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

Ki vehet részt

• Clinical diagnosis of DEB
• Age 12 months 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
• (This inclusion criterion was deleted as of Amendment 1.)
• Confirmation of DEB diagnosis (either dominant [DDEB] or recessive [RDEB] forms) by genetic testing
• 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
• On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline)
• Must have at least one wound site that meets all of the following criteria:; An area ≥20 cm2,; Present for ≥6 months, and; 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
• For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline
• Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit

Ki nem vehet részt

• Active drug or alcohol addiction
• Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application
• 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 systemic infection
• Current evidence or a history of SCC in the area that will undergo EB-101 application
• Hypersensitivity to vancomycin or amikacin
• Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient
• Medical instability limiting ability to travel to the study site or undergo EB-101 treatment
• (This exclusion criterion was deleted as of Amendment 3.)
• Breast-feeding
• Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI
• Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application
• Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study