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

• 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

Exclusion Criteria:

• 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.