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    Summary
    EudraCT Number:2012-002553-38
    Sponsor's Protocol Code Number:TERCELOI
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-04-09
    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 number2012-002553-38
    A.3Full title of the trial
    Mesenchymal stem cell based therapy for the treatment of osteogenesis imperfecta
    Terapia celular basada en células madre mesenquimales aplicada a pacientes pediátricos con Osteogénesis Imperfecta.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Mesenchymal stem cell based therapy for the treatment of osteogenesis imperfecta
    Terapia cellular basada en células madre mesenquimales aplicada a pacientes pediátricos con Osteogénesis Imperfecta.
    A.3.2Name or abbreviated title of the trial where available
    TERCELOI
    TERCELOI
    A.4.1Sponsor's protocol code numberTERCELOI
    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 SponsorItziar Astigarraga Aguirre
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportHOSPITAL UNIVERSITARIO CRUCES
    B.4.2CountrySpain
    B.4.1Name of organisation providing supportMINISTERIO DE SANIDAD- Convocatoria EC Independientes 2010
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHOSPITAL UNIVERSITARIO CRUCES
    B.5.2Functional name of contact pointU. EPIDEMIOLOGIA CLINICA- APOYO INV
    B.5.3 Address:
    B.5.3.1Street AddressPLAZA DE CRUCES S/N
    B.5.3.2Town/ cityBARAKALDO (BIZKAIA)
    B.5.3.3Post code48903
    B.5.3.4CountrySpain
    B.5.4Telephone number34946000002368
    B.5.5Fax number34946006451
    B.5.6E-mailana.irasarrisebastian@osakidetza.net
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namecelulas madre mesenquimales troncales adultas alogenicas de médula ósea no expandida
    D.3.2Product code REF CRUZADA PEI Nº 12-088
    D.3.4Pharmaceutical form Living tissue equivalent
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNcélulas mesenquimales troncales adultas alogénicas de médula ósea no expandidas
    D.3.9.2Current sponsor codecélulas mesenquimales troncales adultas alogénicas de médula ósea no expa (ref cruzada PEI Nº 12-088
    D.3.9.3Other descriptive namecélulas mesenquimales troncales adultas alogénicas de médula ósea no expandidas(ref cruzada PEI nº 12-088)
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    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 Yes
    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 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
    Osteogenesis imperfecta (OI) is a rare genetic disorder with increased bone fragility of varying severity. In the majority of patients the disease is caused by mutations in collagen type I. Severe OI is characterized by osteopenia, frequent fractures, progressive deformity, short stature, loss of mobility, chronic pain and can lead to premature death. At present a cure does not exist.
    La Osteogenesis imperfecta (OI) es una enfermedad rara de base genética, causada en su mayor parte por alteraciones en los genes que codifican para el colágeno tipo I. Se caracteriza por una gran fragilidad ósea, que da lugar a la aparición de fracturas espontáneas o tras sufrir traumatismos mínimos. Las fracturas óseas frecuentes, la disminución de la masa ósea total y la propia formación anómala del colágeno conducen a importantes deformidades esqueléticas así como al retraso del crecimiento.
    E.1.1.1Medical condition in easily understood language
    Osteogenesis Imperfecta (OI), characterized by bone fragility and reduced bone mass, is a heritable disorder caused by mutations in the collagen type I gene in 90 % of cases.
    La osteogénesis imperfecta (OI) es una enfermedad rara caracterizada por un aumento de la fragilidad ósea, hecho por el que se le ha denominado enfermedad de los huesos de cristal
    E.1.1.2Therapeutic area Body processes [G] - Cell Physiological Phenomena [G04]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the safety of the stem cell transplantation process using characterized HLA-identical MSC in OI infants already subjected to pamidronate treatment, but who are not receiving immunosuppresion treatment
    El objetivo principal de este proyecto es determinar la seguridad del tratamiento en
    pacientes con OI severa, que no han sido sometidos a ningún tratamiento previo de inmunosupresión, con MSC alogénicas procedentes de médula ósea de donantes idénticos en antígenos leucocitarios humanos HLA
    E.2.2Secondary objectives of the trial
    To demonstrate the efficacy of the MSCs procedure, to verify that treatment of OI patients with several HLA-identical MSC injections, is not only a safe procedure (primary objective), but will increase the benefits observed with only one HLA-identical MSC injection
    determinar la eficiencia del tratamiento con MSC en dichos pacientes en los que evaluaremos diferentes parámetros relacionados con el metabolismo óseo, disminución del número de fracturas, crecimiento longitudinal y mejoría de la calidad de vida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.-Patient age: older than 6 months and younger than 12 years old.
