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    Summary
    EudraCT Number:2012-001815-21
    Sponsor's Protocol Code Number:ABH_EB-001
    National Competent Authority:Portugal - INFARMED
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-03-28
    Trial results View 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 ConcernedPortugal - INFARMED
    A.2EudraCT number2012-001815-21
    A.3Full title of the trial
    A Multicenter, Prospective, Randomized, Open-label, Intra-patient Controlled Study of the Efficacy and Safety of ABH001 for the Treatment of Stalled Chronic Cutaneous Wounds Associated with Generalized Epidermolysis Bullosa
    Estudo Controlado Intradoentes, Aberto, Aleatório, Prospetivo, Multicêntrico da Eficácia e Segurança do ABH001 para o Tratamento de Feridas Cutâneas Crónicas Estagnadas Associadas a Epidermólise Bolhosa Generalizada
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the safety and efficacy of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.
    A.4.1Sponsor's protocol code numberABH_EB-001
    A.5.4Other Identifiers
    Name:Reformatted protocol numberNumber:EB01-ABH001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/310/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorShire Regenerative Medicine, Inc.
    B.1.3.4CountryUnited States
    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 supportShire Regenerative Medicine, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Regenerative Medicine, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address11095 Torreyana Road
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1858754-5725
    B.5.6E-mailclinicaltrialinfo@shire.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 No
    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/3/11/873
    D.3 Description of the IMP
    D.3.1Product nameHuman dermal fibroblasts cultured on a bioresorbable polyglactin mesh
    D.3.2Product code ABH001
    D.3.4Pharmaceutical form Living tissue equivalent
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman dermal fibroblasts cultured on a bioresorbable polyglactin mesh
    D.3.9.2Current sponsor codeABH001
    D.3.9.3Other descriptive nameHuman fibroblast-derived dermal substitute, Dermagraft
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    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 Yes
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Yes
    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 No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Epidermolysis bullosa
    E.1.1.1Medical condition in easily understood language
    Genetic skin blistering condition
    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 14.1
    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
    To evaluate the efficacy of ABH001 in initiating healing of selected cutaneous, stalled wounds in epidermolysis bullosa patients.
    E.2.2Secondary objectives of the trial
    To evaluate change in wound surface area over time.
    To evaluate durability of wound healing.
    To evaluate parameters related to subject and clinician reported outcomes for the ABH001-treated wound relative to the Control-treated wound.
    To document the safety of ABH001 applications in this subject population.
    To evaluate the time to wound size reduction.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or legal guardian must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent or Assent Form and must be able to and willing to follow study procedures and instructions.
    2. Male and female subjects.
    3. Stable nutritional status.
    4. Subjects with a confirmed diagnosis of generalized Epidermolysis Bullosa and cutaneous wounds meeting the following criteria:
    a. Anatomical location: arms, legs, thorax, or back above the waistline and below the neck.
    b. Documented age (duration) of the wound(s).
    c. One or more wounds capable of potentially meeting the wound selection criteria at the end of the observation period:
    i. Clininically non-infected cutaneous wounds with no clinically meaningful change in wound size during the observation period.
    ii. Matched wounds/wound areas.
    5. Negative urine pregnancy test for women of child-bearing potential.
    6. Female subjects of childbearing potential and male subjects of procreative capacity must agree to use an effective method of contraception.

    E.4Principal exclusion criteria
    1. Pregnant or nursing women.
    2. Diagnosis of non-genetic generalized EB.
    3. Localized, active clinical infection of study wounds.
    4. Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits/procedures.
    5. Known allergy to bovine products.
    6. Known allergy to silver products.
    7. Systemic infection at the time of enrolment in the study.
    8. Currently receiving or have received oral steroid therapy exceeding a total daily dose of 0.5 mg/kg (hydrocortisone equivalent potency) for more than 2 weeks, radiation or other immune-suppressive therapy within the previous 4 weeks.
    9. Participating or having participated in other investigational clinical studies, involving gene therapy, stem cell therapy and/or recombinant DNA/protein therapy.
    10. Received ABH001, or other biologic or cell therapy for the treatment of EB in the study wound sites within the previous 3 months
    11. Documented or suspected hypersensitivity to any of the therapeutic agents, including anaesthetic drugs.
    12. History of malignant skin disease.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the reduction in wound surface area in ABH001-treated versus control-treated wounds.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks
    E.5.2Secondary end point(s)
    Change in wound surface area over time.
    Durability of wound healing
    Change from baseline in wound pain.
    Clinician global impression of change (CGIC).
    Patient global impression of change (PGIC).
    Change from baseline in wound itch.
    Proportion of subjects achieving reduction in wound surface area.
    Time to reduction of wound surface area.
    Incidence and severity of adverse events
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    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 No
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Assessment blinded
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standardised wound care alone
    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 EEA6
    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
    Austria
    Canada
    France
    Germany
    Poland
    Portugal
    Spain
    United States
    E.8.7Trial has a data monitoring committee No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 21
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 11
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Very young children who are unable to provide assent personally. Legal guardian consent will be obtained.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 26
    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 Decision2013-05-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-07
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2013-10-02
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