A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

NCT05725018 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: EB-101-CL-302
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.

Conditions

Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; RDEB

Keywords

Chronic wounds

Outcome Measures

Primary Outcomes (5)

• Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

  The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.

  6 months

• Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)

  The number of patients and wounds that have an infection or any related adverse event (AE).

  6 months

• Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )

  The number of patients and wounds that result in hospitalization (SAE).

  6 months

• Safety Endpoint (Incidence of squamous cell carcinoma)

  The incidence of squamous cell carcinoma (SCC).

  6 months

• Safety Endpoint RCR status

  Replication-competent retrovirus (RCR) status.

  6 months

Other Outcomes (8)

• Proportion of RDEB wounds with ≥50%, ≥75%, and complete

  6 months

• Extent of pain reduction assessed by the Wong-Baker FACES scale

  6 months

• Longitudinal change in scores of Worst Itch Numeric Rating Scale

  6 months

Study Arms (1)

EB-101 Surgical application of RDEB wounds

EXPERIMENTAL

New or Previously Treated RDEB Patients

Biological: EB-101 Surgical application of RDEB wounds

Interventions

EB-101 Surgical application of 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 Names: • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets \[LEAES\]

Also known as: pz-cel

EB-101 Surgical application of RDEB wounds

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of DEB.
• Age 12 months and older.
• Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
• Confirmation of DEB diagnosis (either dominant \[DDEB\] or recessive \[RDEB\] forms) by genetic testing.
• Able to undergo adequate anesthesia during EB-101 treatment.
• All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study.
• On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
• Must have at least one wound site that meets all of the following criteria:
• An area ≥20 cm2,
• Present for ≥6 months, and
• Stage 2 wound defined as an open skin wound with partial thickness loss of dermis that has not extended through the dermis into subcutaneous tissue.
• For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline.
• Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit.

You may not qualify if:

• Medical instability limiting ability to travel to the study site or undergo EB-101 treatment.
• 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 systemic infection.
• Current evidence or a history of 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 investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application.
• Breast-feeding.
• Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI.
• Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient.
• Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application.
• Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.

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

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

• Angela Iheanacho, MS

  Abeona Therapeutics, Inc

  STUDY DIRECTOR

• Sarah Abdelwahab, MD

  Abeona Therapeutics, Inc

  STUDY DIRECTOR

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 23, 2023
• First Posted: February 13, 2023
• Study Start: April 2, 2023
• Primary Completion: September 30, 2025
• Study Completion: September 30, 2025
• Last Updated: June 27, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)