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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03068780
Recruitment Status : Recruiting
First Posted : March 3, 2017
Last Update Posted : October 3, 2019
Sponsor:
Information provided by (Responsible Party):
Amryt Pharma ( Amryt Research Limited )

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.


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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 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 : December 2019
Estimated Study Completion Date : February 2022


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




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 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)


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)



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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
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Contact: Head of Clinical Development +35315180200 easestudy@amrytpharma.com
Contact: Medical Monitor, MD medicalmonitor@amrytpharma.com

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Locations
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United States, Arizona
Phoenix Children's Hospital Not yet recruiting
Phoenix, Arizona, United States, 85016
Contact: Kellie Badger    602-933-2402    kbadger@phoenixchildrens.com   
Principal Investigator: Harper Price, Dr         
United States, California
Encino Research Center Recruiting
Encino, California, United States, 91436
Contact: Sarah Emert    818-788-5060    sarah@drraoof.com   
Principal Investigator: Joseph Raoof, Dr.         
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Kathleen Peoples    720-777-4708    Kathleen.Peoples@childrenscolorado.org   
Principal Investigator: Anna Bruckner, Prof         
United States, Florida
Amjad Plastic Research Recruiting
Miami, Florida, United States, 33144
Contact: Xoana Zampieri    305-267-8885    doctor@driamjad.com   
Principal Investigator: Ibrahim Amjad, Dr.         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Amy Hanson    612-626-4424    amhanson@umn.edu   
Principal Investigator: Kristen Hook, Dr         
United States, New York
Stony Brook University Hospital Recruiting
Stony Brook, New York, United States, 11790
Contact: Jennifer Intravaia    631-638-2328    Jennifer.Intravaia@stonybrookmedicine.edu   
Principal Investigator: Jordan Slutsky, Dr         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Contact: Bret Augsburger       Bret.Augsburger@cchmc.org   
Principal Investigator: Anne Lucky, Dr.         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Lisa Catanoso    215-590-0862    catanosol@email.chop.edu   
Principal Investigator: Leslie Castelo-Soccio, Dr         
Thomas Jefferson University Hospital Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Diana Clarkson    215-503-7031    Diana.Clarkson@jefferson.edu   
Principal Investigator: Bahar Dasgeb, Dr.         
United States, South Carolina
Medical University of South Carolina Not yet recruiting
Charleston, South Carolina, United States, 29425
Contact    843-792-9157    winelee@musc.edu   
Principal Investigator: Lara Wine-Lee, Dr.         
United States, Texas
Texas Dermatology and Laser Specialists Recruiting
San Antonio, Texas, United States, 78218
Contact: Gabriel Leal    210-660-1157    gabe@texasdls.com   
Principal Investigator: John Browning, Dr         
Argentina
Consutorios Medicos (Instituto de Neumonologia y Dermatologia) Recruiting
Buenos Aires, Argentina, 1425
Contact: Paula Luna    +54 9 11 4540 4644    paulacarolinaluna@gmail.com   
Principal Investigator: Margarita Larralde, Dr.         
Centro Médico Dra. De Salvo Recruiting
Buenos Aires, Argentina, 1426
Contact: Alexis Doreski    +54 12 4781-5331    doreski@fundacionrespirar.org   
Principal Investigator: Maria Florencia Fernandez         
Centro de investigaciones Metabolicas, CINME Recruiting
Buenos Aires, Argentina, C1056ABJ
Contact: Maria Laura Galimberti    +54 11 5031 9914    maria.galimberti@hospitalitaliano.org.ar   
Principal Investigator: Ianina Massimo         
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, A/Prof         
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.         
Brazil
IMIP Recruiting
Recife, Pernanbuco, Brazil, 50070550
Contact: Renata Cavalcanti Cauás, Dr.    +55 81 2122 4792    renatacauas@gmail.com   
Principal Investigator: Matilde Carrera, Dr.         
Universidade Regional de Blumenau Recruiting
Blumenau, Santa Catarina, Brazil, 89020400
Contact    +55 47 99911-9905    jeanine.magno@terra.com.br   
Principal Investigator: Jeanine Magno Frantz, Dr.         
Instituto Da Crianca HCFMUSP Recruiting
São Paulo, Brazil, 05403.000
Contact: Ivete Zoboli, Dr.    +55 11 2661 8676    ivete.zoboli@hc.fm.usp.br   
Principal Investigator: Silvia Maria Barbosa, Dr.         
