Clinical trial • Phase IV • Immunology | Rare Disease

SARILUMAB for Systemic juvenile idiopathic arthritis | Juvenile idiopathic arthritis

Phase IV trial of SARILUMAB for Systemic juvenile idiopathic arthritis | Juvenile idiopathic arthritis.

Overview

Trial Therapeutic Area
Immunology | Rare Disease
Trial Disease
Systemic juvenile idiopathic arthritis | Juvenile idiopathic arthritis
Trial Stage
Phase IV
Drug Modality
Monoclonal antibody
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
07-06-2024
First CTIS Authorization Date
17-07-2024

Trial design

open-label, none/not specified-controlled, adaptive Phase IV trial across 27 sites in Italy, Germany, Ireland and others.

Open Label
Yes
Comparator
None/Not specified
Adaptive
True, sequential ascending repeated dose‑finding with dose‑escalation cohorts (sequential, ascending, repeated dose‑finding design).
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
45
Trial Duration For Participant
1092

Eligibility

Recruits 45 paediatric patients.

Pregnancy Exclusion
Pregnant or breast-feeding female adolescent patients.
Vulnerable Population
The trial includes vulnerable populations (children and adolescents aged 1–17 years). Consent is managed using age-appropriate subject information and informed consent/assent documents (parent/guardian consent and child/adolescent assent forms). Country-specific ICF and assent forms are available (multiple languages and versions for different age groups, and forms for participants turning 16/18 and for parental/legal representative consent).

Inclusion criteria

  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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

  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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)."}
  • {"criterion_text":"- Treatment with any investigational biologic or non-biologic product within 8 weeks or 5 half-lives prior to baseline, whichever is longer."}
  • {"criterion_text":"- Exclusion related to tuberculosis."}
  • {"criterion_text":"- Exclusion criteria related to past or current infection other than tuberculosis."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- Exclusion related to history of a systemic hypersensitivity reaction to any biologic drug and known hypersensitivity to any constituent of the product."}
  • {"criterion_text":"- Laboratory abnormalities at the screening visit (identified by the central laboratory)."}
  • {"criterion_text":"- Severe cardiac disease due to sJIA."}
  • {"criterion_text":"- Pregnant or breast-feeding female adolescent patients."}
  • {"criterion_text":"- Uncontrolled severe systemic symptoms and/or Macrophage Activation Syndrome (MAS) within 6 months prior to screening."}
  • {"criterion_text":"- History of or ongoing interstitial lung disease, pulmonary hypertension, pulmonary alveolar proteinosis."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- If oral glucocorticoid taken, dose exceeding equivalent prednisone dose 1 mg/kg/day (or 60 mg/day) within 3 days prior to baseline."}
  • {"criterion_text":"- Use of parenteral or intra-articular glucocorticoid injection within 4 weeks prior to baseline."}
  • {"criterion_text":"- Prior treatment with anti-interleukin 6 (IL-6) or IL-6 receptor (IL-6R) antagonist therapies, including but not limited to tocilizumab or sarilumab."}
  • {"criterion_text":"- 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)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Assessment of PK parameter: maximum serum concentration observed (Cmax); Up to Week 12","definition_or_measurement_approach":"Maximum serum concentration observed (Cmax) measured through pharmacokinetic sampling up to Week 12."}
  • {"endpoint_text":"- 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","definition_or_measurement_approach":"AUC0-t calculated using the trapezoidal method over a dose interval from serum concentration versus time measurements up to Week 12."}
  • {"endpoint_text":"- Assessment of PK parameter: Concentration observed before treatment administration during repeated dosing (Ctrough); Up to Week 12","definition_or_measurement_approach":"Ctrough is the pre-dose (trough) serum concentration measured prior to treatment administration during repeated dosing, assessed up to Week 12."}

