Juvenile Idiopathic ArthritisPhase 2SKYPPNCT02776735
An Open-label, Ascending, Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA)
A Phase 2 study testing Sarilumab in people with Juvenile Idiopathic Arthritis, run by Sanofi at 30 sites in 15 countries.
Trial summary
Primary Objective: To describe the pharmacokinetic (PK) profile of sarilumab in participants aged 2-17 years with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) in order to identify the dose and regimen for adequate treatment of this population Secondary Objective: To describe the pharmacodynamic (PD)… Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Juvenile Idiopathic Arthritis
- What is being tested?
- Sarilumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 102 participants
- Where
- 30 sitesin 15 countries
- Study dates
- 6 Sep 2016to 27 Dec 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female participants aged ≥2 and ≤17 years (or country specified age requirement) at the time of the screening visit.
- Diagnosis of rheumatoid factor-negative or rheumatoid factor positive polyarticular Juvenile Idiopathic Arthritis (JIA) subtype or oligoarticular extended JIA subtype according to the International League of Associations for Rheumatology (ILAR) 2001 Juvenile Idiopathic Arthritis Classification Criteria with at least 5 active joints as per American College of Rheumatology (ACR) definition for "active arthritis" at Screening
- Participant with an inadequate response to current treatment and considered as a candidate for a biologic disease modifying antirheumatic drug (DMARD) as per investigator's judgment
Who cannot join
- Body weight <10 kg or >60 kg for participants enrolled in the 3 ascending dose cohorts, then body weight <10 kg for participants subsequently enrolled at the selected dose-regimen.
- If nonsteroidal anti-inflammatory drugs (NSAIDs) [including cyclo oxygenase-2 inhibitors (COX-2)] taken, dose stable for <2 weeks prior to the baseline visit and/or dosing prescribed outside of approved label.
- If non-biologic DMARD taken, dose stable for <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 0.5 mg/kg/day (or 30 mg/day) within 2 weeks 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.
Show all 16Show fewer
- Treatment with any biologic treatment for pcJIA within 5 half-lives prior to the first dose of sarilumab.
- 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.
- Lipid lowering drug stable for less than 6 weeks prior to screening.
- Exclusion criteria related to past or current infection other than tuberculosis.
- Any live, attenuated vaccine within 4 weeks prior to the baseline visit, such as varicella-zoster, oral polio, rubella vaccines. Killed or inactive vaccine may be permitted based on the Investigator's judgment.
- Exclusion related to history of a systemic hypersensitivity reaction to any biologic drug and known hypersensitivity to any constituent of the product.
- Laboratory abnormalities at the screening visit (identified by the central laboratory).
- Pregnant or breast-feeding female adolescent participants.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
30 sites in 15 countries.
30 sites in 15 countries
Poland5 sites
Investigational Site Number : 6160074
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-667
Investigational Site Number : 6160073
Krakow, Lesser Poland Voivodeship, 31-503
Investigational Site Number : 6160071
Lodz, Lódzkie, 91-738
Investigational Site Number : 6160070
Lublin, Lubusz Voivodeship, 20-093
Investigational Site Number : 6160072
Sosnowiec, Silesian Voivodeship, 41-218
Germany4 sites
Investigational Site Number : 2760064
Berlin, 13125
Investigational Site Number : 2760061
Bremen, 28205
Investigational Site Number : 2760062
Hamburg, 22081
Investigational Site Number : 2760060
Sankt Augustin, 53757
Spain4 sites
Investigational Site Number : 7240050
Esplugues de Llobregat, Catalunya [Cataluña], 08950
Investigational Site Number : 7240052
Madrid, Madrid, Comunidad de, 28046
Investigational Site Number : 7240053
Madrid, 28009
Investigational Site Number : 7240051
Valencia, 46026
Russia3 sites
Investigational Site Number : 6430001
Moscow, 115522
Investigational Site Number : 6430062
Moscow, 117198
Investigational Site Number : 6430063
Moscow, 119991
Argentina2 sites
Investigational Site Number : 0320060
CABA, Buenos Aires, C1270AAN
Investigational Site Number : 0320004
San Miguel de Tucumán, T4000AXL
Mexico2 sites
Investigational Site Number : 4840061
Guadalajara, Jalisco, 44620
Investigational Site Number : 4840060
Monterrey, Nuevo León, 64460
United Kingdom2 sites
Investigational Site Number : 8260033
Liverpool, L12 2AP
Investigational Site Number : 8260031
London, London, City of, WC1N 3JH
United States1 site
Children's Hospital Los Angeles Site Number : 8400416
Los Angeles, California, 90027
About this study
Official title: An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered With Subcutaneous (SC) Injection, in Children and Adolescents, Aged 2 to 17 Years, With Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) Followed by an Extension Phase
Primary Objective:
To describe the pharmacokinetic (PK) profile of sarilumab in participants aged 2-17 years with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) in order to identify the dose and regimen for adequate treatment of this population
Secondary Objective:
To describe the pharmacodynamic (PD) profile, the efficacy and the long-term safety of sarilumab in participants with pcJIA.
