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

EB-101-CL-302

To evaluate and further characterize the safety of EB-101 (LZRSECol7A1 Engineered Autologous Epidermal Sheets \[LEAES\]) for the treatment of RDEB wounds in new and previously EB-101 treated patients 6 years and older.

2023-04-02

2024-08-01

2024-12-01

Recruiting

Early phase I

12

Inclusion Criteria: 1. Clinical diagnosis of RDEB 2. Age 6 years 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. Positive expression of NC1+ amino-terminal fragment of C7 in the skin; 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 and for 24 Weeks post treatment; 8. On stable pain medication regimen for at least 30 days prior to Screening; 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 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. Evidence or history of immune response to C7 by indirect immunofluorescence (IIF) or enzyme-linked immunosorbent assay; 4. Evidence of systemic infection; 5. Current evidence or a history of squamous cell carcinoma (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. Positive pregnancy test or breast-feeding; 10. Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the Principal Investigator; 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-biopsies or inability to manufacture patient's keratinocytes for use in EB-101 application.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

2023-09-21