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Trial record 4 of 19 for:    epidermolysis bullosa | Recruiting, Not yet recruiting, Available Studies

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

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ClinicalTrials.gov Identifier: NCT03068780
Recruitment Status : Recruiting
First Posted : March 3, 2017
Last Update Posted : January 9, 2018
Sponsor:
Information provided by (Responsible Party):

Study Description
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 164 participants are expected to participate.


Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: Oleogel-S10 Drug: Placebo Phase 3

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 164 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 29, 2017
Estimated Primary Completion Date : September 2018
Estimated Study Completion Date : September 2020


Arms and Interventions

Arm Intervention/treatment
Experimental: Oleogel-S10 Drug: Oleogel-S10
10% birch bark extract in 90% sunflower oil
Other Name: Episalvan
Placebo Comparator: Placebo Drug: Placebo
Sunflower oil gel
Other Name: Vehicle gel


Outcome Measures

Primary Outcome Measures :
  1. Proportion of patients with first complete closure of the EB target wound within 45 days of treatment [ Time Frame: 45±7 days ]
    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) 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)


Secondary Outcome Measures :
  1. Time to first complete closure of the EB target wound as evidenced by clinical assessment until Day 90±7. [ Time Frame: 90±7 days ]
    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)


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female patients with any subtype of inherited EB including EB simplex (EBS), junctional EB (JEB), dystrophic EB (DEB), and Kindler syndrome age ≥4 years,
  • Patients with an EB target wound (i.e., EB partial thickness wound of 10 cm² to 50 cm² in size aged ≥21 days),
  • 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:

  • 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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03068780


Contacts
Contact: Head of Clinical Development +35316694606 easestudy@amrytpharma.com
Contact: Medical Monitor, MD medicalmonitor@amrytpharma.com