Read the detailed description
For 73 participants enrolled in the dose-finding and second portions, the total study duration per participant was up to 166 weeks that consists of a 4- week screening, a 12-week core treatment phase, a 144-week extension phase, and a 6-week post-treatment follow-up. For 29 participants enrolled in the third portion, the total study duration per participant was up to 106 weeks that consists of a 4- week screening, a 12-week core treatment phase, a 84-week extension phase, and a 6-week post-treatment follow-up.
- Conditions
- Juvenile Idiopathic Arthritis
- Being tested
- Sarilumab
- Medical subject headings
- Arthritis, Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SarilumabDrug
Also called SAR153191 (REGN88)
Pharmaceutical form:Solution Route of administration: Subcutaneous
| Study group | Role | What they receive |
|---|---|---|
| Sarilumab | Experimental | Participants received one of three ascending dose regimens of sarilumab by subcutaneous (SC) injection based on body weight. All the participants received the selected dose regimen once this was identified. Sarilumab was given during 12-week core treatment phase followed by an extension treatment phase (144 weeks for 73 participants enrolled in dose-finding and second portions and 84 weeks for approximately 29 participants enrolled in third portion) Receives: Drug: Sarilumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Serum Concentration (Cmax) of Sarilumab at Week 12
The Cmax was defined as maximum serum concentration. The values reported are mean and standard deviation.
Measured: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
Area Under the Serum Concentration Versus Time Curve Using the Trapezoidal Method During a Dose Interval (AUC0-t) of Sarilumab at Week 12
The AUC0-t was defined as area under the concentration in serum versus time curve calculated using the trapezoidal method during a dose interval (tau). The values reported are mean and standard deviation.
Measured: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
Concentration Before Treatment Administration During Repeated Dosing (Ctrough) of Sarilumab at Week 12
The Ctrough was defined as concentration observed before treatment administration during repeated dosing from baseline to Week 12. The values reported are mean and standard deviation.
Measured: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
Secondary outcomes (30)
Cohorts 1 and 3: Change From Baseline in High-Sensitivity C-reactive Protein (Hs-CRP) at Week 12
Serum concentrations of hs-CRP was determined to assess the Pharmacodynamic (PD) effects of sarilumab. The values reported are mean and standard deviation.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in High-Sensitivity C-reactive Protein at Weeks 12, 24, 48, 96, and 156
Serum concentrations of hs-CRP was determined to assess the PD effects of sarilumab. The values reported are mean and standard deviation.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Change From Baseline in Interleukin-6 (IL-6) at Week 12
Serum concentrations of IL-6 was determined to assess the PD effects of sarilumab. The values reported are mean and standard deviation.
Measured: Baseline (Day 1) and Week 12
Change From Baseline in Total Soluble Interleukin-6 Receptor (sIL-6R) at Week 12
Serum concentrations of sIL-6R was determined to assess the PD effects of sarilumab. The values reported are mean and standard deviation.