Chile
Fundacion Debra Chile Recruiting
Santiago, Chile, 7760099
Contact: Ignacia Fuentes    +56 9-9310 8821    ignacia.fuentesbustos@gmail.com   
Principal Investigator: Francis Palisson, Dr.         
Colombia
Hospital De San Jose Recruiting
Bogotá, DC, Colombia
Contact: Elda Carolina Pena    +57 1 3538000    ecmelo1@fucsalud.edu.co   
Principal Investigator: Mauricio Torres, Dr.         
Croatia
University Hospital Center Zagreb Recruiting
Zagreb, Croatia, 10000
Contact: Slobodna Murat Susic    +385 012368 915    branka.marinovic@kbc-zagreb.hr   
Principal Investigator: Branka Marinovic, Prof. Dr.         
Czechia
University Hospital Brno, Children´s Hospital Recruiting
Brno, Czechia, 61500
Contact    +42 532 234 521    buckova.hana@fnbrno.cz   
Principal Investigator: Hana Buckova, Dr.         
Denmark
Aarhus University Hospital Recruiting
Aarhus, Denmark, 8000
Contact: Anne-Mette Thorup, Dr.       metsom@rm.dk   
Principal Investigator: Mette Sommerlund, Dr.         
France
Hôpital Necker-Enfants Malades Recruiting
Paris, France, 75015
Contact: Bhia Wiam    +33 144494672    wiam.bhia@aphp.fr   
Principal Investigator: Christine Bodemer, Prof.         
CHU Toulouse - Hospital Larrey Recruiting
Toulouse, France, 31059
Contact: Margot Merlos    +33 567771876    merlos.m@chu-toulouse.fr   
Principal Investigator: Juliette Mazereeuw-Hautier, Dr.         
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.         
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.         
Hungary
Semmelweis University, Faculty of Medicine Recruiting
Budapest, Hungary, 1085
Contact: Sillo Palma, Dr.    +36 20 825 8878    sillo.palma@med.semmelweis-univ.hu   
Principal Investigator: Miklos Sardy, Prof.         
Ireland
Our Ladys Childrens Hospital Recruiting
Dublin, Ireland
Contact: Hilari Howlett    +353 1 4096444    hilari.howlett@ncrc.ie   
Principal Investigator: Fiona Browne, Dr.         
Israel
Tel Aviv Sourasky Medical Center Recruiting
Tel Aviv, Israel, 64239
Contact: Orna Daga    +972 36973768    ornad@tlvmc.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.         
Romania
Centrul Medical Sanador Recruiting
Bucharest, Romania, 011025
Contact: Alina Suru, Dr.       alina_pirvu@yahoo.com   
Principal Investigator: Carmen Maria Salavastru, Prof.         
Russian Federation
State Scientific Center for Dermatovenerology and Cosmetology Recruiting
Moscow, Russian Federation, 107076
Contact: Ekaterina Monchakovskaya    +7 499 785 2015    monchakovskaya@cnikvi.ru   
Principal Investigator: Alexey Kubanov, Dr.         
Scientific Center of Children's Health Recruiting
Moscow, Russian Federation, 119991
Contact: Eduard Ambarchian    +7 915 378 3941    edo_amb@mail.ru   
Principal Investigator: Nikolay Murashkin, Prof         
Serbia
University of Belgrade, School of Medicine Recruiting
Belgrade, Serbia, 11000
Contact: Jovan Lalosevic       drjovan.lalosevic@gmail.com   
Principal Investigator: Milos Nikolic, Prof         
Singapore
Kandang Kerbau (KK) Women's and Children's Hospital Recruiting
Singapore, Singapore, 229899
Contact: Valerie Pui Yoong Ho, Dr.       valerie.ho.p.y@singhealth.com.sg   
Principal Investigator: Mark Jean Aan Koh, Dr.         
Spain
Hospital Sant Joan de Déu Recruiting
Barcelona, Spain, 08950
Contact: Carolina Prat    +34 93 600 9733    avicente@sjdhospitalbarcelona.org   
Principal Investigator: Maria Vincente Villa, Dr.         
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         
Hospital Viamed Santa Ángela de la Cruz Recruiting
Sevilla, Spain, 41014
Contact: Maria Teresa Monserrat       marite_monserrat@hotmail.com   
Principal Investigator: Jose Bernabeau Wittel, Dr.         
Switzerland
Bern University Hospital Recruiting
Bern, Switzerland, 3010
Contact: Mark Wienand    +41 316326983    mark.wienand@insel.ch   
Principal Investigator: Carolina Gouveia, Dr.         
Ukraine
National Children Specialized Hospital "Ohmatdyt" of Ministry of Health of Ukraine Recruiting
Kyiv, Ukraine, 01135
Contact: Tetyana Marchyshyna    +38 0978774665    myroniuk_ebkabinet@ukr.net   
Principal Investigator: Inna Gedeon, Dr         
United Kingdom
Birmingham Children's Hospital NHS Trust Recruiting
Birmingham, United Kingdom
Contact: Victoria Lynne    +44 1213339999    victoria.lynne@nhs.net   
Principal Investigator: Malobi Ogboli, Dr.         
Great Ormond Street hospital Recruiting
London, United Kingdom, WC1N3JH
Contact: Sabrina Marques    +44 2077626892    Sabrina.Marques@gosh.nhs.uk   
Principal Investigator: Anna Martinez, Dr.         
Sponsors and Collaborators
Amryt Research Limited
Investigators
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Principal Investigator: Johannes S Kern, MD PhD Melbourne Health

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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: October 3, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous