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    Summary
    EudraCT Number:2018-004383-65
    Sponsor's Protocol Code Number:SRK-015-002
    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:2019-04-12
    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 number2018-004383-65
    A.3Full title of the trial
    Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy
    Estudio de fase 2 con tratamiento activo para evaluar la eficacia y la seguridad de SRK-015 en pacientes con atrofia muscular espinal de inicio tardío .
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy
    Estudio Clinico de un nuevo posible tratamiento en pacientes con atrofia muscular espinal tipo 2 o 3.
    A.3.2Name or abbreviated title of the trial where available
    The TOPAZ study
    Estudio de TOPAZ
    A.4.1Sponsor's protocol code numberSRK-015-002
    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 SponsorScholar Rock, Inc.
    B.1.3.4CountryUnited States
    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 supportScholar Rock, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace UK
    B.5.2Functional name of contact pointBente Kristiansen
    B.5.3 Address:
    B.5.3.1Street AddressWallace House, 17-21 Maxwell Place
    B.5.3.2Town/ cityStirling
    B.5.3.3Post codeFK8 1JU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4540465215
    B.5.6E-mailRegSubmissions@Medpace.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/3/18/2115
    D.3 Description of the IMP
    D.3.1Product nameSRK-015
    D.3.4Pharmaceutical form Solution for injection/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 INNSRK-015
    D.3.9.2Current sponsor codeSRK-015
    D.3.9.3Other descriptive nameHUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
    D.3.9.4EV Substance CodeSUB193232
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Yes
    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.IMP: 2
    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/3/18/2115
    D.3 Description of the IMP
    D.3.1Product nameSRK-015
    D.3.4Pharmaceutical form Solution for injection/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 INNSRK-015
    D.3.9.2Current sponsor codeSRK-015
    D.3.9.3Other descriptive nameHUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
    D.3.9.4EV Substance CodeSUB193232
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Yes
    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
    Later Onset Spinal Muscular Atrophy (SMA)
    Atrofia Muscular Espinal de comienzo Tardío (AME)
    E.1.1.1Medical condition in easily understood language
    Later Onset Spinal Muscular Atrophy (SMA) is an inherited disease of the motor nerves (the nerves that send signals from your brain to the muscles) causing muscle weakness.
    La Atrofia Muscular Espinal (AME) de inicio tardío es una enfermedad hereditaria de los nervios motores (los nervios que envían señales desde el cerebro a los músculos) causando debilidad muscular.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079415
    E.1.2Term Spinal muscular atrophy type III
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079416
    E.1.2Term Spinal muscular atrophy type II
    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 assess safety and tolerability of SRK-015 in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)
    To assess the efficacy of SRK-015 by assessing changes in motor function outcome measures
    Evaluar la seguridad y la tolerabilidad de SRK 015 en pacientes con atrofia muscular espinal (AME) de inicio tardío (p. ej., tipo 2 y tipo 3).
    Evaluar la eficacia de SRK 015 mediante evaluación de las variaciones de los criterios de valoración de la función motora.
    E.2.2Secondary objectives of the trial
    To characterize the pharmacokinetics (PK) of SRK-015
    To evaluate the pharmacodynamic (PD) effects of SRK-015
    To evaluate time to therapeutic effect between low and high dose SRK-015 in a predefined cohort (Cohort 3)
    To evaluate the immunogenicity of SRK-015
    To evaluate the effect of SRK-015 on quality of life assessments
    Caracterizar la farmacocinética (FC) de SRK 015.
    Evaluar los efectos farmacodinámicos (FD) de SRK 015.
    Evaluar el tiempo que transcurre hasta el efecto terapéutico entre SRK 015 en dosis bajas y altas en una cohorte predefinida (cohorte 3).
    Evaluar la inmunogenicidad de SRK 015.
