EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Eligibility 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.
• 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.