| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Juvenile Idiopathic Arthritis |
| Artrite Idiopatica Giovanile |
|
| E.1.1.1 | Medical condition in easily understood language |
| Childhood Arthritis |
| Artrite Giovanile |
|
| E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
| 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 |
|
| E.1.3 | Condition being studied is a rare disease | Yes |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
| The primary objective of this protocol is to evaluate the long-term safety and tolerability of baricitinib in patients with JIA or sJIA |
| L'obiettivo primario di questo protocollo è quello di valutare la sicurezza a lungo termine e la tollerabilità di baricitinib in pazienti con artrite idiopatica giovanile (AIG) o artrite idiopatica giovanile sistemica (AIGs) |
|
| E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are
-to evaluate the long-term efficacy of baricitinib in children with JIA or sJIA
-to assess the long-term efficacy of baricitinib in children with JPsA
-to evaluate the long-term efficacy of baricitinib in children with ERA or JPsA
-to evaluate the potential effects of baricitinib on the cellular and humoral immune system |
Gli obiettivi secondari dello studio sono:
- Valutare l'efficacia a lungo termine di baricitinib in bambini con AIG o AIGs. - Valutare l'efficacia a lungo termine di baricitinib in bambini con artrite psoriasica giovanile sistemica (APGs)
- Valutare l'efficacia a lungo termine di baricitinib in bambini con artrite associata a entesite (ERA) o APGs.
-Valutare gli effetti potenziali di baricitinib sul sistema immunitario cellulare e umorale |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
- Have either completed participation in or terminated early from Study JAHV or Study JAHU and have not completed Post Treatment Follow up in those studies.
- Both the child or adolescent and a parent or legal guardian are able to understand and fully participate in the activities of the clinical study and sign their assent (if applicable) and consent, respectively, according to local guidelines.
- Male or nonpregnant, nonbreastfeeding female patients. Patients of childbearing potential who are abstinent or in a same-sex relationship must agree to either remain abstinent or stay in a same sex relationship without sexual relationships with the opposite sex. Otherwise, patients and their partners of childbearing potential must agree to use 2 effective methods of contraception. |
- Avere completato la partecipazione o interrotto anticipatamente lo studio JAHV o lo studio JAHU e non avere completato il follow-up post trattamento di questi studi.
- Sia il bambino o l'adolescente che un genitore o un tutore legale sono in grado di comprendere e partecipare interamente alle attività dello studio clinico e firmare il proprio assenso (se applicabile) e consenso, rispettivamente, secondo le linee guida locali.
- Pazienti di sesso maschile o pazienti di sesso femminile non gravide e non in allattamento. Pazienti potenzialmente fertili astinenti o con un rapporto con lo stesso sesso devono acconsentire a rimanere astinenti o a mantenere rapporti con lo stesso sesso senza rapporti sessuali con il sesso opposto. In caso contrario, i pazienti e le loro partner potenzialmente fertili devono acconsentire ad utilizzare 2 metodi contraccettivi efficaci. |
|
| E.4 | Principal exclusion criteria |
- Had investigational product permanently discontinued in the originating study
- Had temporary investigational product interruption at the final study visit of the originating study and, in the opinion of the investigator, this poses an unacceptable risk for the patient's participation in the current study
Medical Conditions
- Have a known hypersensitivity to baricitinib or any component of this investigational product
- Active anterior uveitis or receiving concurrent treatment for anterior uveitis
- Have significant uncontrolled cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, hematological, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that developed during the originator study that, in the opinion of the investigator, could constitute an unacceptable risk to the patient if investigational product continues
- Have any other condition that, in the opinion of the investigator, renders the patient unable to understand the nature, scope, and possible consequences of the study or precludes the patient from following and completing the protocol
- Intends to donate blood during the course of the study
- Intend to receive a live vaccine (except booster immunization with attenuated vaccine for measles, mumps, and rubella [MMR] or varicellazoster virus [VZV]) during the course of the study, or up to 28 days after the last dose of study drug. Booster vaccination for MMR or VZV may be considered if it is essential based on the local guideline and/or in the opinion of the investigator.
- Currently enrolled in any other clinical study (except the originating study at screening for the current study) involving an investigational product or any other type of medical research judged not to be scientifically or medically compatible with this study |
- Ha interrotto in modo permanente il prodotto sperimentale nello studio di origine
- Ha avuto un’interruzione temporanea del prodotto sperimentale alla visita finale dello studio di origine e, secondo l'opinione dello sperimentatore, questo comporta un rischio inaccettabile per la partecipazione del/della paziente allo studio corrente.
Condizioni mediche
- Presentare un’ipersensibilità nota a baricitinib o a uno dei componenti di questo prodotto sperimentale.
- Presentare uveite anteriore attiva o e avere un trattamento concomitante in corso per un’uveite precedente.
- Presentare disturbi cardiovascolari, respiratori, epatici, gastrointestinali, endocrini, ematologici, neurologici o neuropsichiatrici significativamente non controllati o qualsiasi altra malattia grave e/o instabile che si è sviluppata durante lo studio di origine e che, secondo l'opinione dello sperimentatore, potrebbe costituire un rischio inaccettabile per il paziente se il prodotto sperimentale continuasse ad essere somministrato.
- Presentare qualsiasi altra condizione che, secondo l'opinione dello sperimentatore, rende il paziente incapace di comprendere la natura, l'entità e le possibili conseguenze dello studio o preclude il paziente dal seguire e completare il protocollo
- Intende donare il sangue nel corso dello studio
- Intende ricevere un vaccino vivo (fatta eccezione per l'immunizzazione di richiamo con vaccini attenuati per morbillo, parotite e rosolia [MPR] o per il virus varicella-zoster [VZV] nel corso dello studio o fino a 28 giorni dopo l'ultima dose del farmaco dello studio. La vaccinazione di richiamo per MPR e VZV può essere presa in considerazione se è essenzialmente basata sulle linee guida locali e/o secondo l'opinione dello sperimentatore.