Secondary endpoints

  • {"endpoint_text":"- Number of Adverse events; Core treatment phase: Up to Week 12, Extension phase:Up to Week 162","definition_or_measurement_approach":"Count and reporting of adverse events during core treatment (up to Week 12) and extension phase (up to Week 162)."}
  • {"endpoint_text":"- Acceptability assessments (local tolerability); Core treatment phase: Up to Week 12, Extension phase:Up to Week 156","definition_or_measurement_approach":"Assessment of local tolerability and acceptability (e.g., local injection site reactions) during core and extension phases up to specified weeks."}
  • {"endpoint_text":"- 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.","definition_or_measurement_approach":"Proportion of participants with IGA score below prespecified threshold on 1–100 VAS at specified timepoints."}
  • {"endpoint_text":"- 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.","definition_or_measurement_approach":"Proportion of participants with parent/patient global assessment (PGA) of well-being below prespecified threshold on 1–100 VAS at specified timepoints."}
  • {"endpoint_text":"- Assessment 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.","definition_or_measurement_approach":"Assessment of proportion/status of participants meeting criteria for clinically inactive disease at scheduled timepoints."}
  • {"endpoint_text":"- 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.","definition_or_measurement_approach":"Change from baseline in glucocorticoid dose/use measured at scheduled visits."}
  • {"endpoint_text":"- Juvenile Idiopathic Arthritis ACR30/50/70/90/100 (in the absence of fever) response rate. Population according to the 2001 ILAR classification. Core treatment phase: Up to Week 12 Extension phase:At weeks 24, 48, and every 24 weeks up to Week 156.","definition_or_measurement_approach":"ACR response rates (ACR30/50/70/90/100) assessed per JIA ACR criteria (absence of fever) at specified visits."}
  • {"endpoint_text":"- Change from baseline in individual JIA ACR components. Population according to the 2001 ILAR classification. Core treatment phase: Up to Week 12 Extension phase:At weeks 24, 48, and every 24 weeks up to Week 156.","definition_or_measurement_approach":"Changes from baseline for each component of the JIA ACR measures at scheduled timepoints."}
  • {"endpoint_text":"- Change from baseline in Juvenile Arthritis Disease Activity Score-27 (JADAS). Population according to the 2001 ILAR classification. Core treatment phase: Up to Week 12 Extension phase: Up to Week 156.","definition_or_measurement_approach":"Change from baseline in JADAS-27 score measured at scheduled visits up to Week 156."}
  • {"endpoint_text":"- Assessment of participants with disease-related symptoms. Population according to the 2024 EULAR / PReS. At Week 4.","definition_or_measurement_approach":"Assessment of disease-related symptoms according to 2024 EULAR/PReS criteria at Week 4."}
  • {"endpoint_text":"- Changes in IL-6 associated biomarkers. Population according to the 2001 ILAR classification and the 2024 EULAR / PReS. Up to week 12.","definition_or_measurement_approach":"Measurement of IL-6–associated biomarkers and change from baseline up to Week 12."}

Recruitment

Planned Sample Size
45
Recruitment Window Months
125
Consent Approach
Informed consent is obtained from parent(s)/legal guardian(s) for minor participants, with age‑appropriate assent forms for children and adolescents. Separate ICF/assent templates are provided for different age groups (e.g., 6–10, 11–15, 16–17, adolescent-turned-adult and adult forms), parent/guardian information forms, partner/pregnancy information and pharmacogenetic consent forms. Documents exist in multiple languages corresponding to trial countries.

Methods

  • Patient-facing recruitment materials and arrangements available (documented as K1/K2 recruitment arrangements and K2 recruitment materials).
  • Use of illustrated/comic-book materials targeted to children (e.g., 'K2-recruitment-material-comic-book-understanding-your-study' and country-specific comic materials) aimed at explaining the study to pediatric participants.
  • Welcome guides, parent brochures and patient 'passport' materials for families (documents titled welcome-guide, parentsbrochure, skypstudypassport, country-specific versions).
  • Country-specific recruitment arrangements and materials available (multiple language versions: EN, ES, FR, DE, IT, BG, FI, HU, PT, RO, EL, etc.) associated with the member state submissions.

Geography

Total Number Of Sites
27
Total Number Of Participants
37

Italy

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
06-08-2024
Processing Time Days
42
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Pediatric Rheumatology International Trials Organisation
Department Name
Reumatologia
Principal Investigator Name
Alessandro Consolaro
Principal Investigator Email
alessandroconsolaro@gaslini.org
Contact Person Name
Alessandro Consolaro
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Pediatria Immunoreumatologia
Principal Investigator Name
Giovanni Filocamo
Principal Investigator Email
giovanni.filocamo@policlinico.mi.it
Contact Person Name
Giovanni Filocamo
Site Name
Bambino Gesu Childrens Hospital
Department Name
U.O.C. Reumatologia
Principal Investigator Name
Fabrizio De Benedetti
Principal Investigator Email
fabrizio.debenedetti@opbg.net
Contact Person Name
Fabrizio De Benedetti
Contact Person Email
fabrizio.debenedetti@opbg.net

Germany

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
19-07-2024
Processing Time Days
24
Number Of Sites
5
Number Of Participants
7

