EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB
About this trial
This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Chronic wounds
Eligibility Criteria
Inclusion Criteria: Clinical diagnosis of RDEB Age 6 years and older; Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent; Positive expression of NC1+ amino-terminal fragment of C7 in the skin; Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation that parents don't have any evidence of dominant disease); Able to undergo adequate anesthesia during EB-101 treatment; 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; On stable pain medication regimen for at least 30 days prior to Screening; Must have at least one wound site that meets all of the following criteria: An area ≥20 cm2 Present for ≥6 months 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: Medical instability limiting ability to travel to the study site or undergo EB-101 treatment; 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 or history of immune response to C7 by indirect immunofluorescence (IIF) or enzyme-linked immunosorbent assay; 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 investigational therapy for the specific treatment of RDEB in the 3 months prior to EB-101 application; Positive pregnancy test or breast-feeding; Inability to properly follow protocol assessments 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, and these abnormalities will not exclude a patient; Unwillingness or inability to provide 4-biopsies or inability to manufacture patient's keratinocytes for use in EB-101 application.
Sites / Locations
- Stanford UniversityRecruiting
- University of Massachusetts Medical SchoolRecruiting
Arms of the Study
Arm 1
Experimental
EB-101 Surgical application of RDEB wounds
New or Previously Treated RDEB Patients