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    Summary
    EudraCT Number:2016-002066-32
    Sponsor's Protocol Code Number:BEB-13
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-01-17
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2016-002066-32
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial which compare the safety and efficacy of a wound gel, the study treatment, or a sunflower oil-based vehicle gel in patients with Inherited Epidermolysis Bullosa (EB)
    A.3.2Name or abbreviated title of the trial where available
    EASE study
    A.4.1Sponsor's protocol code numberBEB-13
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03068780
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmryt Research Limited
    B.1.3.4CountryIreland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmryt Research Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmryt Research Limited
    B.5.2Functional name of contact pointDerval O'Carroll
    B.5.3 Address:
    B.5.3.1Street Address90 Harcourt Street
    B.5.3.2Town/ cityDublin 2
    B.5.3.4CountryIreland
    B.5.4Telephone number353868141007
    B.5.6E-mailderval.ocarroll@amrytpharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Episalvan gel
    D.2.1.1.2Name of the Marketing Authorisation holderAmryt AG
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/10/845
    D.3 Description of the IMP
    D.3.1Product nameOleogel-S10
    D.3.4Pharmaceutical form Gel
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPCutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBirch bark extract
    D.3.9.1CAS number 1640971-03-4
    D.3.9.2Current sponsor codeDry extract from betulae cortex
    D.3.9.3Other descriptive nameBIRCH BARK EXTRACT
    D.3.9.4EV Substance CodeSUB180085
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product Yes
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboGel
    D.8.4Route of administration of the placeboCutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Inherited Epidermolysis Bullosa
    E.1.1.1Medical condition in easily understood language
    Illness called Epidermolysis bullosa (EB) that makes skin blister and have open sores and wounds
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the double-blind phase is to compare the efficacy of Oleogel-S10 (treatment arm A) with vehicle (treatment arm B) in the promotion of healing of EB partial thickness wounds. This will be assessed as evidenced by the incidence of the 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.
    E.2.2Secondary objectives of the trial
    1/ Compare the efficacy of IMP with placebo as evidenced by several criteria described in the protocol
    2/ Compare the safety of IMP with placebo as evidenced by the incidence, severity, and relatedness of AEs and based on laboratory assessments
    3/ Compare the tolerability of IMP with placebo
    4/ Assess betulin exposure
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A patient will be eligible for study participation only if all of the following criteria apply:
    1. Male and female patients aged ≥4 years with the following subtypes of inherited EB: JEB, DEB, and Kindler syndrome [Note: Children ≥21 days old and <4 years may be included only after confirmation by the Independent Data Monitoring Committee (IDMC) upon review of the safety and bioanalytical data at the interim safety review stage]
    2. 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)
    3. 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
    4. Patient and/or his/her legal representative must be able and willing to follow study procedures and instructions
    E.4Principal exclusion criteria
    A patient will not be eligible to participate in this study if any of the following criteria apply:
    1. Patient has EB subtype EBS
    2. EB target wound that is > 9 months old or has clinical signs of local infection
    3. Use of systemic antibiotics for wound-related infections within 7 days prior to enrolment
    4. 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
    5. Immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrolment
    6. Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB
    7. Current and/or former malignancy including basal cell carcinomas and squamous cell carcinomas
    8. Enrolment in any interventional study or treated with any investigational drug for any disease within 4 weeks prior to study entry
    9. 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
    10. Pregnant or nursing women
    11. Women of childbearing potential, including postmenarchal female adolescents, and men who are 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, vasectomy or vasectomised partner) during participation in the study (and at least 3 months thereafter)
    12.Patient is a member of the investigational team or his/her immediate family
    13.Patient lives in the same household as a study participant.
    E.5 End points
    E.5.1Primary end point(s)
    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
    E.5.1.1Timepoint(s) of evaluation of this end point
    See section E.5.1
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints
    • Time to first complete closure of the EB target wound as evidenced by clinical assessment until EDBP (D90±7)
    • Proportion of patients with first complete closure of the EB target wound at D14±5, D30±7, D60±7, and D90±7 based on clinical assessment by the investigator
    • Proportion of patients with first complete closure of the EB target wound at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 based on patient assessment
    • Proportion of patients with first complete closure of the EB target wound at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7 based on blinded evaluation of photographs
    • Percentage change from baseline (DBP D0) in EB target wound size as evidenced by blinded evaluation of photographs taken at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7
    • 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 D30±7, D60±7, and D90±7
    • Change from baseline (DBP D0) in body surface area percentage (BSAP) of TBSA affected by EB partial thickness wounds as evidenced by clinical assessment based on the ‘Lund and Browder’ chart at D30±7, D60±7, and D90±7
    • The incidence and 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)
    • Change from baseline (DBP D0) in “background” pain using the ‘Face, Legs, Activity, Cry, Consolability’ (FLACC) pain rating scale in patients <4 years of age and the ‘Wong Baker FACES® Pain Rating Scale’ in patients ≥4 years of age before wound dressing changes at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7
    • Change from baseline (DBP D0) in “procedural” pain using the FLACC scale in patients <4 years of age and the ‘Wong-Baker FACES Pain Rating Scale’ in patients ≥4 years of age after wound dressing changes at D7±2, D14±5, D30±7, D45±7, D60±7, and D90±7
    • 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 D7±2, D30±7, D60±7, and D90±7
    • Change from baseline (DBP D0) in impact of wounds on sleep (in patients ≥14 years of age) as measured by differences in 11 point Likert scales at D7±2, D30±7, D60±7, and D90±7
    • The number of days missed from school or from work due to EB as reported by patients at D0 for the last 14 days and cumulatively for all visits until D90±7
    • Evaluation of the treatment response (in patients ≥14 years of age) using the TSQM, Version 9, before wound dressing changes at D7±2, D30±7, D60±7, and D90±7
    E.5.2.1Timepoint(s) of evaluation of this end point
    See section E.5.2
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Austria
    Belgium
    Brazil
    Chile
    Colombia
    Croatia
    Czech Republic
    Denmark
    France
    Germany
    Greece
    Hong Kong
    Hungary
    Ireland
    Israel
    Italy
    Romania
    Russian Federation
    Serbia
    Singapore
    Spain
    Switzerland
    Ukraine
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS estimated in January 2021
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 96
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 48
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 48
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 96
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 150
    F.4.2.2In the whole clinical trial 192
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-02-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-02-26
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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