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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03068780




Registration number
NCT03068780
Ethics application status
Date submitted
27/02/2017
Date registered
3/03/2017
Date last updated
15/07/2019

Titles & IDs
Public title
Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa
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
Secondary ID [1] 0 0
2016-002066-32
Secondary ID [2] 0 0
BEB-13
Universal Trial Number (UTN)
Trial acronym
EASE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Oleogel-S10
Treatment: Drugs - Placebo

Experimental: Oleogel-S10 -

Placebo Comparator: Placebo -


Treatment: Drugs: Oleogel-S10
10% birch bark extract in 90% sunflower oil

Treatment: Drugs: Placebo
Sunflower oil gel

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients with first complete closure of the EB target wound within 45 days of treatment - Proportion of patients with first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm² to 50 cm² in size aged =21 days and <9 months) within 45±7 days of treatment with Oleogel-S10 compared to placebo based on clinical assessment by the investigator (the wound will be rated as "closed" at first appearance of complete reepithelialisation without drainage confirmed by a second observation within the following week)
Timepoint [1] 0 0
45±7 days
Secondary outcome [1] 0 0
Time to first complete closure of the EB target wound as evidenced by clinical assessment until Day 90±7. - Time to first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm² to 50 cm² in size aged =21 days) within 90±7 days of treatment with Oleogel-S10 compared to placebo based on clinical assessment by the investigator (the wound will be rated as "closed" at first appearance of complete reepithelialisation without drainage confirmed by a second observation within the following week)
Timepoint [1] 0 0
90±7 days

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Sydney
Recruitment hospital [2] 0 0
Premier Specialists - Sydney
Recruitment hospital [3] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [4] 0 0
Murdoch Childrens Research Institute Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Sydney
Recruitment postcode(s) [2] 0 0
2217 - Sydney
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment postcode(s) [4] 0 0
3502 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
South Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
Argentina
State/province [10] 0 0
Buenos Aires
Country [11] 0 0
Austria
State/province [11] 0 0
Salzburg
Country [12] 0 0
Brazil
State/province [12] 0 0
Pernanbuco
Country [13] 0 0
Brazil
State/province [13] 0 0
Santa Catarina
Country [14] 0 0
Brazil
State/province [14] 0 0
São Paulo
Country [15] 0 0
Chile
State/province [15] 0 0
Santiago
Country [16] 0 0
Colombia
State/province [16] 0 0
DC
Country [17] 0 0
Croatia
State/province [17] 0 0
Zagreb
Country [18] 0 0
Czechia
State/province [18] 0 0
Brno
Country [19] 0 0
Denmark
State/province [19] 0 0
Aarhus
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
France
State/province [21] 0 0
Toulouse
Country [22] 0 0
Germany
State/province [22] 0 0
Freiburg im Breisgau
Country [23] 0 0
Germany
State/province [23] 0 0
Hannover
Country [24] 0 0
Greece
State/province [24] 0 0
Attiki
Country [25] 0 0
Hong Kong
State/province [25] 0 0
Hong Kong
Country [26] 0 0
Hungary
State/province [26] 0 0
Budapest
Country [27] 0 0
Ireland
State/province [27] 0 0
Dublin
Country [28] 0 0
Israel
State/province [28] 0 0
Tel Aviv
Country [29] 0 0
Italy
State/province [29] 0 0
Milan
Country [30] 0 0
Italy
State/province [30] 0 0
Roma
Country [31] 0 0
Romania
State/province [31] 0 0
Bucharest
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Moscow
Country [33] 0 0
Serbia
State/province [33] 0 0
Belgrade
Country [34] 0 0
Singapore
State/province [34] 0 0
Singapore
Country [35] 0 0
Spain
State/province [35] 0 0
Barcelona
Country [36] 0 0
Spain
State/province [36] 0 0
Madrid
Country [37] 0 0
Spain
State/province [37] 0 0
Sevilla
Country [38] 0 0
Switzerland
State/province [38] 0 0
Bern
Country [39] 0 0
Ukraine
State/province [39] 0 0
Kyiv
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Birmingham
Country [41] 0 0
United Kingdom
State/province [41] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amryt Research Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase III, Efficacy and Safety Study of Oleogel-S10 in Participants with Inherited
Epidermolysis Bullosa (EB).

EB is a rare group of genetic skin fragility disorders characterised by blistering of the
skin in response to minor injury. In most cases, onset of EB is at birth or shortly after.
All participants affected by any type of EB share the main characteristic of repeatedly
developing painful wounds that take days to months to heal. Current treatment of EB is
primarily preventative and supportive including protection from mechanical forces by avoiding
rubbing, early treatment of wounds to prevent infections, and protection of the wound with
adequate non-adhesive dressings to enable healing.

Oleogel-S10 was authorised in 2016 in the European Union for treatment of partial thickness
wounds in adults under the brand name Episalvan®. The active pharmaceutical ingredient in
Oleogel-S10 is a refined birch bark extract, quantified to 72 to 88% betulin.

This clinical study of Oleogel-S10 in patients with inherited EB has been initiated to
investigate whether Oleogel-S10 is effective for treatment of EB wounds and safe in the long
term use.

Oleogel-S10 will be compared to a vehicle gel placebo. The placebo is an identical looking
sunflower oil gel that does not contain any active substance. The participant will receive
either Oleogel-S10 or vehicle gel for a double-blind study phase of 90 days. The probability
that the participant will receive Oleogel-S10 is 50%, which means that they have a 1 in 2
chance of receiving Oleogel-S10. However, in the follow-up phase of the study all
participants will be treated with Oleogel S10 for a period of 24 months.

This clinical study will be performed in several countries; in total, about 250 participants
are expected to participate.
Trial website
https://clinicaltrials.gov/show/NCT03068780
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Johannes S Kern, MD PhD
Address 0 0
Melbourne Health
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Head of Clinical Development
Address 0 0
Country 0 0
Phone 0 0
+35315180200
Fax 0 0
Email 0 0
easestudy@amrytpharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03068780