Locations
Australia, New South Wales
Sydney Children's Hospital Recruiting
Sydney, New South Wales, Australia, 2031
Contact: Rebecca Saad    +61430484799    rebecca.saad@health.nsw.gov.au   
Principal Investigator: Orli Wargon, A/Prof         
Premier Specialists Recruiting
Sydney, New South Wales, Australia, 2217
Contact: Charmaine Peras    02 9598 5800    premierspecnurse@gmail.com   
Principal Investigator: Dédée F. Murrell, Prof.         
Australia, Victoria
The Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Annette Phemister    03 9342 4531    Annette.Phemister@mh.org.au   
Principal Investigator: Johannes Kern, MD         
Murdoch Childrens Research Institute Royal Children's Hospital Recruiting
Parkville, Victoria, Australia, 3502
Contact: Shannon Kokoszka    +61 3 9345 5570    Shannon.Kokoszka@rch.org.au   
Principal Investigator: Susan Robertson, Dr         
Austria
Universitaetsklinik fuer Dermatologie Recruiting
Salzburg, Austria, 5020
Contact: Sophie Kitzmueller    +43 57255-24601    s.kitzmueller@salk.at   
Principal Investigator: Martin Laimer, Prof.         
France
CHU de l'Archet Recruiting
Nice, France, 6200
Contact    +33 492039211    chiaverini.c@chu-nice.fr   
Principal Investigator: Christine Chiaverini, Dr.         
Hôpital Necker-Enfants Malades Recruiting
Paris, France, 75015
Contact: Bhia Wiam    +33 144494672      
Principal Investigator: Christine Bodemer, Prof.         
Germany
Medical Center University Freiburg Recruiting
Freiburg im Breisgau, Germany, 79104
Contact: Aurelia Winter    +49 76127067141    aurelia.winter@uniklinik-freiburg.de   
Principal Investigator: Franziska Schauer, Dr         
Kinder- und Jugendkrankenhaus AUF DER BULT Recruiting
Hannover, Germany, 30173
Contact: Aschemier, Dr.    +49 51181156611    aschemeier@hka.de   
Principal Investigator: Hagen Ott, Dr.         
Hautklinik Kiel, UKSH, Campus Kiel Recruiting
Kiel, Germany, 24105
Contact: Michaela Gertz    +49 4315971579    mgertz@dermatology.uni-kiel.de   
Principal Investigator: Regina Foelster-Holst, Prof.         
Greece
Hospital of Skin and Veneral Diseases "A. Syggros" Recruiting
Athens, Attiki, Greece, 16121
Contact: Ioanna Verroiou, Dr.    +30 2107265130    alkats.duoa@yahoo.gr   
Principal Investigator: Alexandra Katsarou-Katsari, Dr.         
Hong Kong
Prince of Wales Hospital, The Chinese University of Hong Kong Recruiting
Hong Kong, Hong Kong
Contact: Jasmine Yu    +852 3505 1124    jasminewsyu@gmail.com   
Principal Investigator: Ellis Hon Kam Lun, Prof.         
Ireland
St James Hospital Not yet recruiting
Dublin, Ireland, 8
Contact: Jane Richardson    +353 1 410 3903    JARICHAR@tcd.ie   
Principal Investigator: Fiona Browne, Dr.         
Our Ladys Childrens Hospital Not yet recruiting
Dublin, Ireland
Contact: Ann-Marie Day    +353 1 4096444    annmarie.day@ncrc.ie   
Principal Investigator: Fiona Browne, Dr.         
Israel
Tel Aviv Sourasky Medical Center Recruiting
Tel Aviv, Israel, 64239
Contact: Dvora Cohen    +972 36974287    elisp@tasmc.health.gov.il   
Principal Investigator: Eli Sprecher, Prof.         
Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Recruiting
Milan, Italy, 20122
Contact: Ilaria Coro    +39 0255032450    paola.marchisio@unimi.it   
Principal Investigator: Paola Marchisio, Prof.         
Bambino Gesù Children Hospital Recruiting
Roma, Italy, 00165
Contact: Alessandra Simonetti, Dr.    +39 0668592509    andrea.diociaiuti@opbg.net   
Principal Investigator: Andrea Diociaiuti, Dr.         
Istituto Dermopatico dell'Immacolata IDI-IRCCS Recruiting
Roma, Italy, 00167
Contact: Liliana Guerra    +39 0666462010    b.didona@idi.it   
Principal Investigator: Biagio Didona, Dr.         
Spain
Hospital Universitari de la Vall d'Hebron Recruiting
Barcelona, Spain, 8035
Contact: Pedro Barranco    +34 934893475      
Principal Investigator: Elena Arana, Dr.         
Hospital Universitario La Paz Recruiting
Madrid, Spain, 28046
Contact: Vega Mauleon    +34 912 071 876    vegamauleon@gmail.com   
Principal Investigator: Raul De Lucas, Dr         
Switzerland
Bern University Hospital Recruiting
Bern, Switzerland, 3010
Contact: Mark Wienand    +41 316326983    mark.wienand@insel.ch   
Principal Investigator: Carolina Gouveia, Dr.         
United Kingdom
Birmingham Children's Hospital NHS Trust Not yet recruiting
Birmingham, United Kingdom
Contact: Victoria Lynne    +44 1213339999    victoria.lynne@nhs.net   
Principal Investigator: Malobi Ogboli, Dr.         
Great Ormond Street hospital Not yet recruiting
London, United Kingdom, WC1N3JH
Contact: Lauren Holland    +44 2077626892    Lauren.Holland@gosh.nhs.uk   
Principal Investigator: Anna Martinez, Dr.         
Sponsors and Collaborators
Amryt Research Limited
Investigators
Principal Investigator: Johannes S Kern, MD PhD Melbourne Health
More Information

Responsible Party: Amryt Research Limited
ClinicalTrials.gov Identifier: NCT03068780     History of Changes
Other Study ID Numbers: BEB-13
2016-002066-32 ( EudraCT Number )
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: January 9, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous