Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa

Ki vehet részt

• Informed Consent (ICF) form signed by the patient or patient's legal representative. Since all patients with be 12 years old or younger at study entry, signed assent from the patient's legal representative will be required
• Patient (or caretaker) must be willing to comply with all protocol requirements
• Diagnosis of Simplex, Recessive Dystrophic, or JEB-nH (intermediate) EB
• Patient must have 1 target wound (size 10 to 50 cm2)
• Patients must be between 28 days to 12 years at study entry
• Target wound must be present for 21 days or more
• Patients must have wound burden based on BSA of ≥5% at baseline

Ki nem vehet részt

• Use of any investigational drug or approved EB therapy within 30 days before enrollment
• Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment
• Use of systemic or topical steroidal therapy within 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids are allowed)
• Use of systemic antibiotics within 7 days before enrollment
• Selected target wound cannot have clinical evidence of local infection
• Current or former malignancy
• Patients who do not meet the entry criteria outlined above
• Participant is one of the following:; Not of childbearing potential (premenarchal or male/not in possession of a uterus); If of childbearing potential, is nonpregnant (negative urine pregnancy test results at Screening), nonlactating, and practicing 1 of the following medically acceptable methods of birth control:; Abstinence from heterosexual intercourse as a lifestyle choice; Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant's usual menstrual cycle period) before product administration; Intrauterine device. If male, is willing to use a highly effective method of contraception throughout the study period