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    The EU Clinical Trials Register currently displays   43974   clinical trials with a EudraCT protocol, of which   7311   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2016-001060-11
    Sponsor's Protocol Code Number:FSJD-RTB-2015
    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:2017-05-23
    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 number2016-001060-11
    A.3Full title of the trial
    A Phase I, unicentric, open and dose escalation clinical trial to evaluate the safety and the activity of the oncolytic adenovirus VCN-01 in patients with refractory retinoblastoma
    Ensayo clínico fase I, unicéntrico, abierto y de escalado de dosis, para evaluar la seguridad y la actividad del adenovirus oncolítico VCN-01 en pacientes con retinoblastoma refractario
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I clinical trial to evaluate the safety and the activity of the oncolytic adenovirus VCN-01 in patients with refractory retinoblastoma
    Ensayo clínico fase I para evaluar la seguridad y la actividad del adenovirus oncolítico VCN-01 en pacientes con retinoblastoma refractario
    A.4.1Sponsor's protocol code numberFSJD-RTB-2015
    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 SponsorFundació Sant Joan de Déu
    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 supportFondo del Ministerio de Sanidad y Política Social.
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSERMES PLANIFICACION S.L
    B.5.2Functional name of contact pointUnidad de Puesta en Marcha
    B.5.3 Address:
    B.5.3.1Street AddressRufino González 14, Esc.1ª-2ºD
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28037
    B.5.3.4CountrySpain
    B.5.4Telephone number+34913756930
    B.5.5Fax number+34917542721
    B.5.6E-mailstart-up@sermescro.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 nameVCN-01
    D.3.2Product code VCN-01
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVCN-01
    D.3.9.2Current sponsor codeVCN-01
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1800000000000
    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 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 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
    Refractory Retinoblastoma
    Retinoblastoma refractario.
    E.1.1.1Medical condition in easily understood language
    Retinoblastoma
    Retinoblastoma
    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 PT
    E.1.2Classification code 10038916
    E.1.2Term Retinoblastoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determinate safety and tolerability of two intravitreal injections of VCN-01 through the evaluation of the adverse events and the laboratory tests to set the maximum tolerated dose (MTD) of the VCN-01 virus.
    Determinar seguridad y tolerabilidad de dos inyecciones intravítreas de VCN-01 a través de la evaluación de los acontecimientos adversos y los análisis de laboratorio, para establecer la dosis máxima tolerada (MTD) del virus VCN-01.
    E.2.2Secondary objectives of the trial
    - To evaluate the anti-tumor response of VCN-01 in patients with advanced intraocular retinoblastoma with vitreous seeding massive within the limitations imposed by the phase I design of the study.
    - To evaluate the excretion profile of VCN-01.
    - To evaluate the immune response by the presence of neutralizing antibodies in the patients of the study.
    -Evaluar la respuesta antitumoral de VCN-01 en pacientes con retinoblastoma intraocular avanzado con siembras vítreas masivas dentro de las limitaciones que impone el diseño de fase I del estudio.
    -Evaluar el perfil de excreción de VCN-01.
    -Evaluar la respuesta inmune mediante la presencia de anticuerpos neutralizantes en los pacientes del estudio.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients with retinoblastoma both with somatic mutation of the RB1gen with active tumor in a single eye, as with germinal mutation of the RB1gen with active tumor in a single eye and the not affected fellow eye or with active tumor in a single eye and the fellow eye already enucleated relapsed or refractories with the use of systemic chemotherapy, intra-arterial or intravitreal or radiotherapy in whom enucleation is the only advisable treatment.
    2. Normal renal function: serum creatinine: <45µmol/L (0-2 years); <57µmol/L (3-6 years); <60µmol/L (7-10 years); <80µmol/L (11-13 years).
    3. Normal liver function: serum ALT: <0,52µkat/L (from 9 months to 12 years); serum AST: 61-80 g/L (from 8 months to 5 years); 63-83 g/L (5-9 years); 63-82 g/L (9-12 years).
    4. Adequate bone-marrow reserve expressed in an absolute neutrophil count > 1000/mm3, platelets >100.000/mm3 and hemoglobin >8 g/dl, without transfusional support.
    5. Older than one year and youger than 12 years at the time of inclusion in the study.
    6. Signed informed consent form.
    1.Pacientes con retinoblastoma tanto con mutación somática del genRB1 con tumor activo en un solo ojo, como con mutación germinal del RB1 con tumor activo en un solo ojo y el ojo contralateral no afectado o con tumor activo en un solo ojo y el ojo contralateral ya enucleado recaídos o refractarios con el uso de quimioterapia sistémica, intra-arterial o intravítrea o radioterapia en quienes la enucleación sea el único tratamiento recomendable.
    2.Función renal normal: creatinina en suero: <45µmol/L (0-2 años); <57µmol/L (3-6 años); <60µmol/L (7-10 años); <80µmol/L (11-13 años).
    3.Función hepática normal: ALT en suero: <0,52µkat/L (de 9 meses a 12 años); AST en suero: 61-80 g/L (de 8 meses a 5 años); 63-83 g/L (5-9años); 63-82 g/L (9-12 años)
    4.Adecuada reserva medular manifestada en un recuento absoluto de neutrófilos > 1000/mm3, plaquetas >100.000/mm3 y hemoglobina >8 g/dl, sin apoyo transfusional ni de citoquinas por lo menos un mes antes del ingreso al estudio.
    5.Edad mayor de un año y menor de 12 años en el momento de la inclusión en el estudio.
    6.Formulario de consentimiento informado firmado.
    E.4Principal exclusion criteria
    1. Presence of factors that require immediate enucleation of the affected eye as can be glaucoma, rubeosis iridis, commitment on the anterior chamber.
    2. Comorbidity situations: uncontrolled epilepsy with anticonvulsant treatment, no compensated cardiological disease with the treatment.
    3. Active infections.
    4. Other chronic or acute active diseases that would be an exclusion criteria under the criterion of the investigator.
    5. Having received attenuated or live vaccines background in the 30 days prior to the inclusion in the study.
    6. Immunosuppression of any cause.
    7. Trilateral retinoblastoma.
    8. Extraocular extension.
    9. Antecedent of having received treatment for retinoblastoma with chemotherapy or radiotherapy by any route in the 30 days before the inclusion in the study.
    10. Patients who cannot complete the study procedures for psychological or social reasons.
    11. Pregnancy. Female potential of childbearing patients should agree with doing a blood or urine pregnancy test and the result shall be negative to enter into the study.
    1.Presencia de factores que requieran enucleación inmediata de ojo afectado como pueden ser glaucoma, rubeosis iridis, compromiso de la cámara anterior.
    2.Situaciones de comorbilidad: Epilepsia no controlada con el tratamiento anticonvulsivante, enfermedad cardiológica no compensada con el tratamiento.
    3.Infecciones activas.
    4.Otras enfermedades crónicas o agudas activas que bajo criterio del investigador fuera un criterio de exclusión.
    5.Antecedente de haber recibido vacunas atenuadas o vivas en los 30 días previos a la inclusión en el estudio.
    6.Inmunosupresión de cualquier causa.
    7.Retinoblastoma trilateral.
    8.Diseminación extraocular.
    9.Antecedente de haber recibido tratamiento para el retinoblastoma con quimioterapia o radioterapia por cualquier vía en los 30 días previos a la inclusión en el estudio.
    10.Pacientes que no puedan completar los procedimientos del estudio por razones psicológicas o sociales.
    11.Embarazo. Las pacientes de sexo femenino con potencial de procrear deben estar de acuerdo en someterse a una prueba de embarazo en sangre u orina y el resultado debe ser negativo para entrar en el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability: Safety and tolerability of two intravitreal injections of VCN-01.
    Seguridad y tolerabilidad: La seguridad y tolerabilidad de dos inyecciones intravítreas de VCN-01
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout all study
    A lo largo de todo el estudio
    E.5.2Secondary end point(s)
    *Tumor response rate (TRT) to VCN-01 28 days after administration. For this purpose it shall be defined as:
    - Response to treatment: do not meet progressive disease (PD) criteria and notes inactivation, calcification or reduction volume in at least a vitreous seeding or retinal tumor
    - Progressive disease (PD): clear progression of the disease, marked by an increase of tumor volume, of retinal detachment, of vitreous seeding or commitment on the anterior chamber
    - Stable disease: no changes to the current situation for inclusion in the study

