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    Summary
    EudraCT Number:2017-001747-12
    Sponsor's Protocol Code Number:CSEG101B2201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Restarted
    Date on which this record was first entered in the EudraCT database:2019-01-23
    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 number2017-001747-12
    A.3Full title of the trial
    A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of
    Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential,
    Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis
    Estudio de fase 2, multicéntrico, abierto para evaluar la dosis adecuada y la seguridad de crizanlizumab, con o sin hidroxiurea/hydroxicarbamida, en grupos secuenciales de edad descendente de pacientes pediátricos con Enfermedad de Células Falciformes con crisis vaso-oclusivas.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
    Estudio de confirmación y seguridad de la dosis de crizanlizumab en pacientes pediátricos con Enfermedad de Células Falciformes.
    A.4.1Sponsor's protocol code numberCSEG101B2201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03474965
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/077/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Farmacéutica, S.A.
    B.1.3.4CountrySpain
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farmacéutica, S.A.
    B.5.2Functional name of contact pointTrial Monitoring Organization (TMo)
    B.5.3 Address:
    B.5.3.1Street AddressGran Vía de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+34 90 0353036
    B.5.5Fax number+34 93 2479903
    B.5.6E-maileecc.novartis@novartis.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/3/12/1034
    D.3 Description of the IMP
    D.3.1Product namecrizanlizumab
    D.3.2Product code SEG101
    D.3.4Pharmaceutical form Concentrate for solution for 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 INNCRIZANLIZUMAB
    D.3.9.2Current sponsor codeSEG101
    D.3.9.4EV Substance CodeSUB188615
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle Cell Disease with Vaso-Occlusive Crisis
    Enfermedad de Células Falciformes con crisis vaso-oclusivas.
    E.1.1.1Medical condition in easily understood language
    sickle cell disease
    Enfermedad de Células Falciformes
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10002077
    E.1.2Term Anaemia sickle cell
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To confirm and establish appropriate dosing of crizanlizumab in patients ages 6 months to <18
    years at the time of study entry (Part A and B)
    - To evaluate the safety of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Parts A and B)
    -Confirmar y establecer la dosis adecuada en pacientes con edades comprendidas entre los 6 meses hasta <18 años en el momento de entrada al estudio (partes A y B).
    - Evaluar la seguridad de crizanlizumab en pacientes con edades comprendidas entre los 6 meses hasta < 18 años (partes A y B).
    E.2.2Secondary objectives of the trial
    - To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)
    - To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry
    - To characterize long-term PK and PD of crizanlizumab in patients ages 6 months to <18 years at the time of study entry
    -Evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre los 6 meses hasta <18 años en el momento de entrada al estudio(partes A y B)
    -Evaluar otras medidas de seguridad en pacientes con edades comprendidas entre los 6 meses hasta <18 años en el momento de entrada al estudio
    Caracterizar la PK y PD de crizanlizumab a largo plazo en pacientes con edades comprendidas entre los 6 meses hasta < 18 años en el momento de entrada al estudio
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Male or female patients aged 2 to <18 years (Group 3 will be expanded to allow enrolment of patients aged 6 to <24 months (and at least 6 kg) in Part B once the appropriate dose is confirmed in 2 to <6 year old patients)
    •Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbSβ0-thalassemia, HbSβ+-thalassemia, and others) by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) performed locally.
    •Experienced at least 1 VOC within the preceding 12 months, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and should include all the following:
    1.the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1)
    2.either a visit to a medical facility or healthcare professional,
    3.receipt of oral/parenteral opioid or other non-opioid parenteral analgesia.
    •If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening and plan to continue taking at the same dose and schedule during the trial. Dose alterations of HU/HC during Part A are not allowed, and if this occurs, the patient will enter directly to the Part B.
    •Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education
    •Transcranial Doppler (TCD) considered low risk within the past 6 months (for 2 to 16 years).

    Other inclusion criteria as per protocol may apply
    1. Pacientes de ambos sexos (hombres o mujeres) entre los 2 hasta <18 años de edad (se ampliará el grupo 3 para permitir la inclusión de pacientes entre los 6 años hasta < 24 meses [que pesen al menos 6 kg] en la parte B una vez se confirme la dosis adecuada en los pacientes entre los 2 hasta < 6 años de edad).
    2. Diagnóstico confirmado de enfermedad de células falciformes (ECF) (p. ej., cualquier genotipo incluyendo HbSS, HbSC, HbSβ0 talasemia, HbSβ+ talasemia y otros) mediante electroféresis de hemoglobinas o cromatografía líquida de alta resolución (HPLC) realizada de forma local.
    3. Que hayan experimentado al menos una CVO durante los últimos 12 meses, según se determine mediante la historia clínica. La CVO previa se ha tenido que resolver al menos 7 días antes de la primera dosis del estudio y debe incluir todo lo siguiente:
    a. Aparición de síntomas correspondientes (véase la definición de CVO en el apartado 7.2.1.1).
    b. Una visita a un centro médico o a un profesional sanitario.
    c. Haber recibido un opioide oral/parenteral o cualquier analgesia parenteral no opioide.
    4. Si está recibiendo HU/HC o un agente estimulante de eritropoyetina, debe haber estado recibiendo el fármaco durante al menos 6 meses antes de la selección y tener previsto continuar tomando la misma dosis con la misma pauta durante el estudio.
    5. Haber recibido asistencia estándar adecuada a su edad para la ECF incluyendo profilaxis de penicilina, inmunización neumocócica y educación parental.
    6. Doppler transcraneal (DTC) considerado de bajo riesgo durante los 6 meses previos (para edades entre los 2 y 16 años).