Sites

Site Name
Gesundheit Nord gGmbH Klinikverbund Bremen
Department Name
Eltern-Kind-Zentrum Prof. Hess
Principal Investigator Name
Frank Weller-Heinemann
Principal Investigator Email
Frank.Weller@gesundheitnord.de
Contact Person Name
Frank Weller-Heinemann
Contact Person Email
Frank.Weller@gesundheitnord.de
Site Name
HELIOS Klinikum Berlin-Buch GmbH
Department Name
Kinder- und Jugendmedizin
Principal Investigator Name
Ralf Trauzeddel
Principal Investigator Email
ralf.trauzeddel@helios-kliniken.de
Contact Person Name
Ralf Trauzeddel
Site Name
Asklepios Klinik Sankt Augustin GmbH
Department Name
Allgmeine Kinderheilkunde
Principal Investigator Name
Gerd Horneff
Principal Investigator Email
g.horneff@asklepios.com
Contact Person Name
Gerd Horneff
Contact Person Email
g.horneff@asklepios.com
Site Name
Zentrum Fuer Kinder Und Jugendrheumatologie
Department Name
Pediatric Clinic / Kompetenzzentrum fur Sklerodermie im Kindes- und Jugendalter
Principal Investigator Name
Ivan Foeldvari
Principal Investigator Email
foeldvari@t-online.de
Contact Person Name
Ivan Foeldvari
Contact Person Email
foeldvari@t-online.de
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Paediatrie
Principal Investigator Name
Kirsten Minden
Principal Investigator Email
kirsten.minden@charite.de
Contact Person Name
Kirsten Minden
Contact Person Email
kirsten.minden@charite.de

Ireland

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
17-07-2024
Processing Time Days
22
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Our Lady's Childrens Hospital
Department Name
Research and Innovation
Principal Investigator Name
Orla Killeen
Principal Investigator Email
orla.killeen@olchc.ie
Contact Person Name
Orla Killeen
Contact Person Email
orla.killeen@olchc.ie

Spain

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
18-07-2024
Processing Time Days
23
Number Of Sites
7
Number Of Participants
4

Sites

Site Name
Hospital Universitario Regional De Malaga
Department Name
Servicio de Reumatología Pediátrica
Principal Investigator Name
Esmeralda Nunez Cuadros
Principal Investigator Email
esmeralda.nunez.sspa@juntadeandalucia.es
Contact Person Name
Esmeralda Nunez Cuadros
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Unidad de Reumatología Pediátrica
Principal Investigator Name
Lucia Lacruz Perez
Principal Investigator Email
lacruz_lucper@gva.es
Contact Person Name
Lucia Lacruz Perez
Contact Person Email
lacruz_lucper@gva.es
Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Unidad de Reumatología Pediátrica
Principal Investigator Name
Jordi Ánton López
Principal Investigator Email
jordi.anton@sjd.es
Contact Person Name
Jordi Ánton López
Contact Person Email
jordi.anton@sjd.es
Site Name
Hospital Universitario La Paz
Department Name
Unidad de Reumatología Pediátrica
Principal Investigator Name
Agustín Remesal Camba
Principal Investigator Email
agusremesal@hotmail.com
Contact Person Name
Agustín Remesal Camba
Contact Person Email
agusremesal@hotmail.com
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Unidad de Reumatología Pediátrica
Principal Investigator Name
Daniel Clemente Garulo
Principal Investigator Email
daniel.clemente@salud.madrid.org
Contact Person Name
Daniel Clemente Garulo
Site Name
Hospital La Milagrosa S.A.
Department Name
Servicio de Reumatología
Principal Investigator Name
Boris Anthony Blanco Caceres
Principal Investigator Email
blancobc@ext.vithas.es
Contact Person Name
Boris Anthony Blanco Caceres
Contact Person Email
blancobc@ext.vithas.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Servicio de Reumatología Pediátrica
Principal Investigator Name
Estefania Moreno Rizafa
Principal Investigator Email
estefania.moreno@vallhebron.cat
Contact Person Name
Estefania Moreno Rizafa

Bulgaria

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
18-07-2024
Processing Time Days
23
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
University Multiprofile Hospital For Active Treatment Saint Georgi EAD
Department Name
Pediatric Clinic
Principal Investigator Name
Lyubov Chochkova-Bukova
Principal Investigator Email
lbukova@yahoo.com
Contact Person Name
Lyubov Chochkova-Bukova
Contact Person Email
lbukova@yahoo.com