Measured: Baseline (Day 1) and Week 12
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology (JIA ACR) 30 Response at Week 12
JIA ACR30 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 30% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 30 Response at Weeks 12, 24, 48, 96, and 156
JIA ACR30 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 30% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 50 Response at Week 12
JIA ACR50 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 50% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 50 Response at Weeks 12, 24, 48, 96, and 156
JIA ACR50 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 50% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 70 Response at Week 12
JIA ACR70 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 70% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 70 Response at Weeks 12, 24, 48, 96, and 156
JIA ACR70 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 70% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 90 Response at Week 12
JIA ACR90 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 90% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 90 Response at Weeks 12, 24, 48, 96, and 156
JIA ACR90 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 90% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 100 Response at Week 12
JIA ACR100 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 100% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 100 Response at Weeks 12, 24, 48, 96, and 156
JIA ACR100 response was defined as a participant with at least 3 out of the 6 JIA core set variables with >= 100% improvement from baseline with no more than 1 of the remaining variables worsened by >= 30%.
Measured: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Activity Joint Count-71, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using visual analog scale (VAS), Childhood Health Questionnaire Disability Index (CHAQ-DI) and hs-CRP. Activity joint count-71 was calculated as sum (joints with active arthritis)*(71/number of joints with assessment).
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Activity Joint Count-71, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Activity joint count-71 was calculated as sum (joints with active arthritis)*(71/number of joints with assessment).
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Limited Motion Joint Count, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Limited motion joint count was calculated as sum (joints with limited motion)*(67/number of joints with assessment).
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Limited Motion Joint Count, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Limited motion joint count was calculated as sum (joints with limited motion)*(67/number of joints with assessment).
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Childhood Health Assessment Questionnaire Disability Index, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. The CHAQ questionnaire consists of 30 questions referring to 8 domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities. Each domain is scored on a 4 point scale ranges from 0 to 3: 0 (without any difficulty), 1 (with some difficulty), 2 (with much difficulty), and 3 (unable to do). An additional response of "not applicable" is available to indicate activities the participant is unable to perform because he/she is too young. The CHAQ-DI total score is the sum of the domain scores divided by the number of domains that have a non-missing score. This overall score ranges from 0 (best) to 3 (worst). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Childhood Health Assessment Questionnaire Disability Index, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. The CHAQ questionnaire consists of 30 questions referring to 8 domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip and activities. Each domain is scored on a 4 point scale ranges from 0 to 3: 0 (without any difficulty), 1 (with some difficulty), 2 (with much difficulty), and 3 (unable to do). An additional response of "not applicable" is available to indicate activities the participant is unable to perform because he/she is too young. The CHAQ-DI total score is the sum of the domain scores divided by the number of domains that have a non-missing score. This overall score ranges from 0 (best) to 3 (worst). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, C-Reactive Protein, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Serum concentrations of hs-CRP was determined to assess the PD effects of sarilumab.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, C-Reactive Protein, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Serum concentrations of hs-CRP was determined to assess the PD effects of sarilumab.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Physician Global Assessment of Disease Activity, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Physician global assessment of disease activity was assessed on an anchored 100 mm horizontal VAS score ranging from 0 to 10 where 0 is considered the best disease activity (no disease activity) and 10 the worst (most disease activity). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Physician Global Assessment of Disease Activity, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Physician global assessment of disease activity was assessed on an anchored 100 mm horizontal VAS score ranging from 0 to 10 where 0 is considered the best disease activity (no disease activity) and 10 the worst (most disease activity). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Participant/Parent Assessment of Overall Well-Being, at Week 12
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Participant/parent assessment of overall well-being was rated on an anchored 100 mm horizontal VAS score ranging from 0 to 10 where 0 is considered the best disease activity (no disease activity) and 10 the worst (most disease activity). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Participant/Parent Assessment of Overall Well-Being, at Weeks 12, 24, 48, 96, and 156
The JIA ACR components included joints with active arthritis (0 to 71 joints), joints with limited motion (0 to 67 joints), physician global assessment of disease activity and participant/parent assessment of overall well-being using VAS, CHAQ-DI and hs-CRP. Participant/parent assessment of overall well-being was rated on an anchored 100 mm horizontal VAS score ranging from 0 to 10 where 0 is considered the best disease activity (no disease activity) and 10 the worst (most disease activity). Higher scores indicate worse outcome.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Mean Change From Baseline in Juvenile Arthritis Disease Activity Score (JADAS-27) at Week 12
The JADAS is used for assessment of disease activity, and it includes 4 measures: Physician global assessment of disease activity (VAS range: 0 to10; where 0= no activity and 10= maximum activity), parent/participant global assessment of well-being (VAS range: 0 to 10; where 0= no activity and10= maximum activity), count of joints with active disease (range: 0 to 27; where 0= no activity and 27= maximum activity), and index of inflammation determined by hs-CRP or ESR (normalized scale range: 0 to 10; where 0= no disease activity and 10= maximum disease activity). The JADAS total score is calculated as the simple sum of the scores of its 4 components. The total score ranges from 0 to 57 where 0= no disease activity and 57= maximum disease activity. Higher scores indicate higher disease activity. Clinical JADAS-27 is without CRP or ESR component and score ranges from 0 to 47 where 0= no disease activity and 47= maximum disease activity.