    2.-Patients with molecular confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
    3.-Patiens with HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
    4.-All patients that fulfil the inclusion criteria regardless of whether or not they are undergoing biphosphonate treatment
    5.-Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial.
    1.-Edad del paciente: mayor de 6 meses y menor de 12 años.
    2.-Caracterización genotípica: conocimiento previo de las mutaciones de los genes del colágeno (COL1A1, COL1A2) asociadas al fenotipo clínico.
    3.-Pacientes con hermano/a(s) sano(a)(s) potenciales donantes de médula ósea con los que compartan al menos 5 de las 6 identidades del antígeno mayor de histocompatibilidad (HLA).
    4.-Se incluyen todos los pacientes que ya estén o no en régimen de tratamiento intravenoso u oral compasivo con bifosfonatos.
    5.-Pacientes cuyos padres o tutores legales sean capaces de dar consentimiento informado.
    Se ha informado al sujeto y se le ha dado el tiempo suficiente y la oportunidad para considerar su participación y ha otorgado su consentimiento informado por escrito.
    E.4Principal exclusion criteria
    1.-Patient age: older than 12 years old
    2.-Patients lacking confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
    3.-Other pathological subtypes of OI.
    4.-Patiens lacking of HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
    5.-Immunodeficiencies and any other malignancies
    6.-Participation in other clinical trial
    7.-Any medical or psychiatric condition that in the researcher?s opinion could affect the patient´s ability to complete the trial or hamper the participation in the trial.
    8.-Patients whose parents or the legal guardians do not sign the consent forms
    1) Edad mayor de 12 años.
    2) Ausencia de confirmación de la alteración molecular responsable del fenotipo clínico.
    3) Otros subtipos clínico patológicos de OI.
    4) Ausencia de hermanos sanos o con un HLA inferior a 5 identidades.
    5) Inmunodeficiencias, enfermedades malignas, hematológicas u de otro tipo que contraindique el tratamiento.
    6) Participación en otro ensayo clínico.
    7) Cualquier proceso médico o psiquiátrico que, en opinión del investigador, pudiera afectar a la capacidad del paciente para completar el estudio, afecte al seguimiento y evaluación de los resultados o impida la participación en el mismo.
    8) Pacientes cuyos padres o tutores legales no firmen el consentimiento informado
    E.5 End points
    E.5.1Primary end point(s)
    The principal aim of this project is to assess the safety of non-mutated HLA-identical MSC transplantation for OI pediatric patients irrespective of treatment with pamidronate.
    Since MSC are inherently non-immunogenic and do not elicit proliferation of allogeneic lymphocytes in co-culture experiments, a cell therapy based on HLA-identical allogenic MSC or that they shared at least 5 out of 6 HLA antigens it could be accomplished without subjecting the patients to immunosupressor treatment. Adverse secondary effects due to immunosopressor treatment could be avoided using this strategy and patients may reap the benefits of two cellular infusions instead of one. The beneficial effects observed in patients subjected to cell therapy seem to last for approximately six months after the infusion.
    In addition to clinical and analytical analysis of blood and urine tests, the immune responses of treated patients will be evaluated. Humoral and cell-mediated responses will be determined.
    El objetivo principal de este proyecto es determinar la seguridad del tratamiento en pacientes con OI severa, que no han sido sometidos a ningún tratamiento previo de inmunosupresión, con MSC alogénicas procedentes de médula ósea de donantes idénticos ó con una única diferencia en antígenos leucocitarios humanos HLA.
