E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polyarticular-course juvenile idiopathic arthritis (pcJIA); Systemic juvenile idiopathic arthritis (sJIA) |
Artritis idiopática juvenil poliarticular y artritis idiopática juvenil sistémica |
|
E.1.1.1 | Medical condition in easily understood language |
pcJIA: arthritis in children causing joint symptoms that can lead to disability. sJIA: arthritis in children often causing fever with rash, organ inflammation (including of lung, heart and liver) |
AIJP puede causar daño en las articulaciones llegando a producir incapacidad AIJS causa frecuentemente fiebre con picor, e inflamación en los organos (incluyendo pulmon, corazón e higado) |
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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 | 17.0 |
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 long-term safety of SC administration of TCZ in patients with pcJIA and sJIA
To describe the long-term efficacy of SC TCZ in patients with pcJIA and sJIA |
Evaluar la seguridad a largo plazo de la administración SC de TCZ en pacientes con AIJcp y AIJs
Describir la eficacia a largo plazo de tocilizumab (TCZ) subcutáneo (SC) en pacientes con artritis idiopática juvenil de curso poliarticular (AIJcp) y artritis idiopática juvenil sistémica (AIJs) |
|
E.2.2 | Secondary objectives of the trial |
Exploratory objectives:
To assess long-term pharmacodynamics of SC TCZ in patients with pcJIA and sJIA
To assess long-term pharmacokinetics of SC TCZ in patients with pcJIA and sJIA |
Evaluar la farmacodinamia a largo plazo de TCZ SC en pacientes con AIJcp y AIJs
Evaluar la farmacocinética a largo plazo de TCZ SC en pacientes con AIJcp y AIJs |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Completion of either of the JIGSAW studies, study WA28117 (for patients with pcJIA) or study WA28118 (for patients with sJIA)
- Adequate disease control with the use of SC RoActemra/Actemra, as assessed by the investigator
- Written informed consent for study participation obtained from the patient (for patients >/= 18 years old) or the patients parents or guardian, with assent as appropriate by the patient, depending on the level of the patients understanding
- For patients of reproductive potential: use of effective contraception as defined by the study protocol |
Haber completado el estudio WA28117 (pacientes con AIJcp) o el estudio WA28118 (pacientes con AIJs)
Mantener un control adecuado de la enfermedad con el uso de TCZ SC/IV, de acuerdo con el criterio clínico del investigador
Obtener el consentimiento informado por escrito para participar en el estudio, otorgado por el paciente (si tiene ?18 años) o por sus padres o tutor, con el asentimiento del paciente, si procede, dependiendo del nivel de comprensión del paciente
Los pacientes en edad fértil deben comprometerse a seguir los métodos anticonceptivos indicados en el protocolo |
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E.4 | Principal exclusion criteria |
- Therapy with biologic agents (except RoActemra/Actemra) in the period between completion of the JIGSAW study and screening for the current study
- Concurrent treatment with DMARDs (including MTX), NSAIDs, and oral corticosteroids is permitted at the discretion of the investigator
- Use of live or attenuated vaccines and immunosuppressants, such as cyclosporine and cyclophosphamide, is prohibited
- Pregnancy or breast-feeding
- Any significant concurrent medical or surgical conditions or findings that would jeopardize the patients safety or ability to complete the study, including but not limited to disease of the nervous, renal, hepatic, cardiac, pulmonary, gastric, or endocrine system or any infection
- History of alcohol, drug, or chemical abuse within 6 months prior to screening
- History of atypical tuberculosis (TB) or active TB requiring treatment within 2 years prior to screening
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- Tratamiento con agentes biológicos (excepto TCZ) en el período comprendido entre la
terminación del estudio JIGSAW correspondiente y la selección en el estudio actual
- Está permitido el tratamiento concomitante con fármacos antirreumáticos modificadores
de la enfermedad (incluyendo metotrexato), antiinflamatorios no esteroideos y corticosteroides orales, de acuerdo con el criterio del investigador.
- Está prohibida la administración de vacunas vivas o atenuadas e inmunosupresores,
tales como ciclosporina y ciclofosfamida.
- Mujeres que estén en período de lactancia
- Cualquier condición médica o quirúrgica concurrente significativa que pueda comprometer la seguridad del paciente o su capacidad para completar el estudio, incluyendo, aunque no exclusivamente, enfermedades de los sistemas nervioso, renal, hepático, cardíaco,
pulmonar o endocrino
- Antecedentes de abuso de alcohol, drogas o sustancias químicas en los 6 meses previos a la visita de selección
- Antecedentes de tuberculosis (TB) atípica o activa que ha requerido tratamiento en los 2
años previos a la visita de selección
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of adverse events
Incidence of serious adverse events
Incidence of adverse events of special interest
Incidence of clinical laboratory abnormalities
Change in Juvenile Arthritis Disease Activity Score (JADAS-71)
Change in Childhood Health Assessment Questionnaire (CHAQ) score
Inactive disease/clinical remission |
Incidencia y severidad de los acontecimientos adversos (incluyendo reacciones locales en la zona de inyección) y acontecimientos adversos graves
Incidencia y severidad de los acontecimientos adversos de especial interés
Incidencia y severidad de las anomalías de laboratorio clínico
Puntuación de la Juvenile Arthritis Disease Activity Score (JADAS-71)
Puntuación del cuestionario Childhood Health Assessment Questionnaire (CHAQ)
Enfermedad inactiva y remisión clínica |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 3 years |
Hasta 3 años |
|
E.5.2 | Secondary end point(s) |
The exploratory PK outcome measures will be trough TCZ plasma concentrations measured at specified time-points, and those for PD include Soluble IL-6R (sIL-6R) levels, CRP, ESR , and incidence of anti-TCZ antibodies. |
Concentraciones de IL-6R soluble, proteína C reactiva y velocidad de sedimentación globular
Incidencia de anticuerpos anti-TCZ |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 3 years |
Hasta 3 años |
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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 | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 |
long-term extension study |
|
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | 2 |
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 | 4 |
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 |
Argentina |
Australia |
Brazil |
Canada |
France |
Germany |
Italy |
Mexico |
Peru |
Russian Federation |
Spain |
United Kingdom |
United States |
|
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
|
The end of the study will occur when the last patient completes the last scheduled visit of the study or if the Sponsor decides for whatever reason to discontinue the study. Patients will be enrolled in the study until TCZ is commercially available in the applicable region or country. Participation may continue until commercial availability or for a maximum of 3 years. |
El estudio terminará cuando el último paciente complete la última visita planificada del estudio o en caso de que el promotor decida cancelar el estudio por cualquier motivo. Los pacientes serán incluidos en el estudio hasta que TCZ esté disponible comercialmente en la región o el país correspondiente. Cada paciente podrá continuar participando en el estudio hasta que TCZ esté disponible comercialmente o durante un máximo de 3 años, según sea lo que ocurra antes. |
|
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 |