Measured: Baseline (Day 1) and Week 12
Cohort 2: Mean Change From Baseline in Juvenile Arthritis Disease Activity Score at Weeks 12, 24, 48, 96, and 156
The JADAS is used for assessment of disease activity, and it includes 4 measures: Physician global assessment of disease activity (VAS range: 0 to10; where 0= no activity and 10= maximum activity), parent/participant global assessment of well-being (VAS range: 0 to 10; where 0= no activity and10= maximum activity), count of joints with active disease (range: 0 to 27; where 0= no activity and 27= maximum activity), and index of inflammation determined by hs-CRP or ESR (normalized scale range: 0 to 10; where 0= no disease activity and 10= maximum disease activity). The JADAS total score is calculated as the simple sum of the scores of its 4 components. The total score ranges from 0 to 57 where 0= no disease activity and 57= maximum disease activity. Higher scores indicate higher disease activity. Clinical JADAS-27 is without CRP or ESR component and score ranges from 0 to 47 where 0= no disease activity and 47= maximum disease activity.
Measured: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)
An adverse events (AEs) is any untoward medical occurrence in a participant or in a clinical investigation participant administered a medicinal product and which does not necessarily have a causal relationship with the study treatment. An SAE is any untoward medical occurrence that at any dose results in death or is life-threatening or requires inpatient hospitalization or prolongation of existing hospitalization or results in persistent or significant disability/incapacity or is a congenital anomaly/birth defect or is an important medical event. TEAEs are defined as AEs that develop or worsen during the on-treatment period [that is, from the time of first dose of study treatment up to 6 weeks after the last administration of the study treatment].
Measured: From the first administration of study treatment (Day 1) up to end of treatment period, maximum of 156 weeks for portions 1 and 2 and 96 weeks for portion 3
Number of Participants With Local Site Reactions
Participants were observed for at least 30 minutes after each study treatment administration either on site or at home and any local reactions were noted in the diary regardless of being clinically significant.
Measured: From the first administration of study treatment (Day 1) up to end of treatment period, maximum of 156 weeks for portions 1 and 2 and 96 weeks for portion 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2016 (estimated)
- Study start6 Sep 2016
- Primary completion8 Apr 2022
- Study completion27 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Juvenile Idiopathic ArthritisStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 102 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02776735
- Sponsor's protocol ID
- DRI13925
- Other IDs
- U1111-1177-3487, 2015-003999-79, 2015
- Acronym
- SKYPP
- Status last verified
- Oct 2024
- First submitted
- 16 May 2016
- Last updated
- 15 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
De Benedetti F, Penades IC, Nikishina I, Foeldvari I, Spindler AJ, Kozlova A, Rubio-Perez N, Quartier P, Zuber Z, Barria R, Clemente D, Cornejo GV, Marzan K, Liu N, Xu C, DiMartino S, Giannelou A, Cao-Ghoul F, Akinlade B, Baret-Cormel L. Sarilumab in Polyarticular-Course Juvenile Idiopathic Arthritis: Dose-Finding and 1-Year Analysis of a Phase 2b, Open-Label, Multicenter Study. Arthritis Care Res (Hoboken). 2026 Jun;78(6):749-760. doi: 10.1002/acr.25692. Epub 2026 Mar 24.
PubMed 41235938
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered With Subcutaneous (SC) Injection, in Children and Adolescents, Aged 2 to 17 Years, With Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) Followed by an Extension Phase
- ClinicalTrials.gov ID
- NCT02776735
- Acronym / sponsor ID
- SKYPP / DRI13925
- Conditions
- Juvenile Idiopathic Arthritis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 102 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Collaborators
- Regeneron Pharmaceuticals
- Study start
- 6 Sep 2016 (actual)
- Primary completion
- 8 Apr 2022 (actual)
- Study completion
- 27 Dec 2023 (actual)
- First posted
- 18 May 2016 (estimated)
- Results
- Posted 15 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
To describe the pharmacokinetic (PK) profile of sarilumab in participants aged 2-17 years with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) in order to identify the dose and regimen for adequate treatment of this population
Secondary Objective:
To describe the pharmacodynamic (PD) profile, the efficacy and the long-term safety of sarilumab in participants with pcJIA.
Detailed description
For 73 participants enrolled in the dose-finding and second portions, the total study duration per participant was up to 166 weeks that consists of a 4- week screening, a 12-week core treatment phase, a 144-week extension phase, and a 6-week post-treatment follow-up. For 29 participants enrolled in the third portion, the total study duration per participant was up to 106 weeks that consists of a 4- week screening, a 12-week core treatment phase, a 84-week extension phase, and a 6-week post-treatment follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sarilumab | Experimental | Drug: SarilumabParticipants received one of three ascending dose regimens of sarilumab by subcutaneous (SC) injection based on body weight. All the participants received the selected dose regimen once this was identified. Sarilumab was given during 12-week core treatment phase followed by an extension treatment phase (144 weeks for 73 participants enrolled in dose-finding and second portions and 84 weeks for approximately 29 participants enrolled in third portion) |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male and female participants aged ≥2 and ≤17 years (or country specified age requirement) at the time of the screening visit.
- Diagnosis of rheumatoid factor-negative or rheumatoid factor positive polyarticular Juvenile Idiopathic Arthritis (JIA) subtype or oligoarticular extended JIA subtype according to the International League of Associations for Rheumatology (ILAR) 2001 Juvenile Idiopathic Arthritis Classification Criteria with at least 5 active joints as per American College of Rheumatology (ACR) definition for "active arthritis" at Screening
- Participant with an inadequate response to current treatment and considered as a candidate for a biologic disease modifying antirheumatic drug (DMARD) as per investigator's judgment
Key exclusion criteria (16 listed)
- Body weight <10 kg or >60 kg for participants enrolled in the 3 ascending dose cohorts, then body weight <10 kg for participants subsequently enrolled at the selected dose-regimen.
- If nonsteroidal anti-inflammatory drugs (NSAIDs) [including cyclo oxygenase-2 inhibitors (COX-2)] taken, dose stable for <2 weeks prior to the baseline visit and/or dosing prescribed outside of approved label.
- If non-biologic DMARD taken, dose stable for <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 0.5 mg/kg/day (or 30 mg/day) within 2 weeks prior to baseline.
- Use of parenteral or intra-articular glucocorticoid injection within 4 weeks prior to baseline.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SarilumabAlso: SAR153191 (REGN88)Pharmaceutical form:Solution Route of administration: Subcutaneous | Drug | Sarilumab |
Primary outcome
Maximum Serum Concentration (Cmax) of Sarilumab at Week 12
Time frame: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
The Cmax was defined as maximum serum concentration. The values reported are mean and standard deviation.
