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    Summary
    EudraCT Number:2015-004293-15
    Sponsor's Protocol Code Number:UCART19_02(CL1-68587-001)
    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:2018-09-17
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2015-004293-15
    A.3Full title of the trial
    A phase 1, open label, non-comparative, study to evaluate the safety and the ability of UCART19 to induce molecular remission in paediatric patients with relapsed /refractory B-cell acute lymphoblastic leukaemia
    Estudio abierto, no comparativo, de fase 1 para evaluar la seguridad y la capacidad de UCART19 de inducir una remisión molecular en pacientes pediátricos con leucemia linfoblástica aguda de células B recurrente /refractaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the safety and efficacy of UCART19 in children with B cell lymphoblastic leukaemia that has relapsed or not responded to other treatments
    Estudio para evaluar la seguridad y eficacia de UCART19 en niños con una recaída de su leucemia linfoblástica aguda de células B o que no ha respondido a otros tratamientos
    A.3.2Name or abbreviated title of the trial where available
    UCART19_PALL
    UCART19_PALL
    A.4.1Sponsor's protocol code numberUCART19_02(CL1-68587-001)
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02808442
    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 SponsorInstitut de Recherches Internationales Servier (I.R.I.S)
    B.1.3.4CountryFrance
    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 supportLaboratorios Servier
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLaboratorios Servier S. L.
    B.5.2Functional name of contact pointDepartamento de I+D
    B.5.3 Address:
    B.5.3.1Street AddressAvenida de los Madroños
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28043
    B.5.3.4CountrySpain
    B.5.4Telephone number+34917489670
    B.5.5Fax number+3491300 32 49
    B.5.6E-mailmaria.dequintanabarajas@servier.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameUCART19
    D.3.2Product code S68587
    D.3.4Pharmaceutical form Infusion
    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 INNCD19CAR/RQR8+_TCRαβ–_T-cells
    D.3.9.2Current sponsor codeS68587
    D.3.9.3Other descriptive nameUCART19
    D.3.9.4EV Substance CodeSUB180949
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20000000
    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 Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberGene therapy medicinal product EMA/132648/2014
    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 Yes
    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
    Paediatric relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia
    Leucemia linfoblástica aguda de células B positivas en recaída o refractaria en población pediátrica
    E.1.1.1Medical condition in easily understood language
    Children with B-cell lymphoblastic leukaemia that has relapsed or for whom previous treatment has not worked.
    Niños con leucemia linfoblastica aguda de células B que han recaído o para los cuales no han funcionado los tratamientos previos
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060390
    E.1.2Term Leukaemia lymphoblastic acute
    E.1.2System Organ Class 100000004864
    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 safety of UCART19 in paediatric patients with relapsed or refractory B-ALL.
    - Evaluar la seguridad de UCART19 en pacientes pediátricos con LLA-B recurrente o refractaria.
    E.2.2Secondary objectives of the trial
    To determine the ability of UCART19 to achieve molecular remission at D28 after the first UCART19 infusion
    - Evaluar la capacidad de UCART19 de conseguir una remisión completa molecular el D28 tras la primera infusión de UCART19
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients

    2. Age ranging between 6 months and <18 years

    3a. Patients with relapsed or refractory CD19-positive B-acute lymphoblastic leukemia (B-ALL) (National Comprehensive Cancer Network (NCCN), 2017),
    - Morphologically confirmed with ≥ 5% leukemic blasts in the bone marrow
    - or presenting a quantifiable MRD load of 1x10-3 (by multiparameter flow cytometry and/or quantitative polymerase chain reaction) at the end of the last induction treatment
    - Who have exhausted alternative treatment options

    Relapsed disease is defined as:
    - second or subsequent bone marrow relapse or,
    - any bone marrow relapse after allo-SCT.

    Refractory disease is defined by not achieving an initial complete response (CR) after 2 cycles of a standard chemotherapy regimen (primary refractory). Subjects who were refractory to subsequent chemotherapy regimens after an initial remission were considered chemo-refractory

    Patients with Philadelphia chromosome positive (Ph+) ALL are eligible if they are intolerant to or have failed 2 lines of tyrosine kinase inhibitor therapy (TKI), or if TKI therapy is contraindicated.