    Dada la escasa inmunogenicidad que presentan estas células, la terapia celular basada en MSCs alogénicas idénticas o casi idénticas sería factible sin tener que someter al paciente a ningún tratamiento inmunosupresor evitando de esta manera todos los efectos secundarios adversos asociados. Así los pacientes podrán beneficiarse del tratamiento basado en dos infusiones celulares, ya que los efectos causados por una infusión celular parecen remitir al cabo de 6 meses.
    Por ello además de una valoración clínica y analítica en sangre y orina se llevarán a cabo análisis de la respuesta inmune (determinación de la respuesta humoral y de la respuesta celular).
    E.5.1.1Timepoint(s) of evaluation of this end point
    The clinical and analytical evaluations as well as immune response evaluation will take place in the initial visit (before the cell infusion which will be visit 1) to establish baselines and at the following intervals:
    .-visit 2, 7 days post-first infusion
    .-visit 3, 28 days post-first infusion
    .-visit 4, 4 months post-first infusion
    .-visit 5, 5 months post-first infusion, second infusion
    .-visit 6, 7 days post-second infusion
    .-visit 7, 28 days post-second infusion
    .-visit 8, 9 months post-first infusion
    The proliferation of allogeneic lymphocytes in co-culture experiments will be evaluated at the initial visit, visit number 4 and visit number 8.
    La evaluación clínica y analítica básica además del análisis de la respuesta inmune se llevarán a cabo en la visita inicial (antes del tratamiento que será la visita 1) y en las siguientes visitas:
    .-visita 2 a los 7 días de la primera infusión celular
    .-visita 3 a los 28 días de la primera infusión celular
    .-visita 4 a los 4 meses de la primera infusión celular
    .-visita 5 a los 5 meses de la primera infusión celular, segunda infusión
    .-visita 6 a los 7 días de la segunda infusión
    .-visita 7 a los 28 días de la segunda infusión
    .-visita 8 a los 9 meses de la primera infusión
    La determinación de respuesta T frente a poblaciones celulares alogénicas tendrá lugar en la visita inicial, en la visita 4 (antes de la segunda infusión celular) y en la visita final visita 8.
    E.5.2Secondary end point(s)
    The secondary end point is to demonstrate the efficacy of MSC transplantation for OI patients.
    The evaluation of treatment efficacy will be based on physical and clinical evaluation, analytical and radiological parameters associated with bone metabolism, reduction in the frequencies of bone fracture, increase in growth velocity, positive impact in functionality and an improvement in well being.
    El objetivo secundario de este ensayo es demostrar la eficacia que el medicamento celular tendrá sobre estos pacientes. La valoración de la eficacia se basa en diferentes parámetros analíticos y radiológicos relacionados con el metabolismo óseo, la esperable disminución del número de fracturas, el aumento del ritmo esperable de crecimiento longitudinal, la positiva repercusión en la funcionalidad y en el bienestar general.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The clinical and analytical evaluations will take place in the initial visit (before the cell infusion on visit 1) and at the following visits:
    .-visit 2, 7 days post-first infusion
    .-visit 3, 28 days post-first infusion
    .-visit 4, 4 months post-first infusion
    .-visit 5, 5 months post-first infusion, second infusion
    .-visit 6, 7 days post-second infusion
    .-visit 7, 28 days post-second infusion
    .-visit 8, 9 months post-first infusion
    The radiological tests only will be performed at the initial and last visits.
    Los análisis tendrán lugar en la visita inicial (antes del tratamiento que será la visita 1) y en las siguientes visitas:
    .-visita 2 a los 7 días de la primera infusión celular
    .-visita 3 a los 28 días de la primera infusión celular
    .-visita 4 a los 4 meses de la primera infusión celular
    .-visita 5 a los 5 meses de la primera infusión celular, segunda infusión
    .-visita 6 a los 7 días de la segunda infusión
    .-visita 7 a los 28 días de la segunda infusión
    .-visita 8 a los 9 meses de la primera infusión
    Las pruebas de imagen solo se realizarán en la visita basal y en la visita 8 (a los 9 meses de la primera infusión).
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    pilot
    Piloto
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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-Visit 8 is the last visit and the end of the trial
    LVLS-Visita 8 es la última visita y el final del ensayo.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 3
    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: 3
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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.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 Decision2013-08-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-11-12
    P. End of Trial
    P.End of Trial StatusOngoing
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