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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03068780
Ethics application status
Date submitted
27/02/2017
Date registered
3/03/2017
Date last updated
20/07/2023

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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Oleogel-S10
Treatment: Drugs - Control gel

Experimental: Oleogel-S10 -

Placebo comparator: Control Gel -


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

Treatment: Drugs: Control gel
Sunflower oil, Cera flava/yellow wax, and Carnauba wax (matched Oleogel-S10 in terms of texture and visual appearance)
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
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 (D90) or End of Double-blind Phase (EDBP)
Timepoint [1] 0 0
90±7 days
Secondary outcome [2] 0 0
Proportion of Patients With First Complete Closure of the EB Target Wound at D90 or EDBP Based on Clinical Assessment by the Investigator Until D90 or EDBP
Timepoint [2] 0 0
90±7 days
Secondary outcome [3] 0 0
The Incidence of EB Target Wound Infection Between Baseline (DBP D0) and D90 or EDBP as Evidenced by Adverse Events (AEs) and/or Use of Topical and/or Systemic Antibiotics (Related to Wound Infection)
Timepoint [3] 0 0
90±7 days
Secondary outcome [4] 0 0
The Maximum Severity of EB Target Wound Infection Between Baseline (DBP D0) and D90 or EDBP as Evidenced by AEs
Timepoint [4] 0 0
90±7 days
Secondary outcome [5] 0 0
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 or EDBP
Timepoint [5] 0 0
90±7 days
Secondary outcome [6] 0 0
Change From Baseline (DBP D0) in Itching Using the 'Itch Man Scale' in Patients = 4 Years and up to 13 Years of Age Before Wound Dressing Changes at D90 or EDBP
Timepoint [6] 0 0
90±7 days
Secondary outcome [7] 0 0
Change From Baseline (DBP D0) in Itching Using the 'Leuven Itch Scale' in Patients = 14 Years of Age Before Wound Dressing Changes at D90 or End of Double Blind Phase (EDBP)
Timepoint [7] 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 EB 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
21 Days
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patient has EB simplex
* EB target wound that is =9 months old or has 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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/03/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
27/05/2022
Sample size
Target
Accrual to date
Final
223
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
Arizona
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
Czechia
State/province [17] 0 0
Brno
Country [18] 0 0
Denmark
State/province [18] 0 0
Aarhus
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
France
State/province [20] 0 0
Toulouse
Country [21] 0 0
Georgia
State/province [21] 0 0
Tbilisi
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 was 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.

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 carried out to investigate whether Oleogel-S10 is effective for treatment of EB wounds and safe for long-term use.

Oleogel-S10 was compared to a control gel. The control gel matched Oleogel-S10 in terms of texture and visual appearance to allow for double-blinding. The packaging for Oleogel-S10 gel and the control gel were identical. The participant received either Oleogel-S10 or control gel for a double-blind study phase of 90 days. The probability that the participant received Oleogel-S10 was 50%, which means that they had a 1 in 2 chance of receiving Oleogel-S10. However, in the follow-up phase of the study all participants were treated with Oleogel-S10 for a period of 24 months.

This clinical study was performed at 49 study sites in 26 countries (Argentina, Australia, Austria, Brazil, Chile, Colombia, Czech Republic, Denmark, France, Georgia, Germany, Greece, Hong Kong \[China\], Hungary, Ireland, Israel, Italy, Romania, Russia, Serbia, Singapore, Spain, Switzerland, Ukraine, United Kingdom, and the United States); 223 participants participated in total.
Trial website
https://clinicaltrials.gov/study/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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

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