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    Summary
    EudraCT Number:2012-001815-21
    Sponsor's Protocol Code Number:ABH_EB-001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2012-09-26
    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 ConcernedSpain - AEMPS
    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
    Estudio multicéntrico, prospectivo, aleatorizado, abierto, de control intraindividual, sobre la eficacia y seguridad de ABH001 para el tratamiento de
    heridas cutáneas crónicas estancadas asociadas con la epidermólisis bullosa 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.
    Estudio sobre la seguridad y eficacia de ABH001 para tratamiento de pacientes con epidermólisis bullosa con heridas incurables
    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 No
    A.8EMA Decision number of Paediatric Investigation Plan
    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) Yes
    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
    Epidermólisis bullosa
    E.1.1.1Medical condition in easily understood language
    Genetic skin blistering condition
    Enfermedad cutánea genética ampollosa
    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 15.0
    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
    To evaluate the efficacy of ABH001 in initiating healing of selected cutaneous, stalled wounds in epidermolysis bullosa patients.
    Evaluar la eficacia de ABH001 para iniciar la cicatrización de las heridas cutáneas tórpidas en pacientes con epidermólisis bullosa
    E.2.2Secondary objectives of the trial
    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, and the duration of, wound size reduction.
    Evaluar parámetros relacionados con los resultados percibidos por el sujeto y el clínico al comparar las heridas
    tratadas con ABH001 y las heridas tratadas con Control
    Documentar la seguridad de las aplicaciones de ABH001 en esta población de sujetos
    Evaluar el tiempo y la duración de la reducción del tamaño de la herida
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability of subject (or subject?s legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent/assent and authorization to use protected health information in accordance with national and local patient regulations.
    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 following wound selection criteria at the end of the observation period:
    i. Two clinically non-infected cutaneous wounds with no clinically meaningful change in wound size during the observation period.
    ii. Two matched wounds.
    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.
    1. Capacidad de comprender el propósito y los riesgos del estudio y de proporcionar un
    consentimiento/asentimiento firmado y fechado y una autorización para el uso de la información sanitaria
    protegida de acuerdo con la normativa nacional y local aplicable a los pacientes.
    2. Sujetos masculinos y femeninos
    3. Estado nutricional estable
    4. Sujetos con un diagnóstico confirmado de epidermólisis bullosa generalizada y heridas cutáneas que cumplan
    los criterios siguientes:
    a. Ubicación anatómica: brazos, piernas, tórax, espalda por encima de la cintura y por debajo del
    cuello.
    b. Antigüedad documentada (duración) de la(s) herida(s)
    c. Una o más heridas con posibilidades de poder cumplir los siguientes criterios de selección de la herida, el fin del periodo de observación:
    i. Dos heridas cutáneas clínicamente no infectadas con un cambio del área superficial de la herida durante el periodo de observación
    ii. Dos heridas equivalentes
    5. Prueba de embarazo realizada en orina negativa en el caso de mujeres en edad fértil
    6.Los sujetos de sexo femenino en edad fértil y los sujetos de sexo masculino capaces de procrear deben aceptar
    el uso de un método anticonceptivo eficaz
    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. Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, recombinant DNA/protein therapy. If received, other investigational drug treatments for EB that have not met the acceptable washout periods of: 1 month for topical treatments, 3 months for prior ABH001 treatment on the study wound, 6 months for small molecules, and 1 year or five half-lives for biologics.
    9. History of malignant skin disease.
    1. Mujeres embarazadas o lactantes
    2. Diagnóstico de EB generalizada no-hereditaria
    3. Infección clínica activa y localizada de las heridas del estudio
    4. Enfermedades o afecciones que podrían,interferir con la evaluación de la
    seguridad y la eficacia del tratamiento de estudio y con el cumplimiento por parte del sujeto de las visitas o los
    procedimientos
    5. Alergia conocida a productos bovinos
    6. Alergia conocida a productos con plata
    7. Infección sistémica en el momento de la inclusión en el estudio
    8. Estar participando o haber participado en otros estudios clínicos relacionados con la terapia genética, la terapia con
    células madre, la terapia con ADN/proteínas recombinante(s). Si han recibido otros tratamientos farmacológicos en
    investigación para la EB, pero no han cumplido los periodos de reposo farmacológico aceptables de: 1 mes para
    tratamientos tópicos, 3 meses para un tratamiento anterior con ABH001 en la herida del estudio, 6 meses para
    moléculas pequeñas y 1 año o cinco vidas medias para biofármacos.
    9. Historial de neoplasias malignas de la piel
    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
    El criterio de valoración principal es la reducción del área superficial de la herida tratada con ABH001 y la herida equivalente tratada con Control.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks
    24 semanas
    E.5.2Secondary end point(s)
    Change from baseline in wound pain and itch.
    Patient global impression of change (PGIC).
    Clinician global impression of change (CGIC).
    Proportion of subjects achieving reduction in wound surface area.
    Time to reduction of wound surface area and duration of reduction.
    Incidence and severity of adverse events
    Cambio en el dolor y el prurito de la herida desde el inicio. Impresión general del paciente de cambio (IGPC). Impresión general del clínico de cambio (IGCC). Proporción de sujetos que alcanzan reducción de la superficie de la herida. Tiempo y duración de la reducción de la superficie de la herida.. Incidencia y gravedad de efectos adversos.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    Semana 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 EEA5
    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
    Australia
    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: 20
    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: 10
    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.
    Los niños muy pequeños que no pueden dar su consentimiento personalmente. Se obtendrá el consentimiento de su tutor legal.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 25
    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
    Ninguno
    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 Decision2013-01-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-10-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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