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

Running, not enrolling · Phase 3

Conditions studied: Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB

In brief

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.

Key facts

Study ID

NCT05725018

Run by

Abeona Therapeutics, Inc

People needed

12

Starts

2023-04-02

Expected to finish

2025-09-30

Last updated by the study team

2025-06-27

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

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.
• (This inclusion criterion was deleted as of Amendment 1.)
• 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.
• (This exclusion criterion was deleted as of Amendment 3.)
• 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.

Where it is running

• Stanford University — Redwood City, California, United States
• University of Massachusetts Medical School — Worcester, Massachusetts, United States

Full record on ClinicalTrials.gov

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