Juvenile Idiopathic Arthritis
Conditions
Brief summary
Primary Objective: To describe the pharmacokinetic (PK) profile of sarilumab in patients aged 1-17 years with Systemic Juvenile Idiopathic Arthritis (sJIA) in order to identify the dose and regimen for adequate treatment of this population. Secondary Objective: To describe the pharmacodynamics (PD) profile, the efficacy, and the long term safety of sarilumab in patients with sJIA.
Detailed description
The total study duration per patient will be 166 weeks that will consist of a 4- week screening, a 12-week core treatment phase, a 144-week extension phase, and a 6-week post-treatment follow-up.
Interventions
Pharmaceutical form: Solution Route of administration: Subcutaneous
Sponsors
Sanofi
Regeneron Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
: * Male and female patients aged ≥1 and ≤17 years (or country specified age requirement, ≥6 to ≤17 years for Russia) at the time of the screening visit. * Diagnosis of systemic JIA subtype according to the International Associations against Rheumatism (ILAR) 2001 Juvenile Idiopathic Arthritis (JIA) Classification Criteria OR According to 2024 EULAR/PReS recommendation at Screening. * Patient with an inadequate response to current treatment and considered as a candidate for a biologic disease modifying anti rheumatic drug (DMARD) as per investigator's judgment.
Exclusion criteria
* Body weight \<10 kg or \>60 kg for patients enrolled in the ascending dose cohorts, then body weight \<10 kg for patients subsequently enrolled at the selected dose. * Uncontrolled severe systemic symptoms and/or Macrophage Activation Syndrome (MAS) within 6 months prior to screening. * History of or ongoing interstitial lung disease, pulmonary hypertension, pulmonary alveolar proteinosis. * If nonsteroidal anti-inflammatory drugs (NSAIDs) (including cyclo oxygenase-2 inhibitors \[COX-2\]) taken, dose stable for less than 2 weeks prior to the baseline visit and/or dosing prescribed outside of approved label. * If non-biologic DMARD taken, dose stable for less than 6 weeks prior to the baseline visit or at a dose exceeding the recommended dose as per local labeling. * If oral glucocorticoid taken, dose exceeding equivalent prednisone dose 1 mg/kg/day (or 60 mg/day) within 3 days prior to baseline. * Use of parenteral or intra-articular glucocorticoid injection within 4 weeks prior to baseline. * Prior treatment with anti-interleukin 6 (IL-6) or IL-6 receptor (IL-6R) antagonist therapies, including but not limited to tocilizumab or sarilumab. * Treatment with any biologic treatment for sJIA within 5 half-lives prior to the first dose of sarilumab (the required off treatment periods and procedures may vary according to local requirements). * Treatment with a Janus kinase inhibitor within 4 weeks prior to the first dose of sarilumab; and treatment with growth hormone within 4 weeks prior to the first dose of sarilumab (the required off treatment periods and procedures may vary according to local requirements). * Treatment with any investigational biologic or non-biologic product within 8 weeks or 5 half-lives prior to baseline, whichever is longer. * Exclusion related to tuberculosis. *
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assessment of PK parameter: maximum serum concentration observed (Cmax) | Up to Week 12 |
| Assessment of PK parameter: Area under the serum concentration versus time curve calculated using the trapezoidal method during a dose interval (AUC0-t) | Up to Week 12 |
| Assessment of PK parameter: Concentration observed before treatment administration during repeated dosing (Ctrough) | Up to Week 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with adverse events | Core treatment phase: Up to Week 12. Extension phase: Up to Week 162 | — |
| Proportion of participants with local reactions after injection | Core treatment phase: Up to Week 12. Extension phase: Up to Week 156 | — |
| Proportion of participants with Investigator Global Assessment (IGA) of disease activity below a defined value on 1-100 VAS scale | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | — |
| Proportion of participants with Parent / patient Global Assessment (PGA) of well-being below a defined value on 1-100 VAS scale | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | — |
| Proportion of participants with clinically inactive disease (CID) | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | — |
| Changes in glucocorticoid use | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | — |
| Juvenile Idiopathic Arthritis ACR30/50/70/90/100 (in the absence of fever) response rate | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | Population according to the 2001 ILAR classification |
| Change from baseline in individual JIA ACR components | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | Population according to the 2001 ILAR classification |
| Change from baseline in Systemic Juvenile Arthritis Disease Activity Score-10 (sJADAS-10) | Core treatment phase: Up to Week 12. Extension phase: At weeks 24, 48, and every 24 weeks up to Week 156 | Population according to the 2001 ILAR classification |
| Assessment of participants with disease-related symptoms | At Week 4 | Population according to the 2024 EULAR / PReS |
| Changes in IL-6 associated biomarkers | Up to Week 12 | Population according to the 2001 ILAR classification and the 2024 EULAR / PReS |
Countries
Argentina, Canada, Finland, France, Germany, Greece, Ireland, Italy, Russia, Spain, United Kingdom
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
STUDY_DIRECTORClinical Sciences & Operations
Sanofi
Outcome results
None listed