E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
RSV disease |
Enfermedad por VSR |
|
E.1.1.1 | Medical condition in easily understood language |
respiratory viral disease |
enfermedad viral respiratoria |
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E.1.1.2 | Therapeutic area | Diseases [C] - Virus Diseases [C02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066742 |
E.1.2 | Term | Respiratory syncytial virus infection prophylaxis |
E.1.2 | System Organ Class | 100000004865 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of nirsevimab when administered to immunocompromised children ≤ 24 months of age |
Evaluar la seguridad y tolerabilidad de nirsevimab cuando se administra a niños inmunodeprimidos ≤ 24 meses de edad. |
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E.2.2 | Secondary objectives of the trial |
PK: To evaluate the PK of nirsevimab ADA: To evaluate ADA responses to nirsevimab in serum Efficacy: To assess the efficacy of nirsevimab when administered as a single IM dose to infants ≤ 24 months of age |
PK: para evaluar la PK de nirsevimab ADA: para evaluar las respuestas de ADA al nirsevimab en suero Eficacia: para evaluar la eficacia de nirsevimab cuando se administra como dosis única IM a lactantes ≤ 24 meses de edad. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Neonate, infant, or young child ≤ 24 months of age who, per investigator judgment, are: (a) In their first year of life AND entering their first RSV season at the time of dose administration OR (b) In their second year of life AND entering their second RSV season at the time of dose administration 2. The subject must meet at least 1 of the following conditions at the time of informed consent. (a) Diagnosed with combined immunodeficiency (severe combined immunodeficiency, X-linked hyper-immunoglobulin M [IgM] syndrome, etc); antibody deficiency (X linked agammaglobulinemia, common variable immunodeficiency, non-X-linked hyper-IgM syndromes, etc); or other immunodeficiency (Wiskott-Aldrich syndrome, DiGeorge syndrome, etc), or (b) Diagnosed with human immunodeficiency virus infection, or (c) History of organ or bone marrow transplantation, or (d) Subject is receiving immunosuppressive chemotherapy, or (e) Subject is receiving systemic high-dose corticosteroid therapy (prednisone equivalents ≥ 0.5 mg/kg every other day, other than inhaler or topical use), or (f) Subject is receiving other immunosuppressive therapy (eg, azathioprine, methotrexate, mizoribine, mycophenolate mofetil, cyclophosphamide, cyclosporine, tacrolimus, cytokine inhibitors, etc) All efforts will be made to recruit subjects for representation across all the immunocompromised conditions indicated in inclusion criteria (a) - (f). |
1. Neonato, lactante o niño pequeño de ≤24 meses que, según el criterio del investigador, se encuentre: (a) en su primer año de vida E inicie su primera temporada del VRS en el momento de la administración de la dosis; O BIEN (b) en su segundo año de vida E inicie su segunda temporada del VRS en el momento de la administración de la dosis. 2. El sujeto debe cumplir al menos 1 de las siguientes condiciones en el momento del consentimiento informado. (a) Diagnosticado de inmunodeficiencia combinada (inmunodeficiencia combinada grave, síndrome de hipergammaglobulinemia M [IgM] ligado al cromosoma X, etc.); deficiencia de anticuerpos (agammaglobulinemia ligada al cromosoma X, inmunodeficiencia variable común, síndromes de hiper-IgM no ligados al cromosoma X); u otra inmunodeficiencia (síndrome de Wiskott-Aldrich, síndrome de DiGeorge, etc.). (b) Diagnosticado de infección por el virus de la inmunodeficiencia humana. (c) Antecedentes de trasplante de órganos o de médula ósea. (d) El sujeto está recibiendo quimioterapia con inmunodepresores. (e) El sujeto está recibiendo tratamiento sistémico con dosis altas de corticoesteroides (≥0,5 mg/kg de equivalentes de prednisona cada dos días, excepto inhaladores o uso tópico). (f) El sujeto está recibiendo otro tratamiento inmunodepresor (p. ej., azatioprina, metotrexato, mizoribina, micofenolato mofetilo, ciclofosfamida, ciclosporina, tacrolimus, inhibidores de citocinas, etc.). Se hará todo lo posible por inscribir a sujetos que representen todos los tipos de inmunodepresión indicados en los criterios de inclusión (a) - (f). |
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E.4 | Principal exclusion criteria |