    Evaluar el efecto de SRK 015 sobre la calidad de vida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 5 through 21 years old at the time of screening for Cohorts 1 and 2; Age ≥2 years old at the time of screening for Cohort 3
    2. Estimated life expectancy >2 years from screening
    3. Informed consent document signed by the patient if the patient is legally an adult. If the patient is legally a minor, informed consent document signed by the patient’s parent or legal guardian and patient’s oral or written assent obtained, if applicable and in accordance with the regulatory and legal requirements of the participating location
    4. Documented diagnosis of 5q SMA
    5. Diagnosed as later onset (e.g., Type 2 or Type 3) SMA prior to receiving any treatment with therapy approved for SMA
    6. Non ambulatory patients must be able to sit independently (sits up straight with head erect for at least 10 seconds; does not use arms or hands to balance body or support position) per World Health Organization (WHO) motor milestones definition at screening. Patients who never had the ability to walk independently will be classified as Type 2. Patients who previously had the ability to walk unaided will be classified as Type 3.
    7. Ambulatory patients must have the ability to independently ambulate without aids or orthotics over 10 meters in 30 seconds or less at screening
    8. For Cohort 1, Revised Hammersmith Scale (RHS) score no greater than 63 at screening
    9. For Cohorts 2 and 3, Hammersmith Functional Motor Scale Expanded (HFMSE) score no less than 10 at screening
    10. Receiving the same background SMA therapy (e.g., on an approved survival motor neuron (SMN) upregulator therapy such as nusinersen, or not on any SMA therapy) for at least 6 months prior to screening and anticipated to remain on that therapy throughout the duration of the study
    10a. If receiving the SMN upregulator therapy nusinersen, must have completed the loading regimen and initiated maintenance dosing (i.e., completed at least one maintenance dose) with at least 4 weeks after the first maintenance dose having elapsed prior to screening
    11. Nutritional status stable over the past 6 months and anticipated to be stable throughout the duration of the study
    12. Have no physical limitations that would prevent the patient from undergoing motor function outcome measures throughout the duration of the study
    13. Able to receive study drug infusions and provide blood samples through the use of a peripheral intravenous (IV) or a long-term IV access device that the patient has placed for reasons independent from the study (i.e., for background medical care and not for the purpose of receiving SRK-015 in the study), throughout the duration of the study
    14. Able to adhere to the requirements of the protocol, including travel to the study center and completing all study procedures and study visits
    15. For patients who are expected to have reached reproductive maturity by the end of the study, adhere to study specific contraception requirements
    15a. Females of childbearing potential must have a negative pregnancy test at screening and agree to employ highly effective contraceptive measures (failure rate of 1% or less per year when used consistently and correctly) for the duration of the study and for 12 weeks following the last dose of study drug. Effective contraception methods are restricted to abstinence, intrauterine contraceptive devices, bilateral tubal occlusion, vasectomized partner, or a licensed hormonal product in combination with a barrier method.
    15b. Male patients with female partners of childbearing potential must be abstinent or agree to employ the use of a condom with or without spermicide throughout the duration of the study and for 12 weeks following the last dose of study drug.
    1.Edad de 5 a 21 años en el momento de la selección para las cohortes 1 y 2; edad ≥ 2 años en el momento de la selección para la cohorte 3.
    2.Esperanza de vida estimada más de 2 años desde la selección.
    3.Documento de consentimiento informado firmado por el paciente si tiene la mayoría legal de edad. Si el paciente es menor de edad legalmente, se obtendrá el consentimiento informado firmado por el progenitor o –representante legal del paciente y el asentimiento verbal o escrito del paciente, si procede y de conformidad con los requisitos legales y reglamentarios del centro participante.
    4.Diagnóstico documentado de AME 5q
    5.AME diagnosticada como de inicio tardía (p. ej., tipo 2 o tipo 3) antes de recibir cualquier tratamiento autorizado para la AME.
