778-0. Epub 2025 Jun 23.

  PMID: 40570869 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases

Study Officials

• Jean Tang, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2020
• First Posted: January 13, 2020
• Study Start: January 10, 2020
• Primary Completion: October 18, 2022
• Study Completion: October 18, 2022
• Last Updated: December 5, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)