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    Summary
    EudraCT Number:2011-004801-25
    Sponsor's Protocol Code Number:TCIM/ELA
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-01-11
    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 number2011-004801-25
    A.3Full title of the trial
    A PHASE I/II CLINICAL TRIAL OF THE BONE MARROW'S AUTOLOGOUS STEM CELLS IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS
    ENSAYO CLÍNICO EN FASE I/II DE INFUSIÓN INTRAMUSCULAR DE CÉLULAS MADRE DE MÉDULA ÓSEA AUTÓLOGAS EN PACIENTES CON ESCLEROSIS LATERAL AMIOTRÓFICA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    CLINICAL TRIAL ON THE USE OF BONE MARROW OWN PATIENT'S WITH AMYOTROPHIC LATERAL SCLEROSIS
    ENSAYO CLÍNICO DE LA UTILIZACIÓN DE CÉLULAS MADRE DE MÉDULA ÓSEA DEL PROPIO PACIENTE EN LA ESCLEROSIS LATERAL AMIOTRÓFICA
    A.4.1Sponsor's protocol code numberTCIM/ELA
    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 SponsorFundación Sanitaria para el Fomento de la Investigación Sanitaria y Biomédica de la Comunidad Valenciana (FISABIO)
    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 supportMinisterio de Sanidad, Servicios Sociales e Igualdad
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCRO-Fundación de la C.Valenciana para la Investigación en el Hospital General Universitario de Alicante
    B.5.2Functional name of contact pointRocío Caño Alameda
    B.5.3 Address:
    B.5.3.1Street AddressPintor Baeza nº12
    B.5.3.2Town/ cityAlicante
    B.5.3.3Post code03010
    B.5.3.4CountrySpain
    B.5.4Telephone number0034965913868
    B.5.5Fax number0034965913896
    B.5.6E-mailcanyo_roc@gva.es
    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 namecélulas troncales hematopoyéticas autólogas adultas extraidas de médula ósea
    D.3.4Pharmaceutical form Solution for injection/infusion in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNautologous bone marrow mononucleated cells
    D.3.9.1CAS number no available
    D.3.9.2Current sponsor codeTCIM/ELA
    D.3.9.3Other descriptive nameautologous bone marrow mononucleated cells
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntramuscular use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    amyotrophic lateral sclerosis
    esclerosis lateral amiotrófica
    E.1.1.1Medical condition in easily understood language
    esclerosis lateral amiotrófica
    amyotrophic lateral sclerosis
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system 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 determine the safety of intramuscular infusion of stem cells from autologous bone marrow in patients with amyotrophic lateral sclerosis.
    To determine the ability of stem cells from autologous bone marrow to modify the time course of progressive loss of motor units that characterizes amyotrophic lateral sclerosis in tibialis anterior muscle.
    Determinar la seguridad de la infusión intramuscular de células madre de médula ósea autólogas en pacientes con esclerosis lateral amiotrófica.
    Determinar la capacidad de las células madre de médula ósea autólogas de modificar el curso temporal de pérdida progresiva de unidades motoras que caracteriza a la esclerosis lateral amiotrófica en músculo tibial anterior.
    E.2.2Secondary objectives of the trial
    Determine the usefulness of ultrasound for monitoring muscle muscle morphology in the evolution of patients with ALS.

    Correlate the maximum force of isometric contraction muscle and muscle morphology (obtained from ultrasound) with the estimated number of motor units in the evolution of patients with ALS.
    Determinar la utilidad de la ecografía muscular para el seguimiento de la morfología muscular en la evolución de pacientes con ELA.

