Treatment for chronic wounds in patients with Recessive Dystrophic Epidermolysis Bullosa

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

This study is testing a new treatment using gene-corrected skin cells to see if it can help heal large chronic wounds in people with Recessive Dystrophic Epidermolysis Bullosa.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 12 Months and up
• Sex: All
• Sponsor: Abeona Therapeutics, Inc (industry)
• Locations: 2 sites (Redwood City, California and 1 other locations)
• Trial ID: NCT05725018 on ClinicalTrials.gov

What this trial studies

This Phase 3b clinical trial evaluates the safety of EB-101, a gene-corrected keratinocyte treatment, for large chronic wounds in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). The study involves a one-time surgical application of autologous keratinocyte sheets that express type VII collagen. Patients aged 12 months and older will be monitored for 24 weeks post-treatment, with evaluations at various intervals to assess wound healing and safety. The trial aims to gather data from approximately 10-12 participants across multiple centers.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 months and older with a clinical diagnosis of RDEB and specific genetic mutations.

Not a fit: Patients with wounds that do not meet the size or duration criteria, or those with dominant disease patterns, may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly improve wound healing and quality of life for patients with RDEB.

How similar studies have performed: While this approach is innovative, similar gene therapy strategies have shown promise in other conditions, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Clinical diagnosis of RDEB.
2. Age 12 months and older.
3. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
4. (This inclusion criterion was deleted as of Amendment 1.)
5. Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation that parents don't have any evidence of dominant disease).
6. Able to undergo adequate anesthesia during EB-101 treatment.
7. 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.
8. On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
9. Must have at least one wound site that meets all of the following criteria:

   1. An area ≥20 cm2,
   2. Present for ≥6 months, and
   3. 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.

Exclusion Criteria:

1. Medical instability limiting ability to travel to the study site or undergo EB 101 treatment.
2. 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.
3. (This exclusion criterion was deleted as of Amendment 3.)
4. Evidence of systemic infection.
5. Current evidence or a history of SCC in the area that will undergo EB-101 application.
6. Active drug or alcohol addiction.
7. Hypersensitivity to vancomycin or amikacin.
8. Receipt of chemical or biological investigational therapy for the specific treatment of RDEB in the 3 months prior to EB-101 application.
9. Breast-feeding.
10. Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI.
11. 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, and these abnormalities will not exclude a patient.
12. Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application.
13. Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.

Where this trial is running

Redwood City, California and 1 other locations

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

Study contacts

• Study coordinator: Abeona Therapeutics

Conditions: Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB, Chronic wounds