    4. Estimated life expectancy ≥ 12 weeks (according to investigator’s judgement)

    6a. Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at the time of assent/consent) performance status ≥ 50

    35.a Adequate organ function defined as:
    a. Creatinine clearance ≥ 30 mL/min (as calculated using the method standard for the institution). In equivocal
    cases, a 24-hour urine collection test can be used to estimate the creatinine clearance more accurately.
    b. Serum ALT/AST ≤ 3 x ULN
    c. Total bilirubin ≤ 1.5 x ULN (unless the patient has a history of Gilbert’s Syndrome, in which case, total bilirubin must be ≤ 2.5 ULN)
    d. Left Ventricular Ejection Fraction (LVEF) ≥ 45% and no clinically significant ECG findings

    44. Availability of a donor for potential allo-HSCT in the event of persistent marrow aplasia without evidence of residual leukemia

    7. Written informed consent from parent(s) or legal representative and or written assent from patient when applicable obtained prior any study-specific procedure of the protocol
    1. Pacientes masculinos o femeninos
    2. Edad entre 6 meses y <18 años
    3a. Pacientes con leucemia linfoblástica aguda de células B (LLA-B) CD19 positivas recurrente /refractaria ((National Comprehensive Cancer Network (NCCN), 2017),
    - Confirmada morfológicamente con > 5% de blastos leucémicos en la médula ósea
    - o presentando una carga cuantificable de enfermedad mínima residual de 1x10-3 (por citometría de flujo y/o reacción en cadena de la polimerasa cuantitativa) al final del último tratamiento de inducción
    - que haya agotado las opciones de tratamiento alternativas
    - Se define enfermedad recurrente como:
    - segunda o posterior recurrencia en medula ósea
    - cualquier recidiva en médula ósea tras un trasplante alogénico de células madre hematopoyéticas.
    - Se define enfermedad refractaria cuando no se alcanza una respuesta completa (CR) tras dos ciclos de un régimen de quimioterapia estándar (refractaria primaria). Los pacientes refractarios a regímenes de quimioterapia posteriores tras una remisión inicial son considerados quimio-refractarios.
    - Los pacientes con LLA cromosoma Filadelfia positiva (Ph+) son candidatos si son intolerantes o han fracasado tras dos líneas de terapia con inhibidores de la tirosina kinasa (TKIs), o si la terapia con TKIs está contraindicada.
    4. Esperanza de vida estimada > de 12 meses (a criterio del investigador)
    6a. Estado funcional Lansky (edad < 16 años en el momento del consentimiento/asentimiento) o Karnofsky (edad > 16 años en el momento del consentimiento/asentimiento) > 50.
    35. Función orgánica adecuada, definida como:
    a. Aclaramiento de creatinina > 30 mL/min: (calculado utilizando el método estándar del centro). En casos dudosos, se puede utilizar una prueba de recogida de orina de 24 horas para estimar el aclaramiento de creatinina con mayor precisión).
    b. ALT/AST en suero < 3 x LSN
    c. Bilirrubina total < 1,5 x LSN (salvo que el paciente tenga antecedentes de Síndrome de Gilbert, en cuyo caso, la bilirrubina total debe ser < 2,5 x LSN)
    d. Fracción de eyección del ventrículo izquierdo (FEVI) > 45% y ausencia de hallazgos significativos en el ECG
    44. Disponibilidad de un donante para un potencial trasplante alogénico de células madre hematopoyéticas en el caso de aplasia medular persistente sin evidencia de leucemia residual
    7. Consentimiento informado por escrito del/los progenitor(es) o representante legal y o asentimiento por escrito del paciente cuando sea aplicable, obtenido antes de realizar cualquier procedimiento específico del estudio
    E.4Principal exclusion criteria
    34. Patients unwilling to undergo a safety follow-up for 15 years

    9. Foreseeable poor compliance to the study procedures

    10. Previous treatment with gene or gene-modified cell therapy medicine products. Prior treatment with blinatumomab is allowed

