E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polyarticular Course Juvenile Idiopathic Arthritis |
Artritis idiopática juvenil de evolución poliarticular |
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E.1.1.1 | Medical condition in easily understood language |
Polyarticular Course Juvenile Idiopathic Arthritis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10059176 |
E.1.2 | Term | Juvenile idiopathic arthritis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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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 pharmacokinetics, safety, and tolerability of multiple doses of upadacitinib in pediatric subjects with pcJIA. To evaluate the long-term safety and tolerability of multiple doses of upadacitinib in pediatric subjects with pcJIA. |
Evaluar la farmacocinética, la seguridad y la tolerabilidad de dosis múltiples de upadacitinib en pacientes pediátricos con AIJp. Evaluar la seguridad y la tolerabilidad a largo plazo de dosis múltiples de upadacitinib en pacientes pediátricos con AIJp. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the palatability of upadacitinib oral solution in pediatric subjects. |
Evaluar la palatabilidad de la solución oral de upadacitinib en pacientes pediátricos. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Male or female subjects, ages 2 to less than 18 years, and total body weight of 10 kg or higher at the time of Screening. • Diagnosed with pcJIA (rheumatoid factor-positive or rheumatoid factor-negative polyarticular JIA, extended oligoarticular JIA, or systemic JIA with active arthritis and without active systemic features) with a history of arthritis affecting at least 5 joints during the first 6 months of disease (for extended oligoarticular JIA: ≤ 4 joints during the first 6 months of disease and > 4 joints thereafter). • Have 5 or more active joints at the time of Screening, defined as the presence of swollen joints (not due to deformity) or, in the absence of swelling, joints with limitation of movement (LOM) plus pain on motion and/or tenderness with palpation, with LOM present in at least three of the active joints. • If receiving methotrexate (MTX), have been taking MTX for at least 12 weeks immediately before and including Study Day 1 on a stable dose of 10 to 20 mg/m2 for at least 8 weeks before and including Study Day 1; in addition, subjects should take either folic acid or folinic acid according to local standard of care. • If on oral glucocorticoids, must have been taking oral glucocorticoids at a stable dose (no greater than 10 mg/day or 0.2 mg/kg/day, whatever is lower) for at least 1 week before and including Study Day 1. |
•Varones o mujeres de 2 a menos de 18 años de edad y con un peso corporal total de 10 kg o superior en el momento de la selección. •Diagnóstico de AIJp (AIJ poliarticular con factor reumatoide positivo o negativo, AIJ oligoarticular extendida o AIJ sistémica con artritis activa y sin manifestaciones sistémicas activas) con antecedentes de artritis que afecte al menos a 5 articulaciones durante los 6 primeros meses de la enfermedad (para la AIJ oligoarticular extendida: ≤ 4 articulaciones durante los 6 primeros meses de la enfermedad y luego > 4 articulaciones). •Tener 5 o más articulaciones activas en el momento de la selección, definidas como la presencia de articulaciones inflamadas (no debidas a deformidad) o, en ausencia de inflamación, articulaciones con limitación del movimiento (LDM) más dolor con el movimiento o dolor con la palpación, con presencia de LDM en al menos tres de las articulaciones activas. •En caso de estar recibiendo metotrexato (MTX), el paciente deberá haberlo recibido durante al menos 12 semanas inmediatamente antes del día 1 del estudio, inclusive, en una dosis estable de 10 a 20 mg/m2 durante al menos 8 semanas antes del día 1 del estudio, inclusive; además, los pacientes deberán tomar ácido fólico o ácido folínico conforme a las normas asistenciales locales. •Si recibe glucocorticoides orales, deberá haberlos recibido en una dosis estable (no superior a 10 mg/día o 0,2 mg/kg al día, lo que corresponda a un valor más bajo) durante al menos una semana antes del día 1 del estudio, inclusive. |
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E.4 | Principal exclusion criteria |
• Subject must not have a diagnosis of enthesitis-related arthritis (ERA) or juvenile psoriatic arthritis (JPSA). • No prior exposure to JAK inhibitor. |
•El paciente no debe estar diagnosticado de artritis relacionada con entesitis (ARE) o artritis psoriásica juvenil (APsJ). •No haber estado previamente expuesto a un inhibidor de JAK. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The values for the pharmacokinetic parameters of upadacitinib including Cmax, time to maximum observed plasma concentration (Tmax), area under the plasma concentration versus time curve during a dosing interval (AUCtau) on Day 7, and apparent oral clearance at steady state (CL/F) will be determined using non-compartmental methods. |
Los valores de los parámetros farmacocinéticos de upadacitinib incluyendo Cmax, tiempo hasta la concentración plasmática máxima observada (Tmax), área bajo la curva de concentración plasmática versus tiempo durante un intervalo de dosificación (AUCtau) en el día 7 y aclaramiento oral aparente en estado estable (CL / F) se determinarán utilizando métodos no compartimentales. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
To evaluate the palatability of upadacitinib oral solution in pediatric subjects. |
Evaluar la palatabilidad de la solución oral de upadacitinib en pacientes pediátricos. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1 and Day 7 |
Dia 1 y Dia 7 |
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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 | No |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
First in pediatric subjects with pcJIA |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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.8.3 |
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 |
Germany |
Hungary |
Italy |
Spain |
United States |
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E.8.7 | Trial 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
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |