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

Launched by CHIESI FARMACEUTICI S.P.A. · Apr 2, 2025

Keywords

ClinConnect Summary

This clinical trial is exploring the safety and effectiveness of a new gel called Oleogel-S10 for treating skin wounds in people with a rare condition called epidermolysis bullosa (EB). Specifically, the study focuses on two types of EB: junctional EB and dystrophic EB. Children and adults who are at least 21 days old may be eligible to participate if they have a confirmed diagnosis of one of these types and have at least three specific wounds meeting certain size and duration criteria. Participants will need to be of Japanese descent, with both parents and all four grandparents also being Japanese.

During the trial, participants will apply Oleogel-S10 to their wounds at least once every four days for 45 days and will have check-ups every two weeks with the study team. The researchers want to find out if the gel helps close wounds or reduces their size and to monitor any potential side effects. If participants complete the first part of the study, they may have the option to continue using the gel until it becomes available for purchase in Japan or until the study ends. It’s important to know that this study is not yet recruiting participants, so there will be more information to come on how to join.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female aged ≥ 21 days
• • 2. Confirmed diagnosis of either JEB or DEB
• • 3. Both biological parents and all 4 grandparents of Japanese descent
• 4. 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
• 5. A female subject must meet one of the following criteria:
• * If of childbearing potential, she must:
• • 1. Have a negative pregnancy test result at Screening and Baseline Visits, AND
• 2. 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)
• • 6. 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
• • 7. Subject and/or subject's legal representative is able and willing to follow all study procedures and instructions
• Exclusion Criteria:
• • 1. Hypersensitivity to Oleogel-S10 or any of its excipients
• • 2. Diagnosis of EB subtypes simplex or Kindler EB
• • 3. Receipt of systemic antibiotics for wound-related infections within 7 days prior to enrollment
• • 4. 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
• • 5. 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
• • 6. Receipt of systemic gene therapy for the treatment of inherited EB
• • 7. 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
• • 8. Receipt of topical gene therapy for the treatment of inherited EB (e.g., beremagene geperpavec) within 3 months prior to enrollment
• • 9. Receipt of a JACE® skin graft on any of the target wounds
• • 10. Current and/or former malignancy, including skin malignancies such as basal cell carcinoma and squamous cell carcinoma
• • 11. 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
• • 12. Enrollment in any other interventional study or treated with any other investigational drug for any disease within 4 weeks prior to enrollment
• • 13. 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