Area Under the Serum Concentration Versus Time Curve Using the Trapezoidal Method During a Dose Interval (AUC0-t) of Sarilumab at Week 12
Time frame: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
The AUC0-t was defined as area under the concentration in serum versus time curve calculated using the trapezoidal method during a dose interval (tau). The values reported are mean and standard deviation.
Concentration Before Treatment Administration During Repeated Dosing (Ctrough) of Sarilumab at Week 12
Time frame: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12
The Ctrough was defined as concentration observed before treatment administration during repeated dosing from baseline to Week 12. The values reported are mean and standard deviation.
30 secondary outcomes
Cohorts 1 and 3: Change From Baseline in High-Sensitivity C-reactive Protein (Hs-CRP) at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in High-Sensitivity C-reactive Protein at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Change From Baseline in Interleukin-6 (IL-6) at Week 12
Time frame: Baseline (Day 1) and Week 12
Change From Baseline in Total Soluble Interleukin-6 Receptor (sIL-6R) at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology (JIA ACR) 30 Response at Week 12
Time frame: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 30 Response at Weeks 12, 24, 48, 96, and 156
Time frame: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 50 Response at Week 12
Time frame: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 50 Response at Weeks 12, 24, 48, 96, and 156
Time frame: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 70 Response at Week 12
Time frame: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 70 Response at Weeks 12, 24, 48, 96, and 156
Time frame: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 90 Response at Week 12
Time frame: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 90 Response at Weeks 12, 24, 48, 96, and 156
Time frame: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 100 Response at Week 12
Time frame: Week 12
Cohort 2: Percentage of Participants With Juvenile Idiopatic Arthritis American College of Rheumatology 100 Response at Weeks 12, 24, 48, 96, and 156
Time frame: Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Activity Joint Count-71, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Activity Joint Count-71, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Limited Motion Joint Count, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Limited Motion Joint Count, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Childhood Health Assessment Questionnaire Disability Index, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Childhood Health Assessment Questionnaire Disability Index, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, C-Reactive Protein, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, C-Reactive Protein, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Physician Global Assessment of Disease Activity, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Physician Global Assessment of Disease Activity, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Participant/Parent Assessment of Overall Well-Being, at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Change From Baseline in Juvenile Idiopatic Arthritis American College of Rheumatology Component, Participant/Parent Assessment of Overall Well-Being, at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Cohorts 1 and 3: Mean Change From Baseline in Juvenile Arthritis Disease Activity Score (JADAS-27) at Week 12
Time frame: Baseline (Day 1) and Week 12
Cohort 2: Mean Change From Baseline in Juvenile Arthritis Disease Activity Score at Weeks 12, 24, 48, 96, and 156
Time frame: Baseline (Day 1) and Weeks 12, 24, 48, 96, and 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)
Time frame: From the first administration of study treatment (Day 1) up to end of treatment period, maximum of 156 weeks for portions 1 and 2 and 96 weeks for portion 3
Number of Participants With Local Site Reactions
Time frame: From the first administration of study treatment (Day 1) up to end of treatment period, maximum of 156 weeks for portions 1 and 2 and 96 weeks for portion 3
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41235938De Benedetti F, Penades IC, Nikishina I, Foeldvari I, Spindler AJ, Kozlova A, Rubio-Perez N, Quartier P, Zuber Z, Barria R, Clemente D, Cornejo GV, Marzan K, Liu N, Xu C, DiMartino S, Giannelou A, Cao-Ghoul F, Akinlade B, Baret-Cormel L. Sarilumab in Polyarticular-Course Juvenile Idiopathic Arthritis: Dose-Finding and 1-Year Analysis of a Phase 2b, Open-Label, Multicenter Study. Arthritis Care Res (Hoboken). 2026 Jun;78(6):749-760. doi: 10.1002/acr.25692. Epub 2026 Mar 24.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
30 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sarilumab in people with Juvenile Idiopathic Arthritis, run by Sanofi at 30 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Poland, Germany, Spain, Russia, Argentina and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Serum Concentration (Cmax) of Sarilumab at Week 12 (time frame: Pre-dose on Days 1, 3, 5, 8, 12 and Weeks 2, 4, 8 and 12). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02776735, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2024.