- È attualmente arruolato a un altro studio clinico (fatta eccezione per lo studio di origine allo screening per lo studio attuale) che comporta l'uso di un prodotto sperimentale o a qualsiasi tipo di ricerca medica valutata non compatibile con questo studio dal punto di vista scientifico o medico |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
- Treatment-emergent adverse events (TEAEs), adverse events of special
interest (AESIs), and serious adverse events (SAEs)
- Temporary investigational product interruptions and permanent
investigational product discontinuations
- Vital signs, growth and development, and laboratory evaluations
(including chemistry and hematology) |
- Eventi avversi emergenti dal trattamento (TEAE), eventi avversi di speciale interesse (AESI) ed eventi avversi gravi (SAE)
- Temporanee interruzioni del prodotto sperimentale e sospensioni permanenti del prodotto sperimentale
- Parametri vitali, crescita e sviluppo e valutazioni di laboratorio (incluse chimica ed ematologia) |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| through Week 264 |
| fino alla settimana 264 |
|
| E.5.2 | Secondary end point(s) |
Through Week 264:
- Proportion of patients who achieve PedACR30/50/70/90/100 response rates using baseline of the originator study
- Proportion of patients who demonstrate durability of
PedACR30/50/70/90/100 response rates from the time of randomization in the originator study.
- Proportion of patients who maintain PedACR30/50/70/90/100 response rates.
- Proportion of patients who have disease flare.
- Changes in each of the 6 individual core set component variables of the PedACR from baseline of the originator study
- Change from baseline of the originating study in health-related quality of life (QoL) as measured by Child Health Questionnaire-Parent Form 50 (CHQ-PF50).
- Change from baseline of originating study in caregiver burden as measured by the parent/family domains of the CHQ-PF50.
- Proportion of patients with inactive disease (as defined by Wallace et al 2011).
- Proportion of patients with minimal disease activity (as defined by Consolaro et al 2012).
- Proportion of patients in remission (as defined by Wallace et al 2012)
- Change from baseline of originating study in Juvenile Arthritis Disease Activity Score-27 (JADAS27).
- Change from baseline of originating study in arthritis-related pain, as measured by the CHAQ pain item.
In patients with JPsA:
- Change from baseline of originating study in Psoriasis Area and Severity Index (PASI)
In patients with ERA or JPsA:
- Change from baseline of originating study in SPARCC enthesis index
- Change from baseline of originating study in Juvenile Spondyloarthritis Disease Activity Index (JSpADA)
- Change from baseline of originating study in immunoglobulin levels and peripheral blood immunophenotyping (including T and B cells, T cell subsets, and NK cells)
- Change of IgG titers from pre-vaccination to 4 weeks and 12 weeks post vaccination in patients eligible for vaccination with tetanus, diphtheria, and pertussis (TDaP) and/or pneumococcal according to local guidelines. |
Through Week 264:
- Proportion of patients who achieve PedACR30/50/70/90/100 response rates using baseline of the originator study
- Proportion of patients who demonstrate durability of PedACR30/50/70/90/100 response rates from the time of randomization in the originator study.
- Proportion of patients who maintain PedACR30/50/70/90/100 response rates.
- Proportion of patients who have disease flare.
- Changes in each of the 6 individual core set component variables of the PedACR from baseline of the originator study
- Change from baseline of the originating study in health-related quality of life (QoL) as measured by Child Health Questionnaire-Parent Form 50 (CHQ-PF50).
- Change from baseline of originating study in caregiver burden as measured by the parent/family domains of the CHQ-PF50.
- Proportion of patients with inactive disease (as defined by Wallace et al 2011).
- Proportion of patients with minimal disease activity (as defined by Consolaro et al 2012).
- Proportion of patients in remission (as defined by Wallace et al 2012)
- Change from baseline of originating study in Juvenile Arthritis Disease Activity Score-27 (JADAS27).
- Change from baseline of originating study in arthritis-related pain, as measured by the CHAQ pain item.
In patients with JPsA:
- Change from baseline of originating study in Psoriasis Area and Severity Index (PASI)
In patients with ERA or JPsA:
- Change from baseline of originating study in SPARCC enthesis index
- Change from baseline of originating study in Juvenile Spondyloarthritis Disease Activity Index (JSpADA)
- Change from baseline of originating study in immunoglobulin levels and peripheral blood immunophenotyping (including T and B cells, T cell subsets, and NK cells)
- Change of IgG titers from pre-vaccination to 4 weeks and 12 weeks post vaccination in patients eligible for vaccination with tetanus, diphtheria, and pertussis (TDaP) and/or pneumococcal according to local guidelines. |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
| through Week 264 |
| fino alla settimana 264 |
|
| 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 | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | No |
| E.6.7 | Pharmacodynamic | Yes |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | No |
| E.6.10 | Pharmacogenetic | Yes |
| E.6.11 | Pharmacogenomic | Yes |
| 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) | 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 | 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 | 10 |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 42 |
| 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 |
| Australia |
| Brazil |
| China |
| India |
| Israel |
| Japan |
| Mexico |
| Russian Federation |
| Turkey |
| Austria |
| Belgium |
| Denmark |
| France |
| Germany |
| Italy |
| Poland |
| Spain |
| United Kingdom |
| Czechia |
| Argentina |
|
| 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 date of the last visit or last scheduled procedure for the last patient in the 28-day follow-up period. |
| La data dell'ultima visita dell' ultimo paziente durante il periodo di follow-up di 28 giorni |
|
| 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 | 2 |
| 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 | 2 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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