E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polyarticular Course Juvenile Idiopathic Arthritis |
Artrite Idiopatica Giovanile ad Esordio Poliarticolare |
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E.1.1.1 | Medical condition in easily understood language |
Polyarticular Course Juvenile Idiopathic Arthritis |
Artrite Idiopatica Giovanile ad Esordio Poliarticolare |
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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 | 23.1 |
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. |
Valutare il profilo di farmacocinetica, sicurezza e tollerabilità di dosi multiple di upadacitinib in soggetti pediatrici affetti da pcJIA. Valutare la sicurezza e tollerabilità a lungo termine di dosi multiple di upadacitinib in soggetti pediatrici affetti da pcJIA. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the palatability of upadacitinib oral solution in pediatric subjects. |
Valutare la gradevolezza al palato di upadacitinib soluzione orale per i soggetti pediatrici. |
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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. |
• Soggetti di ambo i sessi, di età compresa fra 2 anni e < 18 anni, e peso corporeo totale pari o superiore a 10 kg al momento dello Screening • Diagnosi di pcJIA (AIG poliarticolare FR-positiva oppure FR-negativa, AIG oligoarticolare estesa oppure AIG sistemica con artrite attiva e assenza di malattia sistemica attiva) con storia di artrite a carico di almeno 5 articolazioni nel corso dei primi 6 mesi di malattia (nel caso di AIG oligoarticolare estesa: = 4 articolazioni nel corso dei primi 6 mesi della malattia e > 4 articolazioni in seguito). • Presenza di 5 o più articolazioni con artrite attiva al momento dello Screening, ovvero presenza di articolazioni tumefatte (non causate da deformità) oppure, in assenza di tumefazione, articolazioni con limitazione del movimento (limitation of movement, LOM) in aggiunta a dolore al movimento e/o dolorabilità alla palpazione, con limitazione del movimento presente in almeno tre delle articolazioni con artrite attiva. • Se in trattamento con metotressato (MTX), tale trattamento dovrà essere in corso da almeno le 12 settimane immediatamente precedenti il Giorno 1 (compreso) dello Studio Clinico, a dose stabile compresa fra 10 e 20 mg/m2 da almeno 8 settimane prima del Giorno 1 (compreso) dello Studio Clinico; inoltre, i soggetti devono essere in trattamento con acido folico o acido folinico, secondo quanto previsto dallo standard di cura locale. • Se in trattamento con glucocorticoidi orali, il trattamento con glucocorticoidi orali deve essere a dose stabile (non superiore a 10 mg/die oppure 0,2 mg/kg/die, quale dei due dosaggi sia inferiore) da almeno 1 settimana prima del Giorno 1 (compreso) dello Studio Clinico. |
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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. |
• Soggetto non deve avere una diagnosi di artrite correlata a entesite (enthesitis-related arthritis, ERA) oppure di artrite psoriasica giovanile (juvenile psoriatic arthritis, JPSA). • Nessuna esposizione pregressa a un inibitore della proteina 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. |
I valori per i parametri di farmacocinetica per upadacitinib, compresi il Cmax, tempo alla massima concentrazione plasmatica osservata (Tmax), area sotto la concentrazione plasmatica rispetto alla curva tempo nell’intervallo fra somministrazioni (AUCtau) il Giorno 7, e la clearance orale apparente allo stato stazionario (CL/F) saranno determinati utilizzando metodi non compartimentali |
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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. |
Valutare la gradevolezza al palato di upadacitinib soluzione orale per i soggetti pediatrici. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1 and Day 7 |
Giorno 1 e Giorno 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 |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | Yes |
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) | Yes |
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 | 2 |
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 | Information not present in EudraCT |
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 |
United States |
Germany |
Hungary |
Italy |
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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 |