    Otros creiterios de inclusion pueden aplicar segun protocolo.
    E.4Principal exclusion criteria
    •History of stem cell transplant.
    •Received any blood products within 30 days of Day 1 dosing.
    •Participating and maintaining in a chronic transfusion program (preplanned series of transfusions for prophylactic purposes).
    •Patients with bleeding disorders
    •Planning on undergoing an exchange transfusion during the duration of the study. Patients requiring episodic transfusion in response to worsened anemia or VOC are permitted.
    •Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation.
    •Planning to initiate or terminate HU/HC while on study, other than for safety reasons
    •Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator.

    Other exclusion criteria as per protocol may apply
    1. Antecedente de trasplante de células madre.
    2. Haber recibido algún hemoderivado durante los 30 días previos a la administración de la dosis del día 1.
    3. Participación y continuación en un programa de transfusión crónico (series programadas de transfusiones con finalidad profiláctica ).
    4. Pacientes con trastornos de sangrado.
    5. Tener programado someterse a una exanguinotransfusión durante el transcurso del estudio. Se permiten pacientes que requieran una transfusión puntual como respuesta a un empeoramiento de la anemia o por CVO.
    6. Contraindicación o hipersensibilidad a algún fármaco de clase similar al fármaco del estudio o a cualquier excipiente de la formulación del fármaco del estudio.
    7. Tener programado iniciar o finalizar HU/HC durante el estudio, por otros motivos que no sean de seguridad.
    8. Infección o inmunodeficiencia activa significativa (incluyendo el uso crónico de fármacos inmunosupresores) según el criterio del investigador.

    Otros creiterios de exclusion pueden aplicar segun protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    1.PK (AUCd15) after 1st dose
    Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A)


    2.PD (AUCd15) after 1st dose
    Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A)


    3.PK (AUCtau) after 5th dose
    Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old


    4.PD (AUCtau) after 5th dose
    Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old


    5.PK (Cmax) after 1st dose and 5th dose
    Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A)


    6.PK pre-dose concentrations
    Confirm appropriate dosing of crizanlizumab in patients aged 6 months to less than 24 months of age (Part B)


    7.Frequency of any adverse events (AEs) as a measure of safety and tolerability
    Safety of crizanlizumab in patients aged 6 months to < 18 years (Parts A and B)
    1. Parámetros de PK (AUCd15)después de la dosis inicial.
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.(Partes A)
    2. Parámetro de PD (AUCd15) después de la dosis inicial.
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.(Partes A)
    3. Parámetros de PK (AUCtau) después de la quinta dosis.
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.
    4. Parámetros de PD (AUCtau) después de la quinta dosis.
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años
    5.Parámetros de PK (Cmax) después de la dosis inicial y después de la quinta dosis.
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.(Partes A)
    6. Concentraciones Predosis de PK
    Confirmar la dosis apropiada de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.(Parte B)
    7. Frecuencia de cualquier evento adverso (EAs) como medida de la seguridad y tolerabilidad
    Seguridad de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años.(Partes A y B)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Day 15
    2. Day 15
    3. Week 15
    4. Week 15
    5. Week 1 and Week 15
    6. Week 3 and Week 19
    7. 6 months, 2 years
    1. Dia 15
    2. Dia 15
    3. Semana 15
    4. Semana 15
    5. Semana 1 y Semana 15
    6. Semana 3 ySemana 19
    7. 6 meses, 2 años
    E.5.2Secondary end point(s)
    1.Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    2.Number of Vaso Occusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    3.Number of each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    4.Number of hospitalizations and ER visits (both overall and VOC-related)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    5.Number of days of ER/hospitalization (both overall and VOC-related)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    6.Number of dactylitis events
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)


    7.Number, seriousness, severity, and causality assessments of treatement emergent adverse events and other data as considered appropiate.
    To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry


    8.Absolute change from baseline in hemoglobin
    To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry


    9.Immunogenicity: measurement of anti-drug antibodies (ADA) to crizanlizumab
    To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry


    10.Electrocardiogram (ECGs) at relevant PK time points
    To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry


    11.Growth and sexual maturation assessments (Tanner stage)
    To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry


    12.PK pre-dose concentrations prior to each study drug dose.
    Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years


    13.PD pre-dose concentrations prior to each study drug dose.
    Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years