    Correlacionar la fuerza máxima muscular de contracción isométrica y la morfología muscular (obtenidos a partir de ecografía) con el número estimado de unidades motoras, en la evolución de pacientes con ELA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Aged between 18 and 70 years.
    - Diagnosis of definite or probable ALS according to the criteria
    established by the World Federation of Neurology.
    - Patient providing sufficient guarantees of adherence to protocol.
    - Neurophysiological data confirming lower motor neuron involvement of lumbar level.
    - No motor deficit in dorsiflexion of both feet (4 + or 5 points on the MRC)
    - Edad comprendida entre los 18 y los 70 años.
    - Diagnóstico de ELA definida o probable de acuerdo con los criterios
    establecidos por la World Federation of Neurology.
    -Paciente que ofrezca garantías suficientes de adhesión al protocolo.
    - Datos neurofisiológicos que confirmen afectación de motoneurona inferior a nivel lumbar.
    - Valoración del déficit motor en la flexión dorsal de ambos pies (4+ó 5 puntos en la escala MRC)
    E.4Principal exclusion criteria
    - Diabetes Mellitus.
    - Other diseases that may occur with polyneuropathy.
    - A previous history of stroke.
    - A previous pathology of peripheral nervous system that affects one or both lower limbs with or without clinically evident neurologic sequelae.
    - Patients who are pregnant or breastfeeding active
    - Patients physiologically capable of becoming pregnant, unless they are using reliable contraception (Annex III)
    - Patients with heart, kidney, liver, systemic immune that may influence patient survival during the test.
    - Positive serology for hepatitis B, hepatitis C or HIV.
    - Clinical criteria and anesthesia sedation contraindicating either itself or extraction MO (Altered coagulation system or anticoagulated patient inability to withdraw anticoagulation, hemodynamic instability, altered skin puncture area, etc.)
    - Included in other clinical trials in the last 6 months.
    - Inability to understand informed consent.
    - Diabetes Mellitus.
    - Otras enfermedades que puedan cursar con polineuropatías.
    - Historia previa de ictus cerebral.
    - Patología previa del sistema nervioso periférico que afectara a uno o ambos miembros inferiores con o sin secuelas neurológicas clínicamente evidentes.
    - Pacientes embarazadas o en período de lactancia activa
    - Pacientes fisiológicamente capaces de quedarse embarazadas, excepto que estén utilizando un método anticonceptivo fiable (Anexo III)
    - Pacientes con enfermedad cardiaca, renal, hepática, sistémica, inmune que pueda influir en la supervivencia del paciente durante el ensayo.
    - Serología positiva para hepatitis B, hepatitis C o VIH.
    - Criterios clínicos y anestésiológicos que contraindiquen bien la sedación o bien la propia extracción de MO (Alteración del sistema de la coagulación o paciente anticoagulado con imposibilidad para retirar la anticoagulación, inestabilidad hemodinámica, alteración cutánea en la zona de punción, etc.)
    - Inclusión en otros ensayos clínicos en los últimos 6 meses.
    - Incapacidad de comprender el consentimiento informado.
    E.5 End points
    E.5.1Primary end point(s)
    1)Absence of serious adverse events (SAE) related to the procedure.

    2) Rate of non-serious adverse events regarding possible, probable or defined with the procedure.

    3) Neurophysiological parameters (measured in both TA muscles):

    - Motor units number estimation (MUNE)
    - fiber density (FD)
    - Amplitude and area of the compound muscular action potential (CMAP).

    4) Neurological:

    - Maximum strength developed in an isometric contraction in both TA muscles.
    - The measurement will be performed with a digital dynamometer during dorsiflexion of the foot.

    Neurophysiological and neurological assessments were made in the times that are specified in the protocol: at baseline and at 30, 90 and 180 days of infusion.Also, there will be a neurological assessment at 7 days of infusion. See Appendix B (Protocol)
    1)Ausencia de acontecimientos adversos graves (AAG) con relación posible, probable o definida con el procedimiento.

    2)Tasa de acontecimientos adversos no graves con relación posible, probable o definida con el procedimiento.

    3) Neurofisiológicas:
    - Número estimado de unidades motoras
    - Densidad de fibras: Nº
    - Potencial de acción muscular compuesto (PAMC): amplitud del PAMC y área del PAMC

    4) Neurológicas:
    - Fuerza máxima desarrollada en una contracción isométrica del músculo TA: Newtons.
    - La medición será realizada con un dinamómetro digital durante la flexión dorsal del pie.

