Resumen

This is a multi-center, open-label single-arm study to investigate the pharmacokinetics and safety of tocilizumab (RoActemra/Actemra) in participants less than 2 years old with active sJIA. Participants will receive tocilizumab infusions every 2 weeks. The anticipated time on study treatment is 12 weeks (Main evaluation period). Participants will have the option to continue tocilizumab treatment until participant reaches 2 years of age or up to one year from baseline, whichever is longer. An optional extension period will follow the main evaluation period.

Elegibilidad

Edad máxima: 24 MonthsSexo: ALL

Criterios de inclusión

Fulfils international league of associations for rheumatology (ILAR) classification criteria for sJIA
Duration of sJIA symptoms lasting for at least 1 months subsequent to diagnosis of sJIA
Presence of active disease as determined by the presence of:
Greater than or equal to (\>=) 2 active joints at screening and baseline, with at least 14 consecutive days of temperature recordings, which may include the presence or absence of fever (\>=38 degree Celsius) during the time between screening and baseline; or
Not currently receiving corticosteroids (CS) or if taking oral CS like prednisone or equivalent, the dose should be less than or equal to (\<=) 1 milligram per kilogram per day (mg/kg/day) and the dose has remained stable for at least 2 weeks prior to baseline
Not currently receiving methotrexate (MTX) or if taking MTX (together with either folic acid or folinic acid according to local standard-of-care), the dose has remained stable or has been discontinued for at least 4 weeks prior to baseline
Not currently receiving non-steroidal anti-inflammatory drugs (NSAIDs) or if taking NSAID, the dose has remained stable or has been discontinued for at least 2 weeks prior to baseline
If the participants has received previous treatment with any of the following biologic agents, these must have been discontinued according to the following timelines prior to the baseline visit and are not permitted during the study:
Etanercept must have been discontinued within \>= 2 weeks prior to baseline
Anakinra must have been discontinued within \>= 4 days prior to baseline
Abatacept must have been discontinued within \>= 12 weeks prior to baseline
Infliximab or adalimumab must have been discontinued within \>= 8 weeks prior to baseline
Canakinumab must have been discontinued within \>= 20 weeks prior to baseline
Rilonacept must have been discontinued within \>= 6 weeks prior to baseline
Golimumab must have been discontinued within \>= 10 weeks prior to baseline
Certrolizumab pegol must have been discontinued within \>= 10 weeks prior to baseline
History of inadequate clinical response (in the opinion of the treating physician) to NSAIDs and CS

Criterios de exclusión

Any autoimmune, rheumatic disease or overlap syndrome other than sJIA
Not fully recovered from recent surgery or less than 6 weeks since surgery, at the time of screening visit; or planned surgery during the study (except for myringotomy surgery, which is permitted)
Any significant concurrent medical or surgical condition which would jeopardize the participant's safety or ability to complete the trial
History of significant allergic or infusion reactions to prior biologic therapy or to any of the excipients listed in tocilizumab product labelling documents
Inborn conditions characterized by a compromised immune system
Known human immunodeficiency virus (HIV) infection or other acquired forms of immune compromise
Evidence of serious uncontrolled concomitant diseases including but not limited to the nervous system, renal, hepatic or endocrine systems
Asthma for which the participant has required the use of oral or parenteral corticosteroids for \>=2 weeks within 6 months prior to baseline visit
Any active acute, subacute, chronic or recurrent bacterial, viral or systemic fungal infection
History of atypical tuberculosis (TB)
Active TB requiring treatment at any point prior to screening visit
Positive TB test result at screen, unless treated with anti-TB therapy for at least 4 weeks prior to receiving study medication and chest radiograph is negative for active TB within 6 months of screening visit consistent with local practice
Any major episode of infection requiring hospitalization or treatment during screening or treatment with IV antibiotics completing within 4 weeks of the screening visit or oral antibiotics completing within 2 weeks of the screening visit
History of reactivation or new onset of a systemic infection such as herpes zoster or Epstein Barr virus within 2 months of the screening visit
History of hepatitis B or hepatitis C infection
Chronic hepatitis - viral or autoimmune
Significant cardiac or pulmonary disease
History or concurrent serious gastrointestinal disorders such as ulcer or inflammatory bowel disease, ulcerative colitis or other symptomatic lower gastrointestinal conditions, including ulcer and perforation
History of or current cancer or lymphoma
History of macrophage activation syndrome (MAS) within 3 months prior to the screening visit
Uncontrolled diabetes mellitus with elevated hemoglobin A1c (HbA1c) as defined by age-specific standards
Excluded Previous or Concomitant Therapy:
Participation in another interventional clinical trial within the past 30 days or 5 serum half-lives of the investigative medication, whichever is longer
Previous treatment with tocilizumab
Administration of IV immunoglobulin within 4 weeks prior to the baseline visit
Previous treatment with any cell depleting therapies, including investigational agents
Prior stem cell transplant at any time
Live or attenuated vaccines within 4 weeks prior to the baseline visit, or intending to receive while on study medication or 8 weeks following the last dose of study medication
Serum creatinine \>1.5 ULN (upper limit of normal for age and sex)
AST or ALT \> 1.5 ULN (upper limit of normal for age and sex)
Total bilirubin \> 1.3 mg/dL (\> 23 umol/L)
Platelet count \< 200 x103/μL (\< 200,000/mm3)
Hemoglobin \< 7.0 g/dL (\< 4.3 mmol/L)
WBC count \< 6,200/mm3 (\< 6.2 x 109/L)
Neutrophil count \< 2,500/ mm3 (\< 2.5x 109/L)

A Phase I Pharmacokinetic and Safety Study of Tocilizumab (TCZ) in Patients Less Than 2 Years Old With Active Systemic Juvenile Idiopathic Arthritis (sJIA)

Resumen

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Tocilizumab

Ubicaciones

Hospital Gral de Niños Pedro Elizalde

Patrocinadores