    11a. Use of previous anti-leukemic therapy (including approved therapies and other investigational products) within 5 half-lives prior to UCART 19 administration. Inotuzumab ozogamicin must be stopped at least 28 days prior to UCART19 administration. Participation in non-interventional registries or epidemiological studies is allowed

    12. CD19-negative B-cell leukaemia

    15. Burkitt cell acute leukaemia (L3 ALL)

    45a. Clinically suspected extramedullary involvement (except CNS and isolated skin involvement)

    37a. Evidence of disease progression after cytoreduction, if administered

    17a. Active CNS leukemia

    28. Clinically active significant CNS dysfunction

    29. Known history of severe neurological toxicity related to blinatumomab

    30. Primary immunodeficiency or bone marrow failure syndrome

    14. Weight< 8.8 kgs

    16a. Allogeneic HSCT within 6 months prior to screening; any donor lymphocyte infusions must be completed > 6 weeks prior to Screening


    31. Radioimmunotherapy, radiotherapy, within 8 weeks (except prophylaxis of CNS involvement) prior to Inclusion

    18. Use of rituximab and other anti CD20 antibodies known to have the same epitope as rituximab or anti CD20 for which the epitope is unknown within 3 months prior to UCART19 infusion

    19. Presence of donor-specific anti-HLA antibodies directed against UCART19

    20a. Active, acute or chronic GvHD requiring systemic use therapy

    21. Patients currently treated with immunosuppressive agents that cannot be stopped

    22. A known hypersensitivity to any of the test materials or related compounds including murine and bovine products

    24. Active systemic bacterial, fungal, protozoal or viral infection not controlled by adequate treatment, and presence of positive blood cultures within 7 days before Inclusion

    33. Patients tested positive for human immunodeficiency virus (HIV) and/or or human T-lymphotropic
    virus (HTLV)

    25a. Abnormal findings during the screening period, any other medical condition(s) or laboratory findings that in the opinion of the investigator, might jeopardize the patient’s safety

    26. Any planned medical/surgical treatment that might interfere with the ability to comply with the study requirements

    27a. Risk of pregnancy or non compliance with contraception (if applicable) .Girls of childbearing potential must have been tested negative in a pregnancy test within 7 days prior to inclusion. Within the frame of this study, female participants of childbearing potential and male participants with partners of childbearing potential must use an effective method of birth control, as well as their partners, from the screening period up to 12 months after the last dose of Investigational Medicinal Product (IMP) administration.

    36a. Any known contraindication to any of the drugs that will be used for the lymphodepletion (fludarabine, cyclophosphamide, alemtuzumab) or other drugs proposed for safety issues (including tocilizumab, rituximab).

    ELIGIBILITY CRITERIA FOR UCART19 ADMINISTRATION (WITHIN 24 HOURS PRIOR TO INFUSION)

    38a. Adequate organ functions including renal and hepatic function based on the last assessment performed within the lymphodepletion period, defined as:
    - Creatinine clearance ≥ 30 mL/min (as calculated using the method standard for the institution. In equivocal
    cases, a 24-hour urine collection test can be used to estimate the creatinine clearance more accurately.
    - Serum ALT/AST ≤ 3 x ULN
    - Total bilirubin ≤ 1.5 x ULN (unless the patient has a history of Gilbert’s Syndrome, in which case, total bilirubin must be ≤ 2.5 ULN)

    39. No clinical signs of cardiac failure

    40. No active systemic bacterial, fungal, protozoal or viral infection not controlled by adequate treatment, and absence of positive blood cultures within 7 days before UCART19 administration

    41a. No evidence of disease progression after lymphodepletion (e.g., significant increase in peripheral blast count)

