A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa

Ki vehet részt

• Male or female aged ≥ 21 days
• Confirmed diagnosis of either JEB or DEB
• Both biological parents and all 4 grandparents of Japanese descent
• At least 3 EB wounds that meet the following criteria at the time of enrollment:; All are located outside of the anogenital region; All are distinguishable from any other wounds on the subject's body (separated by a minimum of 5 cm); At least one has been present for a minimum of 21 days but less than 9 months AND is 10 cm2 to 50 cm2 in size; At least two have been present for a minimum of 21 days but less than 9 months AND/OR are 10 cm2 to 50 cm2 in size
• A female subject must meet one of the following criteria:; If of childbearing potential, she must:; Have a negative pregnancy test result at Screening and Baseline Visits, AND; Agree to use one of the following highly reliable methods of contraception from the day of the informed consent signature until the day after the last Oleogel- S10 application. The following methods are acceptable:; Placement of an intrauterine device (IUD) or intrauterine releasing system (IUS); Combined (both estrogen and progestogen) hormonal contraception (oral) associated with inhibition of ovulation, supplemented with a barrier method (preferably male condom); Bilateral tubal occlusion; Sexual abstinence, defined as refraining from heterosexual intercourse during the entire study period; Partner vasectomy, provided that the partner is the sole sexual partner and has received medical verification of the surgical success; Be of non-childbearing potential, defined as one of the following:; Pre-menarche, OR; Post-menopausal (12 consecutive months of amenorrhea without an alternative medical cause), OR; Permanently sterile following hysterectomy, bilateral salpingectomy, or bilateral oophorectomy (supporting evidence required)
• Subject and/or subject's legal representative has been informed about the study, has read and understood the information provided, and has given written informed consent
• Subject and/or subject's legal representative is able and willing to follow all study procedures and instructions

Ki nem vehet részt

• Receipt of systemic antibiotics for wound-related infections within 7 days prior to enrollment
• Receipt of systemic or topical (on EB wounds) steroids within 30 days prior to enrollment Exceptions: Inhaled, ophthalmic, or mucosal applications, such as budesonide suspension for esophageal strictures
• Receipt of immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrollment Note: Dupilumab to manage pruritis is allowed if the subject has been on a stable dose for more than 3 months prior to baseline
• Receipt of systemic gene therapy for the treatment of inherited EB
• Receipt of short-acting stem cell therapy (infusion of Muse cells, allogeneic adipocyte-derived MSC sheet (topically applied), TEMCELL (allogeneic MSCs, subcutaneous injection)) within 6 months prior to study enrollment
• Receipt of topical gene therapy for the treatment of inherited EB (e.g., beremagene geperpavec) within 3 months prior to enrollment
• Enrollment in any other interventional study or treated with any other investigational drug for any disease within 4 weeks prior to enrollment
• Hypersensitivity to Oleogel-S10 or any of its excipients
• Current and/or former malignancy, including skin malignancies such as basal cell carcinoma and squamous cell carcinoma
• Diagnosis of EB subtypes simplex or Kindler EB
• Receipt of a JACE® skin graft on any of the target wounds
• Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until study participation ends
• Presence of any factor in the subject and/or legal representative that could interfere with study compliance, such as the ability to attend the scheduled study visits or to properly manage the dressing changes at home