    6.Los pacientes que no tengan capacidad de ambulación deben ser capaces de sentarse de forma independiente (sentarse rectos con la cabeza erguida durante al menos 10 segundos; no utilizar los brazos ni las manos para equilibrar el cuerpo o mantener la posición) según la definición de los hitos motores de la Organización Mundial de la Salud (OMS) en la selección. Los pacientes que nunca hayan tenido la capacidad de caminar de forma independiente se clasificarán como de tipo 2. Los pacientes con capacidad previa para caminar sin ayuda se clasificarán como de tipo 3.
    7.Los pacientes con capacidad de ambulación deberán ser capaces de caminar de forma independiente sin ayudas ni aparatos ortopédicos durante 10 metros en 30 segundos o menos en la selección.
    8.Para la cohorte 1, puntuación no superior a 63 en la escala revisada de Hammersmith (RHS) en la selección.
    9.Paralas cohortes 2 y 3, puntuación en la escala de valoración Funcional motora de Hammersmith Expandida (HFMSE) no inferior a 10 en la selección.
    10.Haber recibido el mismo tratamiento de base para la AME (p. ej., con un tratamiento autorizado de sobreregulaciónde la proteína de supervivencia de motoneuronas (SMN), como nusinersén, o no haber recibido ningún tratamiento para la AME) durante al menos 6 meses antes de la selección y tener previsto continuar con dicho tratamiento durante todo el estudio.
    10a.En caso de recibir el tratamiento de sobreregulaciónde la proteína de supervivencia de motoneuronas con nusinersén, tendrá que haber finalizado la pauta de carga y haber iniciado la dosis de mantenimiento (es decir, haber finalizado al menos una dosis de mantenimiento) y deberán haber transcurrido, como mínimo, 4 semanas desde la primera dosis de mantenimiento antes de la selección.
    11.Estado nutricional estable durante los últimos 6 meses y previsión de que se mantenga estable durante todo el estudio.
    12.No presentar limitaciones físicas que impidan al paciente someterse a los procedimientos de los criterios de valoración de la función motora durante todo el estudio.
    13.Capacidad para recibir perfusiones del fármaco del estudio y proporcionar muestras de sangre mediante el uso de una vía intravenosa (in) periférica o a través de un dispositivo de acceso iv a largo plazo que el paciente lleve colocada por motivos independientes del estudio (es decir, para la asistencia médica de base y no para recibir SRK-015 en el estudio), durante todo el estudio.
    14.Capacidad de cumplir los requisitos del protocolo, entre otros, el desplazamiento al centro del estudio y la realización de todos los procedimientos y visitas del estudio.
    15.Para los pacientes que se prevé que hayan alcanzado la madurez reproductiva al final del estudio, tienen que cumplir los requisitos de anticoncepción específicos del estudio.
    15a.Las mujeres en edad fértil deberán tener una prueba de embarazo negativa en la selección y comprometerse a utilizar métodos anticonceptivos muy eficaces (tasa de fallo del 1 % o menos al año cuando se utilicen de forma constante y correcta) durante todo el estudio y durante 12 semanas posteriores a la última dosis del fármaco del estudio. Los métodos anticonceptivos eficaces se limitan a la abstinencia, los dispositivos intrauterinos, la oclusión tubaria bilateral, la vasectomía de la pareja o un producto hormonal autorizado en combinación con un método de barrera.
    15b.Los varones con parejas en edad fértil deberán practicar la abstinencia o comprometerse a utilizar preservativo con o sin espermicida durante todo el estudio y hasta 12 semanas después de la última dosis del fármaco del estudio.
    E.4Principal exclusion criteria
    1. Use of tracheostomy with positive pressure
    2. Use of chronic daytime non invasive ventilatory support for >16 hours daily in the 2 weeks prior to dosing, or anticipated to regularly receive such daytime ventilator support chronically over the duration of the study
    3. Any acute or co morbid condition interfering with the well being of the patient within 14 days of screening, including active systemic infection, the need for acute treatment or inpatient observation due to any reason
    4. Severe scoliosis and/or contractures at screening. Based on clinical judgement, any scoliosis or contractures present must be stable over the past 6 months, anticipated to be stable for the duration of the study and not prevent the patient from being evaluated on any functional outcome measures throughout the duration of the study.