    42. Baseline oxygen saturation level > 92% in room air

    43a. No Abnormal findings during the lymphodepletion period, neither any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the patient’s safety
    34. Pacientes que no desean ser sometidos a un seguimiento de seguridad durante 15 años.
    9.Mal cumplimiento previsible de los procedimientos del estudio
    10.Tratamiento previo con productos de terapia génica o terapia con células modificadas genéticamente. Se permite el tratamiento previo con blinatumomab.
    11a.Utilización de terapia anti-leucémica previa (incluyendo terapias aprobadas y otros productos en investigación) en un periodo de cinco semividas antes de la administración de UCART19. Se debe interrumpir el tratamiento con Inotuzumab ozogamicina al menos 28 días antes de la administración de UCART19. Se permite la participación en registros sin intervención o estudios epidemiológicos.
    12.Leucemia de células B CD19 negativas
    15.Leucemia aguda de células de Burkitt (LLA L3)
    45a.Sospecha clínica de afectación extramedular (excepto afectación del SNC y aislada de piel)
    37a.Evidencia de progresión de la enfermedad tras la citorreducción, si se ha administrado
    17a.Leucemia activa del SNC
    28.Disfunción significativa y clínicamente activa del SNC
    29.Antecedentes conocidos de toxicidad neurológica severa por blinatumomab
    30.Inmunodeficiencia primaria o síndrome de fracaso medular
    14.Peso < 8.8 kg
    16a.Trasplante alogénico de células madre hematopoyéticas en los 6 meses previos a la selección: cualquier infusión de linfocitos de donante debe haber terminado >6 semanas antes de la selección.
    31.Radioinmunoterapia, radioterapia, en las 8 semanas previas a la inclusión (excepto profilaxis de la afectación del SNC)
    18.Utilización de rituximab y otros anticuerpos antiCD20 de los que se sabe que tienen el mismo epítope que rituximab o antiCD20 de los que no se conoce el epítope, en los 3 meses previos a la infusión de UCART19.
    19.Presencia de anticuerpos anti-HLA específicos del donante dirigidos contra UCART19
    20.Enfermedad injerto contra huésped activa aguda o crónica que requiera terapia de uso sistémico.
    21.Pacientes actualmente en tratamiento con agentes inmunosupresores que no puedan ser interrumpidos
    22.Hipersensibilidad conocida a cualquiera de los materiales de prueba o compuestos relacionados incluyendo productos murinos y bovinos.
    24.Infección activa sistémica bacteriana, fúngica, protozoaria o vírica no controlada con tratamiento adecuado, y presencia de cultivos sanguíneos positivos en los 7 días previos a la inclusión.
    33.Pacientes positivos para el virus de la inmunodeficiencia humana (VIH) y/o virus linfotrópico de células T humanas (HTLV)
    25a.Hallazgos anormales durante el periodo de selección, cualquier enfermedad o hallazgo de laboratorio que, en opinión del investigador, pudiera comprometer la seguridad del paciente
    26.Cualquier tratamiento médico/quirúrgico que pudiera interferir con la capacidad de cumplir con los requisitos del estudio
    27a.Riesgo de embarazo o no cumplimiento con la contracepción (si es aplicable). Las niñas en edad fértil deben haber dado negativo en un test de embarazo en los 7 días previos a la inclusión. En el marco de este estudio, las participantes femeninas en edad fértil y los participantes masculinos con parejas en edad fértil deben utilizar un método efectivo de contracepción, así como sus parejas, desde el periodo de selección hasta 12 meses tras la última administración de la dosis del medicamento en investigación (MEI).
    36.Cualquier contraindicación conocida para cualquiera de los fármacos que se utilizarán para la linfodepleción (fludarabina, ciclofosfamida, alemtuzumab) u otros fármacos propuestos por seguridad (incluyendo tocilizumab, rituximab).
    Criterios de elegibilidad para la administración de UCART19 (en las 24 horas previas a la infusión)
    38.Función orgánica adecuada incluyendo función hepática y renal en base a la última evaluación realizada en el periodo de linfodepleción, definida como:
    - Aclaramiento de creatinina > 30 mL/min (calculado utilizando el método estándar del centro). En casos dudosos, se puede utilizar una prueba de recogida de orina de 24 horas para estimar el aclaramiento de creatinina con mayor precisión).
    - ALT/AST en suero < 3 x LSN
    - Bilirrubina total < 1,5 x LSN (salvo que el paciente tenga antecedentes de Síndrome de Gilbert, en cuyo caso, la bilirrubina total debe ser < 2,5 x LSN)
    39.Ausencia de signos clínicos de insuficiencia cardiaca
    40.Ausencia de infección activa sistémica bacteriana, fúngica, protozoaria o viral no controlada con tratamiento adecuado, y ausencia de cultivos sanguíneos positivos en los 7 días previos a la administración de UCART19.
    41a.Ausencia de evidencia de progresión de la enfermedad tras la linfodepleción (por ejemplo, aumento significativo del recuento de blastos periféricos)
    42.Nivel de saturación de oxígeno basal >92% en aire ambiente
    43a.Ausencia de hallazgos anormales durante el periodo de linfodepleción, y de cualquier otra enfermedad o hallazgo de laboratorio que, en la opinión del investigador, pudiera comprometer la seguridad del paciente.
    E.5 End points
    E.5.1Primary end point(s)
    - Adverse events graded according to the National Cancer Institute Common Toxicity Criteria for Adverse Event (NCI-CTCAE) version V4.3 June 14, 2010, throughout the study, except CRS, TLS and acute GvHD events that will be graded according to the grading systems of Lee, Cairo and Harris, respectively ;

