A study to assess the pharmacokinetics and safety of tocilizumab in patients with Active Systemic Juvenile Idiopathic Arthritis.
- Conditions
- Systemic Juvenile Idiopathic Arthritis (sJIA)MedDRA version: 18.0Level: PTClassification code 10059176Term: Juvenile idiopathic arthritisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2015-000435-33-FR
- Lead Sponsor
- F. Hoffmann-La Roche Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 10
- Age less than 24 months at baseline
- Fulfills International League of Associations for Rheumatology classification criteria for sJIA
- Duration of sJIA lasting at least 1 month since onset of sJIA symptoms
- Presence of active disease as determined by the presence of >=2 active joints at screening and baseline, with at least 14 consecutive days of temperature recordings to identify presence or absence of fever (>=38°C) during screening and baseline; or >=2 active joints at screening and baseline, with a fever >=38°C for at least 5 consecutive days during screening and baseline; under these circumstances a patient does not need to complete a full 14 days of temperature diary entries to meet this inclusion criteria
- Not currently receiving corticosteroids (CS) OR if taking oral CS, receiving prednisone or equivalent at a stable dose of <=1 milligrams per kilograms per day (mg/kg/day); and the dose remained stable for at least 2 weeks prior to baseline
- Not currently receiving methotrexate (MTX) OR if taking MTX, the dose has remained stable or has been discontinued for at least 4 weeks prior to baseline
- Not currently receiving non-steroidal anti-inflammatory drug (NSAID) OR if taking NSAID, the dose has remained stable or has been discontinued for at least 2 weeks prior to baseline
- Never treated with biologics except anakinra which has been discontinued >=1 week prior to the baseline visit
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Any other auto-immune, rheumatic disease or overlap syndrome other than sJIA
- Any significant concurrent medical or surgical condition which would jeopardize the patient’s safety or ability to complete the trial or planned surgery during the study (myringotomy surgery does not exclude patient)
- Evidence of serious uncontrolled concomitant diseases including but not limited to the nervous system, renal, hepatic, or endocrine systems
- Asthma for which the patient has required the use of oral or parenteral corticosteroids for >=2 weeks within 6 months prior to the baseline visit
- Any active acute, subacute, chronic or recurrent bacterial, viral, or systemic fungal infection including but not limited to: acute or chronic renal / bladder infections; acute or chronic pulmonary infections
- History of atypical tuberculosis (TB) and active TB requiring treatment at any point prior to screening visit and positive TB test result at screen, unless treated with anti-tuberculosis therapy for at least 4 weeks prior to receiving study medication and chest radiograph is negative for active tuberculosis
- Any major episode of infection requiring hospitalization or treatment during screening or treatment with intravenous 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
- 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 within 1 month prior to the screening visit
- Administration of intravenous immunoglobulin within 4 weeks prior to the baseline visit
- Previous treatment with any cell depleting therapies, including investigational agents (e.g. anti-CD19 and anti-CD20)
- 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the pharmacokinetics of Tocilizumab (TCZ) over 12 weeks in patients less than 2 years of age with sJIA. ;Secondary Objective: To evaluate safety of TCZ over 12 weeks in combination with stable ongoing therapy. <br>To evaluate PD and appropriate efficacy of TCZ over 12 weeks in combination with stable ongoing therapy.;Primary end point(s): Pharmacokinetic: Serum TCZ concentration assessed by area under the serum concentration-time profile (AUC[2weeks]), maximum concentration observed (Cmax), and minimum concentration under steady-state conditions within a dosing interval (Cmin);Timepoint(s) of evaluation of this end point: Week 12
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Safety: Adverse events<br>2. Safety: Clinical laboratory results<br>3. Safety: Physical examination including vital signs<br>;Timepoint(s) of evaluation of this end point: 1. Up to 18 weeks<br>2-3. Up to 21 weeks<br>