    5. Pregnant or breastfeeding
    6. Major orthopedic or other interventional procedure, including spine or hip surgery, considered to have the potential to substantially limit the ability of the patient to be evaluated on any functional outcome measures, within 6 months prior to screening, or anticipated for the duration of the study
    7. Prior history of a hypersensitivity reaction to a monoclonal antibody or recombinant protein bearing an Fc domain (such as a soluble receptor-Fc fusion protein)
    8. Use of systemic corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
    9. Treatment with investigational drugs within 3 months prior to screening
    10. Use of therapies with potentially significant muscle effects (such as androgens, insulin like growth factor, growth hormone, systemic beta agonist, botulinum toxin, or muscle relaxants) or muscle-enhancing supplements within 60 days prior to screening
    11. Patient has any other condition, which in the opinion of the Investigator may compromise safety or compliance, would preclude the patient from successful completion of the study, or interfere with the interpretation of the results
    1.Uso de traqueostomía con presión positiva.
    2.Uso diurno crónico de respiración asistida no invasiva durante más de 16 horas al día en las 2 semanas anteriores a la administración de la dosis, o previsión de tener periódicamente respiración asistida diurna de forma crónica durante el estudio.
    3.Cualquier enfermedad aguda o concomitante que interfiera con el bienestar del paciente en los 14 días anteriores a la selección, como una infección sistémica activa, la necesidad de tratamiento agudo u observación intrahospitalaria por cualquier motivo.
    4.Escoliosis severa y/o contracturas en la selección. Según el criterio clínico, todas las escoliosis o contracturas presentes deberán permanecer estables durante los últimos 6 meses, estar previsto que se mantengan estables durante todo el estudio y que no impidan que se pueda evaluar al paciente en relación con criterios de valoración funcionales durante todo el estudio.
    5.Embarazo o lactancia
    6.Cirugía ortopédica mayor u otro procedimiento intervencionista, incluida la cirugía de la columna o la cadera, que se considere que tienen el potencial de limitar sustancialmente la capacidad del paciente para ser evaluado con cualquier criterio de valoración funcional, en los 6 meses previos a la selección, o que se prevé durante el estudio.
    7.Antecedentes de reacción de hipersensibilidad a un anticuerpo monoclonal (AcM) o a una proteína recombinante portadora de un dominio Fc (como una proteína de fusión del receptor soluble-Fc).
    8.Uso de corticosteroides sistémicos en los 60 días previos a la selección. Se permiten los esteroides inhalados o tópicos.
    9.Tratamiento con fármacos en investigación en los 3 meses previos a la selección.
    10.Uso de tratamientos con efectos musculares potencialmente importantes (como andrógenos, factor de crecimiento similar a la insulina, hormona de crecimiento, agonista beta sistémico, toxina botulínica o relajantes musculares) o suplementos que potencien el músculo en los 60 días previos a la selección.
    11.El paciente presenta cualquier otra enfermedad que, en opinión del investigador, pueda poner en peligro su seguridad o cumplimiento, impida –su finalización satisfactoria del estudio o interfiera en la interpretación de los resultados.
    E.5 End points
    E.5.1Primary end point(s)
    Cohort 1; Ambulatory Type 3 patients:
    Change from Baseline in the revised Hammersmith Scale (RHS).
    Administration of the RHS includes the timed rise from floor and 10 meter walk/run tests. The RHS is designed for SMA, validated in SMA, and considered clinically meaningful.