France

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
18-07-2024
Processing Time Days
23
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
Hospices Civils De Lyon
Department Name
Service de Nephrologie, Rhumatologie et dermatologie Pediatriques
Principal Investigator Name
Alexandre Belot
Principal Investigator Email
alexandre.belot@chu-lyon.fr
Contact Person Name
Alexandre Belot
Contact Person Email
alexandre.belot@chu-lyon.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Unite d'Immuno-hematologie et Rhumatologie pediatrique
Principal Investigator Name
Pierre Quartier-dit-Maire
Principal Investigator Email
pierre.quartier@aphp.fr
Contact Person Name
Pierre Quartier-dit-Maire
Contact Person Email
pierre.quartier@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Service de Medecine pediatrique, des Maladies Infectieuses et d'Immunologie Clinique
Principal Investigator Name
Eric Jeziorski
Principal Investigator Email
e-jeziorski@chu-montpellier.fr
Contact Person Name
Eric Jeziorski
Contact Person Email
e-jeziorski@chu-montpellier.fr

Finland

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
19-07-2024
Processing Time Days
24
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Meilahti Hospital
Department Name
Hematology
Principal Investigator Name
Kristiina Aalto
Principal Investigator Email
kristiina.aalto@hus.fi
Contact Person Name
Kristiina Aalto
Contact Person Email
kristiina.aalto@hus.fi

Hungary

Earliest CTIS Part Ii Submission Date
05-12-2025
Latest Decision Or Authorization Date
22-12-2025
Processing Time Days
17
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Semmelweis University
Department Name
Reumatológia-Immunológia Osztály
Principal Investigator Name
Tamas Constantin
Principal Investigator Email
constantin.tamas@semmelweis.hu
Contact Person Name
Tamas Constantin
Contact Person Email
constantin.tamas@semmelweis.hu

Greece

Earliest CTIS Part Ii Submission Date
14-11-2025
Latest Decision Or Authorization Date
09-01-2026
Processing Time Days
56
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Nosokomeio Paidon I Agia Sofia
Department Name
A' pediatric Clinic of University of Athens
Principal Investigator Name
Eleni Tsitsami
Principal Investigator Email
e.tsitsami@paidon-agiasofia.gr
Contact Person Name
Eleni Tsitsami
Contact Person Email
e.tsitsami@paidon-agiasofia.gr
Site Name
Ippokratio General Hospital Of Thessaloniki
Department Name
A’ pediatric Clinic of Aristotle University of Thessaloniki
Principal Investigator Name
Vasiliki Sgouropoulou
Principal Investigator Email
vsgouro@auth.gr
Contact Person Name
Vasiliki Sgouropoulou
Contact Person Email
vsgouro@auth.gr

Romania

Earliest CTIS Part Ii Submission Date
19-01-2026
Latest Decision Or Authorization Date
16-02-2026
Processing Time Days
28
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Spitalul Clinic De Urgenta Pentru Copii Cluj-Napoca
Department Name
First Pediatric clinic
Principal Investigator Name
Mirela Crisan
Principal Investigator Email
Mirelacrisan_cluj@yahoo.co.uk
Contact Person Name
Mirela Crisan
Contact Person Email
Mirelacrisan_cluj@yahoo.co.uk

Portugal

Earliest CTIS Part Ii Submission Date
07-01-2026
Latest Decision Or Authorization Date
21-01-2026
Processing Time Days
14
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Unidade Local De Saude De Sao Jose E.P.E.
Department Name
Pediatric Rheumatology Unit
Principal Investigator Name
Marta Conde
Principal Investigator Email
marta.conde@ulssjose.min-saude.pt
Contact Person Name
Marta Conde
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Rheumatology and Pediatrics Department
Principal Investigator Name
Filipa Ramos
Principal Investigator Email
filipa.ramos@ulssm.min-saude.pt
Contact Person Name
Filipa Ramos

Sponsor

Primary sponsor

Full Name
Sanofi-Aventis Recherche & Developpement
Organisation Type
Pharmaceutical company
Country Of Registered Address
France

Third parties

  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Spain","full_name":"Alcura Health Espana S.A.","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
  • {"country":"Italy","full_name":"Ospedale Pediatrico Bambino Gesu","duties_or_roles":"code 13","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Early Development Laboratories Inc.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"code 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"code 15; Centralized 24-Hour Emergency System: eSMS","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Regeneron Pharmaceuticals Inc.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
sarilumab
Active Substance
SARILUMAB
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
Subcutaneous injection
Authorisation Status
Authorised (prodAuthStatus:1)
Maximum Dose
300 mg (maxDailyDoseAmount: 300 mg indicated in product data)

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