    Las valoraciones neurofisiológica y neurológica se realizarán en los tiempos que quedan especificados en el protocolo: en la visita basal y a los 30, 90 y 180 días de la infusión.Asimismo, se realizará una valoración neurológica a los 7 días de la infusión. Ver Apéndice B (Protocolo)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Number of serious adverse events related to the procedure. Follow-up: Visit 0 (Day 0) up to visit 6 (day +180)
    ? neurophysiological parameters: MUNE, DF, PAMC. Follow-up: baseline visit (day -14), visit 2 (day +30), visit 4 (day +90) and visit 6 (day +180)
    ? Muscular strength developed in an isometric contraction. Follow-up: baseline visit (day -14), visit 2 (day +30), visit 4 (day +90) and visit 6 (day +180)
    ? Número de eventos adversos severos relacionados con el procedimiento. Seguimiento: Visita 0 (día 0) hasta visita 6 (día +180)
    ? Parámetros neurofisiológicos: MUNE, DF, PAMC. Seguimiento: Visita basal (día -14), visita 2 (día +30), visita 4 (día +90) y visita 6 (día +180)
    ? Fuerza muscular desarrollada en una contracción isométrica. Seguimiento: Visita basal (día -14), visita 2 (día +30), visita 4 (día +90) y visita 6 (día +180)
    E.5.2Secondary end point(s)
    1)Neurological variables: Assessment of motor function.
    The muscle force data are collected according to the scale of the Medical Research Council (1943). Muscle strength is graded on a scale of 0 to 5, with 5 being normal muscle strength and 0 absence of muscle contraction.

    2) Sonographic variables: - Presence of local complications related to the process (edema, abscesses, muscle necrosis, etc.): Yes / No
    - Maximum transverse area of both TA muscles.


    The ultrasound study was performed using a linear probe of 12 to 15 MHz


    These assessments will be made in the times that are specified in the protocol: at baseline, at 7, 30, 90 and 180 days of infusion
    1)Neurológicas:
    -Fuerza muscular MRC score: Nº

    Los datos de la fuerza muscular se recogerán de acuerdo con la escala del Medical Research Council (1943). La fuerza muscular está graduada en una escala de 0 a 5, siendo 5 fuerza muscular normal y 0 ausencia de contracción muscular.

    2)Ecográficas:
    - Presencia de complicaciones locales relacionadas con el procedimiento (Edema, abscesos, necrosis muscular, otras): Si/No
    -Área transversal máxima del músculo TA: cm2

    El estudio ecográfico se llevará a cabo utilizando una sonda lineal de 12 a 15 MHz.

    Estas valoraciones se realizarán en los tiempos que quedan especificados en el protocolo: en la visita basal, a los 7, 30, 90 y 180 días de la infusión
    E.5.2.1Timepoint(s) of evaluation of this end point
    ? Variables Neurological Evaluation of the TA muscle motor function of both lower limbs using the MRC scale.
    ? Ultrasound Variables: Analysis of the TA muscle of both legs to assess muscle mass and maximum cross-sectional area, analyzing the appearance of edema, abscess, or necrosis.
    ? Variables neurológicas: Evaluación de la función motora del músculo TA de ambos miembros inferiores mediante la escala MRC.
    ? Variables ecográficas: Análisis del músculo TA de ambos miembros inferiores para evaluar la masa muscular y el área transversal máxima, analizando la aparición de edema, abscesos o necrosis muscular.
    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 No
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.7.1Other trial design description
    prospectivo
    prospective
    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 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 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
    The protocol will be terminated (and the patient excluded from the trial) if there are any signs of contamination during the processing of the bone marrow to obtain mononucleated cells. Also in the following cases:
    The presence of a serious adverse event.
    Any violation of the protocol.
    Facultative decision
    Patient refusal to continue the study.
    Losses of patient follow-up.
    El tratamiento se interrumpirá si se sospecha una contaminación durante el procesamiento de la médula ósea para la obtención de las céluas mononucleares.

    También se interrumpirá prematuramente en los siguientes casos:

    Presencia de AAG.
    Otras violaciones del protocolo.
    Decisión facultativa
    Renuncia del paciente a continuar en el estudio.
    Pérdida de seguimiento del paciente.
    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 months
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state20
    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)
    There is currently no effective treatment against the disease, which determines which patients suffer an inexorable decline since diagnosis with evidence of progressive paralysis usually upward, until the inevitable respiratory arrest. The knowledge of this development leads in both the patients and the family great anguish and despair, with a negative impact on quality of life and often severe alterations in mood.
    Actualmente no existe ningún tratamiento eficaz contra la enfermedad, lo que determina que los pacientes sufran un deterioro inexorable desde el diagnóstico con evidencia de parálisis progresiva habitualmente ascendente, hasta la inevitable parada respiratoria. El conocimiento de esta evolución provoca tanto en los pacientes como en el entorno familiar una gran angustia y desesperanza, con un impacto negativo en la calidad de vida y con frecuencia alteraciones severas del estado de ánimo.
    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 Decision2014-07-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-30
    P. End of Trial
    P.End of Trial StatusCompleted
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