    Cohort 2 and Cohort 3;Type 2 and non ambulatory Type 3 patients:
    Change from Baseline in Hammersmith Functional Motor Skills Expanded (HFMSE) Scores. HFMSE is designed for SMA, validated in SMA, and considered clinically meaningful.
    Cohorte 1: Pacientes con capacidad ambulatoria de tipo 3
    Variación de la puntuación RHS con respecto al momento basal.
    La administración del RHS incluye una variación con respecto al valor basal de la prueba de marcha/carrera de 10 metros . El RHS está diseñado para AME, validado para AME y considerado clínicamente significativo.

    Cohorte 2 y cohorte 3: Pacientes de tipo 2 y de tipo 3 sin capacidad ambulatoria
    Variación con respecto al momento basal de la puntuación HFMSE. HFMSE está diseñado para AME, validado para AME y considerado clínicamente significativo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ongoing throughout the study
    En curso a lo largo del estudio.
    E.5.2Secondary end point(s)
    Cohort 1; Ambulatory Type 3 patients:
    Proportion of patients achieving various magnitudes of change in RHS score from Baseline
    Change from Baseline in 6 minute walk test (6MWT)
    Change from Baseline in 30 second sit to stand
    Change from Baseline in 10 meter walk/run (from the RHS)
    Change from Baseline in timed rise from floor (from the RHS)

    Cohort 2 and Cohort 3;Type 2 and non ambulatory Type 3 patients:
    Proportion of patients achieving various magnitudes of change in HFMSE score from Baseline
    Change from Baseline in Revised Upper Limb Module (RULM)
    Change from Baseline in number of WHO motor development milestones attained
    Proportion of patients achieving various magnitudes of change in RULM score from Baseline
    Proportion of patients who attain a new WHO motor development milestone relative to Baseline
    Cohorte 1: Pacientes con capacidad ambulatoria de tipo 3
    Proporción de pacientes que alcanzan diversas magnitudes de variación de la puntuación RHS con respecto al momento basal.
    Variación con respecto al valor basal de la prueba de marchade 6 minutos
    Variación con respecto al momento basal de la posición de sedestación a bipedestación en 30 segundos.
    Variación con respecto al valor basal de la prueba de marcha/carrera de 10 metros (con respecto a la RHS)
    Variación con respecto al valor basal del tiempo para levantarse del suelo (con respecto a la RHS).
    Cohorte 2 y cohorte 3: Pacientes de tipo 2 y de tipo 3 sin capacidad ambulatoria
    Proporción de pacientes que alcanzan diversas magnitudes de variación de la puntuación HFMSE con respecto al momento basal.
    Variación con respecto al valor basal del módulo revisado para la evaluación de las extremidades superiores (RULM).
    Variación con respecto al momento basal del número de hitos del desarrollo motor de la OMS alcanzados.
    Proporción de pacientes que alcanzan diversas magnitudes de variación de la puntuación RULM con respecto al momento basal.
    Proporción de pacientes que alcanzan un nuevo hito de desarrollo motor de la OMS con respecto al momento basal.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ongoing throughout the study
    En curso a lo largo del estudio.
    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 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 Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    Cohort 1 and 2 are open label and Cohort 3 is double blind randomized
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    For Cohort 3: two different doses (2 and 20 Mg/kg) of the study drug
    E.8.2.4Number of treatment arms in the trial2
    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 EEA8
    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
    Canada
    Germany
    Italy
    Netherlands
    Spain
    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
    LVLS
    Ultima visita del ultimo 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 months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months11
    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: 51
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 35
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 16
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    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 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 16
    F.4.2.2In the whole clinical trial 55
    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)
    Patients that complete the 52-week treatment period may have the option to roll into an open-label extension study under a separate protocol.
    Los pacientes que finalicen el período de tratamiento de 52 semanas tendrán la opción de incorporarse a un estudio de extensión abierto con un protocolo aparte.
    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 Decision2019-06-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-28
    P. End of Trial
    P.End of Trial StatusOngoing
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