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

Phase 3

Not yet recruiting

• Conditions: Epidermolysis Bullosa; Junctional Epidermolysis Bullosa; Epidermolysis Bullosa, Dystrophic; Epidermolysis Bullosa, Junctional; Skin Abnormalities; Congenital Abnormalities; Connective Tissue Disease; Collagen Diseases; Skin Diseases; Genetic Diseases, Inborn
• Interventions: Drug: Oleogel-S10

• Registration Number: NCT06917690
• Lead Sponsor: Chiesi Farmaceutici S.p.A.

Brief Summary

The goal of this clinical trial is to learn if Oleogel-S10 gel works to treat skin wounds from two types of inherited epidermolysis bullosa (EB): junctional EB (JEB) or dystrophic EB (DEB) in the Japanese population. Children and adults may participate. The trial also looks at the safety of Oleogel-S10 gel. The main questions it will answer are:

* Does Oleogel-S10 gel close wounds from JEB or DEB within 45 days or reduce the size of the wounds?

* Are there any medical problems when using Oleogel-S10 gel?

* How much of the drug ends up in your blood?

The study has 2 parts. In Part 1, participants will:

* Apply Oleogel-S10 gel to the EB wound dressings at least once every 4 days for 45 days.

* Visit the clinic once every 2 weeks for checkups and tests. If participants complete Part 1, they may participate in Part 2 if they choose to. In Part 2, participants will continue to receive Oleogel-S10 gel until the product is available to buy in Japan or until the trial is stopped by the company.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-03-17
• Status Verified: 2025-04
• Study Start: 2025-04
• Study First Submitted QC: 2025-04-02
• Last Update Submitted: 2025-04-02
• Study First Posted: 2025-04-08
• Last Update Posted: 2025-04-08
• Primary Completion: 2026-03
• Study Completion: 2029-09

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 6

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 oestrogen 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Oleogel-S10	Oleogel-S10	-

Primary Outcome Measures

Name	Time	Method
To evaluate the efficacy of Oleogel-S10 in Part I	Enrollment to 45 days	The number and percentage of closed wounds within Part I (up to Day 45).

Secondary Outcome Measures

Name	Time	Method
To continue to evaluate the efficacy of Oleogel-S10 up to Day 90 in Part II	Enrollment to 90 days	Evaluations will include the proportion of participants achieving complete wound closure of at least one target wound, the change in surface area of the wound, patient and clinician impression of improvement, frequency of dressing changes, and incidence of wound infections.
To evaluate the safety and tolerability of Oleogel-S10 in Part I and Part II	Enrollment through study completion, which is estimated for 2029	Safety will be assessed through monitoring of adverse events, physical examinations, clinical laboratory tests (blood and urine samples), and vital signs from baseline up to study completion.
To evaluate the extent of systemic exposure to betulin	Enrollment to 45 days	The concentration of betulin in blood at baseline and on Day 45.

Trial Locations

Locations (3)

Kurume University Hospital; 🇯🇵 Fukuoka, Japan

Niigata University Medical and Dental Hospital; 🇯🇵 Niigata, Japan

Hokkaido University Hospital; 🇯🇵 Sapporo-shi, Japan