VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

EB-101-CL-301

The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).

2020-01-10

2022-10-18

2022-10-18

Completed

Early phase I

11

Inclusion Criteria: * 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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 11, 'type': 'ACTUAL'}}

2022-12-05