    - Clinical examination and cardiac evaluation (ECG, echocardiography) ;

    - Vital signs (blood pressure, heart rate, body temperature, respiratory rate, oxygen saturation level),

    - Laboratory data assessments: haematology, biochemistry, coagulation parameters ;

    - Viral / bacterial / protozoal infection monitoring,
    - Acontecimientos adversos graduados según los criteros National Cancer Institute Common Toxicity Criteria for Adverse Event (NCI-CTCAE) version V4.3 Junio 14, 2010, durante el estudio, excepto ´síndrome de liberación de citoquinas, síndrome de lisis tumoral y enfemedad injerto contra huesped
    los cuales se graduarán siguiendo los sistemas de graduación deLee, Cairo y Harris respectivamente

    - Exploración clínica y evaluación cardiaca (ECG, ecocardiografía)

    - Signos vitales (tensión arterial, frecuencia cardiaca, frecuencia respiratoria, temperatura corporal, nivel de saturación de oxígeno)

    - Parámetros analíticos: hematología, coagulación y bioquímica.

    - Monitorización de infecciones por virus/bacterias/protozoos
    E.5.1.1Timepoint(s) of evaluation of this end point
    safety will be evaluated on an on-going basis throughout the trial
    La seguridad se evaluará de forma continua a lo largo del ensayo
    E.5.2Secondary end point(s)
    1- The activity of UCART19 will include a careful examination and valuation of the blood and the marrow if indicated.
    2- MRD evaluation by multiparameter flow cytometry and/or qPCR
    1- La actividad de UCART19 incluye análisis y evaluación de sangre minuciosa y en aspirado medular si está indicado.
    2- Enfermedad mínima residual mediante citometría de flujo y/o qPCR
    E.5.2.1Timepoint(s) of evaluation of this end point
    1: Day 28 after the first UCART19 infusion
    2: During the screening period, at D-1, (D14), D28, D56, D84, M4, M6, M9, M12
    1: Día 28 tras la administración de la infusión de UCART19
    2: Durante el periodo de screening, en D-1 (previo a la administración de UCART19), en los días (14), 28, 56, Y 84 post administración de UCART19 y en los meses 4, 6, 9 y q2 post-administración UCART19.
    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 Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Analysis of safety and/or efficacy biomarkers
    Análisis de seguridad y/o biomarcadores de eficacia
    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) 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 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 EEA4
    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
    Belgium
    France
    Spain
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    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: 18
    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: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    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 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
    minors subjects
    Sujetos menores
    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 13
    F.4.2.2In the whole clinical trial 18
    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)
    After the discontinuation of the study, the participant will access to an appropriate medical care by his doctor.
    After completion of the study or at time of premature discontinuation, patients will be rolled-over to a separate long-term follow-up study and will be followed for 15 years.
    Tras la discontinuación del estudio, los participantes recibirán los cuidados adecuados por parte de sus médicos.
    Tras completar este estudio o en caso de discontinuación del mismo de forma prematura, los pacientes entrarán en otro estudio de seguimiento a a largo plazo de 15 años de duración
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-18
    P. End of Trial
    P.End of Trial StatusOngoing
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