    14.Percentage P-selectin inhibition prior to dosing
    Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years
    1. Número de CVO que deriven en visitas a clínica/urgencias/hospital,
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    2. Número de CVO tratadas en el domicilio (basado en la documentación del seguimiento telefónico del paciente por parte del profesional sanitario).
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    3. Número de cada subcategoría de CVO (crisis de dolor no complicada, síndrome agudo coronario, secuestro hepático, secuestro esplénico, priapismo),
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    4. Número de hospitalizaciones y visitas a urgencias (generales y relacionadas con CVO),
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    5. Número de días en urgencias/hospitalización (general y relacionados con las CVO),
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    6. Número de acontecimientos de dactilitis.
    Para evaluar la eficacia a largo plazo de crizanlizumab en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio (Partes A y B)
    7. Número, gravedad, severidad y evaluaciones de causalidad de los eventos adversos emergentes del tratamiento y otros datos que se consideren apropiados.
    Para evaluar otras medidas de seguridad en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio.
    8. Cambio absoluto respecto a la basal en la hemoglobina
    Para evaluar otras medidas de seguridad en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio.
    9. Inmunogenicidad: medición de anticuerpos contra el fármaco (ADA) crizanlizumab.
    Para evaluar otras medidas de seguridad en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio.
    10. ECG en momentos de PK relevantes
    Para evaluar otras medidas de seguridad en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio.
    11. Evaluaciones de crecimiento y maduración sexual (estadio de Tanner).
    Para evaluar otras medidas de seguridad en pacientes con edades comprendidas entre 2 meses y hasta < 18 años a la entrada del estudio.
    12. Predosis de PK (concentración del fármaco) antes de cada dosis de cada estudio.
    Caracterizar la PK y la PD de crizanlizumab a largo plazo en pacientes con edades comprendidas entre 2 meses y hasta < 18 años
    13. Predosis de PD (concentración del fármaco) antes de cada dosis de cada estudio
    Caracterizar la PK y la PD de crizanlizumab a largo plazo en pacientes con edades comprendidas entre 2 meses y hasta < 18 años
    14. Porcentaje de inhibición de P-selectina antes de cada dosis .
    Caracterizar la PK y la PD de crizanlizumab a largo plazo en pacientes con edades comprendidas entre 2 meses y hasta < 18 años
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 6 months, 2 years
    2. 6 months, 2 years
    3. 6 months, 2 years
    4. 6 months, 2 years
    5. 6 months, 2 years
    6. 6 months, 2 years
    7. 6 months, 2 years
    8. 6 months, 2 years
    9. Week 1, Week 3, Week 15, Week 27 and End of Treatment (EOT)
    10. Screening, Week 7, Week 11, week 15, week 27 and Week 51
    11. Screening, Week 51 and End of Treatment (EOT)
    12. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51
    13. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51
    14. Week 3, Week 15, Week 27 and Week 51
    1. 6 meses, 2 años
    2. 6 meses, 2 años
    3. 6 meses, 2 años
    4. 6 meses, 2 años
    5. 6 meses, 2 años
    6. 6 meses, 2 años
    7. 6 meses, 2 años
    8. 6 meses, 2 años
    9. Semana 1, 3, 15, 27 y Final del tratamiento (EOT)
    10. Selección, Semana 7, 11, 15, 27 y 51
    11. Selección, Semana 51 y Final del tratamiento (EOT)
    12. Semana 1, 3, 7, 15, 19, 23, 27, 31, 35, 39, 43, 47 y 51
    13. Semana 1, 3, 7, 15, 19, 23, 27, 31, 35, 39, 43, 47 y 51
    14. Semana 3, 15, 27 y 51
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) 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) Yes
    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.8.2.4Number of treatment arms in the trial3
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Brazil
    Canada
    Colombia
    France
    Germany
    India
    Italy
    Lebanon
    Oman
    Spain
    Switzerland
    Turkey
    United Kingdom
    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
    The end of the study will occur when all the patients have either completed or discontinued the
    study treatment and/or the 105-days follow-up period.
    El fin del estudio se producirá cuando todos los pacientes hayan completado o interrumpido el tratamiento del estudio y / o el período de seguimiento de 105 días.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months7
    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 months7
    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: 100
    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: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 44
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 100
    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)
    Novartis will make every effort to supply SEG101 to patients who may benefit from continued treatment after the end of study, as per the Investigator’s opinion; Novartis will provide the study treatment to the investigator as per local regulations and safety will be monitored and reported to the Health Authorities per regulatory requirements.
    Novartis hará todo lo posible para suministrar SEG101 a los pacientes que puedan beneficiarse de un tratamiento continuado después del fin de estudio, según la opinión del investigador; Novartis proporcionará el tratamiento del estudio al investigador según las regulaciones locales y la seguridad se monitorizará y se informará a las Autoridades Sanitarias según los requisitos reglamentarios.
    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 Decision2018-12-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-11
    P. End of Trial
    P.End of Trial StatusRestarted
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