    * Presence of the virus in blood samples, aqueous humor, swab of ocular or nasal surface and its difference between viral particles before and after the treatment to evaluate the excretion profile of the VCN-01

    * Presence of neutralizing antibodies in blood samples in the patients of the study to evaluate the immune response
    * Tasa de respuesta tumoral (ORR) al VCN-01 a los 28 días post-administración. Para tal fin se definirá como;
    - Respuesta al tratamiento: no cumple criterios de enfermedad progresiva (EP) y se observa inactivación, calcificación o reducción del volumen de al menos una siembra vítrea o tumor retiniano
    - Enfermedad progresiva (EP): Evidente progresión de enfermedad, manifestada por aumento del volumen tumoral, del desprendimiento de retina, de las siembras vítreas o subretinianas. Presencia de glaucoma o compromiso de la cámara anterior
    - Enfermedad estable: Sin cambios con respecto a la situación a la inclusión en el estudio

    * Presencia del virus en muestras de sangre, humor acuoso, hisopo de superficie ocular y nasal, y su diferencia entre la partículas virales antes y después del tratamiento para evaluar el perfil de excreción del VCN-01

    * La presencia de anticuerpos neutralizantes en muestras de sangre en los pacientes del estudio para evaluar la respuesta inmune
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0, 1, 2, 7, 15, 16, 21, 28, 42 y 180
    Día 0, 1, 2, 7, 15, 16, 21, 28, 42 y 180
    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 Yes
    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
    Establish safety and tolerability in pediatric population by means of dose escalation
    Establecer seguridad y tolerabilidad en población pediátrica mediante escalado de dosis
    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 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
    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 months1
    E.8.9.1In the Member State concerned 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: 13
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 11
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    pediatric patients
    Pacientes pediátricos
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state13
    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 Decision2017-08-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-02
    P. End of Trial
    P.End of Trial StatusOngoing
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