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

Sponsor
Amryt Research Limited (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03068780
Collaborator
(none)
223
Enrollment
51
Locations
2
Arms
63.1
Anticipated Duration (Months)
4.4
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

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 control gel. The control gel is an identical looking sunflower oil gel that does not contain any active substance. The participant will receive either Oleogel-S10 or control 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
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
223 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 :
Mar 29, 2017
Actual Primary Completion Date :
Jun 11, 2020
Anticipated Study Completion Date :
Jul 1, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Oleogel-S10

Drug: Oleogel-S10
10% birch bark extract in 90% sunflower oil
Other Names:
  • Episalvan

    		•
    Placebo Comparator: Control Gel

    Drug: Control gel
    Sunflower oil gel
    Other Names:
  • 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 [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) in patients with inherited EB (subtypes JEB, DEB, or Kindler syndrome) within 45±7 days of treatment with Oleogel S10 compared to vehicle based on clinical assessment by the investigator (the wound will be rated as "closed" at first appearance of complete reepithelialisation without drainage)

    Secondary Outcome Measures

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

    2. 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. [90±7 days]

    3. 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) [90±7 days]

    4. 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) [90±7 days]

    5. 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 [90±7 days]

    6. 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 [90±7 days]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    21 Days and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Phoenix Children's Hospital Phoenix Arizona United States 85016
    2 Children's Hospital Colorado Aurora Colorado United States 80045
    3 Amjad Plastic Research Miami Florida United States 33144
    4 University of Minnesota Minneapolis Minnesota United States 55455
    5 Stony Brook University Hospital Stony Brook New York United States 11790
    6 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    7 The Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    8 Thomas Jefferson University Hospital Philadelphia Pennsylvania United States 19107
    9 Medical University of South Carolina Charleston South Carolina United States 29425
    10 Texas Dermatology and Laser Specialists San Antonio Texas United States 78218
    11 Consutorios Medicos (Instituto de Neumonologia y Dermatologia) Buenos Aires Argentina 1425
    12 Centro Médico Dra. De Salvo Buenos Aires Argentina 1426
    13 Centro de investigaciones Metabolicas, CINME Buenos Aires Argentina C1056ABJ
    14 Sydney Children's Hospital Sydney New South Wales Australia 2031
    15 Premier Specialists Sydney New South Wales Australia 2217
    16 The Royal Melbourne Hospital Parkville Victoria Australia 3050
    17 Murdoch Childrens Research Institute Royal Children's Hospital Parkville Victoria Australia 3502
    18 Universitaetsklinik fuer Dermatologie Salzburg Austria 5020
    19 IMIP Recife Pernanbuco Brazil 50070550
    20 Universidade Regional de Blumenau Blumenau Santa Catarina Brazil 89020400
    21 Instituto Da Crianca HCFMUSP São Paulo Brazil 05403.000
    22 Fundacion Debra Chile Santiago Chile 7760099
    23 Hospital De San Jose Bogotá DC Colombia
    24 University Hospital Brno, Children´s Hospital Brno Czechia 61500
    25 Aarhus University Hospital Aarhus Denmark 8000
    26 Hôpital Necker-Enfants Malades Paris France 75015
    27 CHU Toulouse - Hospital Larrey Toulouse France 31059
    28 S/R National Center of Dermatology and Venerology Tbilisi Georgia 159
    29 Medical Center University Freiburg Freiburg im Breisgau Germany 79104
    30 Kinder- und Jugendkrankenhaus AUF DER BULT Hannover Germany 30173
    31 Hospital of Skin and Veneral Diseases "A. Syggros" Athens Attiki Greece 16121
    32 Prince of Wales Hospital, The Chinese University of Hong Kong Hong Kong Hong Kong
    33 Semmelweis University, Faculty of Medicine Budapest Hungary 1085
    34 Our Ladys Childrens Hospital Dublin Ireland
    35 Tel Aviv Sourasky Medical Center Tel Aviv Israel 64239
    36 Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy 20122
    37 Bambino Gesù Children Hospital Roma Italy 00165
    38 Istituto Dermopatico dell'Immacolata IDI-IRCCS Roma Italy 00167
    39 Centrul Medical Sanador Bucharest Romania 011025
    40 State Scientific Center for Dermatovenerology and Cosmetology Moscow Russian Federation 107076
    41 Scientific Center of Children's Health Moscow Russian Federation 119991
    42 University of Belgrade, School of Medicine Belgrade Serbia 11000
    43 Kandang Kerbau (KK) Women's and Children's Hospital Singapore Singapore 229899
    44 Hospital Sant Joan de Déu Barcelona Spain 08950
    45 Hospital Universitari de la Vall d'Hebron Barcelona Spain 8035
    46 Hospital Universitario La Paz Madrid Spain 28046
    47 Hospital Viamed Santa Ángela de la Cruz Sevilla Spain 41014
    48 Bern University Hospital Bern Switzerland 3010
    49 National Children Specialized Hospital "Ohmatdyt" of Ministry of Health of Ukraine Kyiv Ukraine 01135
    50 Birmingham Children's Hospital NHS Trust Birmingham United Kingdom
    51 Great Ormond Street hospital London United Kingdom WC1N3JH

    Sponsors and Collaborators

    • Amryt Research Limited

    Investigators

    • Principal Investigator: Johannes S Kern, MD PhD, Melbourne Health

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Amryt Research Limited
    ClinicalTrials.gov Identifier:
    NCT03068780
    Other Study ID Numbers:
    • BEB-13
    • 2016-002066-32
    First Posted:
    Mar 3, 2017
    Last Update Posted:
    Mar 17, 2022
    Last Verified:
    Mar 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Epidermolysis Bullosa

    Study Results

    No Results Posted as of Mar 17, 2022