Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa

• Register: ClinicalTrials.gov
• Last refreshed on: 10 October 2022
• Main ID: NCT03068780
• Date of registration: 27/02/2017
• Prospective Registration: Yes
• Primary sponsor: Amryt Research Limited
• Public title: Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa EASE
• Scientific title: Double Blind, Randomised, Vehicle Controlled, Phase III, Efficacy and Safety Study With 24-month Open-label Follow up of Oleogel-S10 in Patients With Inherited Epidermolysis Bullosa
• Date of first enrolment: March 29, 2017
• Target sample size: 223
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03068780
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Chile, Colombia, Croatia, Czechia, Denmark, France, Georgia, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Romania, Russian Federation, Serbia, Singapore, Spain, Switzerland, Ukraine, United Kingdom, United States

Contacts

• Name: Johannes S Kern, MD PhD
• Affiliation: Melbourne Health

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male and female patients with the following subtypes of inherited EB: junctional EB
(JEB), dystrophic EB (DEB), and Kindler syndrome aged =21 days,

- Patients with an EB target wound (i.e., EB partial thickness wound of 10 cm² to 50 cm²
in size aged =21 days and <9 months),

- Patient and/or his/her legal representative has/have been informed, has/have read and
understood the patient information/informed consent form, and has/have given written
informed consent,

- Patient and/or his/her legal representative must be able and willing to follow study
procedures and instructions.

Exclusion Criteria:

- Patient has EB simplex

- EB target wound with clinical signs of local infection,

- Use of systemic antibiotics for wound-related infections within 7 days prior to
enrolment,

- Administration of systemic or topical steroids (except for inhaled, ophthalmic or
topical applications, such as budesonide suspension for oesophageal strictures [e.g.,
Pulmicort respules® 0.25 mg/2 mL or 0.5 mg/2 mL]) within 30 days before enrolment,

- Immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrolment,

- Patient has undergone stem cell transplant or gene therapy for the treatment of
inherited EB,

- Current and/or former malignancy including basal cell carcinomas and squamous cell
carcinomas,

- Enrolment in any interventional study or treated with any investigational drug for any
disease within 4 weeks prior to study entry,

- Factors present in the patient and/or his/her legal representative that could
interfere with study compliance such as inability to attend scheduled study visits or
compliance with home dressing changes,

- Pregnant or nursing women and women of childbearing potential including postmenarchal
female adolescents not willing to use an effective form of birth control with failure
rates <1% per year (e.g., implant, injectable, combined oral contraceptive,
intrauterine contraceptive device, sexual abstinence, vasectomised partner) during
participation in the study (and at least 3 months thereafter),

- Patient is a member of the investigational team or his/her immediate family,

- Patient lives in the same household as a study participant.



Age minimum: 21 Days
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa

Intervention(s)

Drug: Control gel

Drug: Oleogel-S10

Primary Outcome(s)

Proportion of patients with first complete closure of the EB target wound within 45 days of treatment [Time Frame: 45±7 days]

Secondary Outcome(s)

The maximum severity of wound infection between baseline (DBP D0) and D90±7 as evidenced by AEs and/or use of topical and/or systemic antibiotics (related to wound infection) [Time Frame: 90±7 days]

Time to first complete closure of the EB target wound as evidenced by clinical assessment until EDBP (D90±7) [Time Frame: 90±7 days]

Change from baseline (DBP D0) in total body wound burden as evidenced by clinical assessment using Section I (assessment of the skin except for the anogenital region) of the 'EB Disease Activity and Scarring Index' (EBDASI) at D90±7 [Time Frame: 90±7 days]

Change from baseline (DBP D0) in itching using the 'Itch Man Scale' in patients = 4 years and up to 13 years of age and the 'Leuven Itch Scale' in patients = 14 years of age before wound dressing changes at D90±7 [Time Frame: 90±7 days]

Proportion of patients with first complete closure of the EB target wound at D90±7 based on clinical assessment by the investigator until Day 90±7. [Time Frame: 90±7 days]

The incidence of wound infection between baseline (DBP D0) and D90±7 as evidenced by AEs and/or use of topical and/or systemic antibiotics (related to wound infection) [Time Frame: 90±7 days]

Secondary ID(s)

BEB-13

2016-002066-32

Source(s) of Monetary Support

Please refer to primary and secondary sponsors