1. Subject who meets any of the indications other than (including those approved for palivizumab in Japan) the immunocompromised conditions below. (a) Subject born at ≤ 28 weeks gestation and is ≤ 12 months of age (b) Subject born at 29 to 35 weeks gestation and is ≤ 6 months of age (c) Age ≤ 24 months with a history of bronchopulmonary dysplasia requiring medical management within the past 6 months (d) Age ≤ 24 months with current hemodynamically significant CHD (e) Age ≤ 24 months with Down syndrome 2. Requirement for oxygen supplementation, mechanical ventilation, extracorporeal membrane oxygenation, continuous positive airway pressure, or other mechanical respiratory or cardiac support at screening. 3. A current, active infection, including RSV infection, at the time of screening or at the time of investigational product administration. 4. Any fever (≥ 100.4°F [≥ 38.0°C], regardless of route) or acute illness within 7 days prior to investigational product administration. 5. Any serious concurrent medical condition (renal failure, hepatic dysfunction, suspected active or chronic hepatitis infection, seizure disorder, unstable neurologic disorder, etc), except those resulting in an immune deficiency condition. 6. Clinically significant congenital anomaly of the respiratory tract. 7. Receipt of palivizumab. 8. Any known allergy or history of allergic reaction to any component of nirsevimab. 9. Any known allergy or history of allergic reaction to immunoglobulin products, blood products, or other foreign proteins. 10. Concurrent enrollment in another interventional study, or prior receipt of any investigational agent. 11. Anticipated survival of less than 1 year at the time of informed consent. |
1. El sujeto cumple cualquiera de las indicaciones (incluidas las aprobadas para palivizumab en Japón) distintas de las inmunodeficiencias indicadas a continuación. (a) Sujeto nacido con ≤28 semanas de gestación y con ≤12 meses. (b) Sujeto nacido a las 29-35 semanas de gestación y con ≤6 meses; (c) Edad ≤24 meses con antecedentes de displasia broncopulmonar que requiera tratamiento médico en los últimos 6 meses. (d) Edad ≤24 meses con CPC hemodinámicamente significativa en curso. (e) Edad ≤24 meses con síndrome de Down. 2. Necesidad de suplementos de oxígeno, ventilación mecánica, oxigenación por membrana extracorpórea, presión positiva continua en las vías respiratorias u otro tipo de asistencia respiratoria mecánica o cardíaca en la selección. 3. Infección actual activa, incluida infección por el VSR, en el momento de la selección o en el momento de la administración del producto en investigación. 4. Fiebre (≥38,0 °C [≥100,4 °F], independientemente de la vía) o enfermedad aguda en los 7 días previos a la administración del producto en investigación. 5. Cualquier afección médica concurrente grave (insuficiencia renal, disfunción hepática, sospecha de infección activa o crónica de hepatitis, trastorno convulsivo, trastorno neurológico inestable, etc.), excepto aquellas que causen una inmunodeficiencia. 6. Anomalía congénita de las vías respiratorias clínicamente significativa. 7. Haber recibido palivizumab. 8. Cualquier alergia conocida o antecedentes de reacción alérgica a cualquier componente del nirsevimab. 9. Cualquier alergia conocida o antecedentes de reacción alérgica a los productos de inmunoglobulina, hemoderivados u otras proteínas extrañas. 10. Inscripción simultánea en otro estudio intervencionista o haber recibido previamente cualquier fármaco en investigación. 11. Supervivencia prevista de menos de 1 año en el momento del consentimiento informado. |
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E.5 End points |
E.5.1 | Primary end point(s) |
All TEAEs, TESAEs, AESIs, and NOCDs |
Todos AAST, AAGST, AAEI y ECNA |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Through 360 days after dosing |
Hasta 360 días después de la dosificación |
|
E.5.2 | Secondary end point(s) |
PK: Summary of nirsevimab serum concentrations ADA: Incidence of ADA to nirsevimab in serum Efficacy: Incidence of medically attended LRTI (inpatient and outpatient) and hospitalizations due to RT-PCR-confirmed RSV through 150 days after administration of nirsevimab |
PK:Resumen de las concentraciones séricas de nirsevimab. AAF: Incidencia de AAF frente a nirsevimab en suero. Eficacia: Incidencia de IVRI atendidas médicamente (intrahospitalaria y ambulatoria) y hospitalizaciones debidas al VRS confirmadas mediante RT-PCR durante los 150 días posteriores a la administración de nirsevimab. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
PK, ADA through 360 days after dosing. Efficacy through 150 days after dosing. |
PK, AAF hasta 360 días después de la dosificación. Eficacia hasta 150 días después de la dosificación. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 11 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Japan |
South Africa |
Ukraine |
United States |
Belgium |
Poland |
Spain |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
The end of the study (“study completion”) is defined as the date of the last protocol-specified visit/assessment for the last subject in the study. |
El final del estudio (“finalización del estudio”) se define como la fecha de la última visita / evaluación especificada por el protocolo para el último sujeto del estudio. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |