Extension Study Evaluating the Safety and Efficacy of Subcutaneous Tocilizumab (RoActemra/Actemra) Administration in Systemic and Polyarticular-Course Juvenile Idiopathic Arthritis

Long-Term Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Tocilizumab in Patients With Polyarticular-Course and Systemic Juvenile Idiopathic Arthritis

This open-label extension of the JIGSAW studies (WA28117 [NCT01904279] and WA28118 [NCT01904292]) is designed to evaluate the long-term safety and efficacy of subcutaneous (SC) tocilizumab treatment in participants with polyarticular-course and systemic juvenile idiopathic arthritis (pJIA and sJIA). Participants from the 2 JIGSAW studies will continue to receive 162 milligrams (mg) of SC tocilizumab with treatment schedule according to arthritis subtype and body weight. Participants will receive the treatment until commercial availability of the drug or for a maximum of 5 years, whichever is earlier.

Study Overview

• Status: Completed
• Conditions: Juvenile Idiopathic Arthritis
• Intervention / Treatment: Drug: Tocilizumab

• Study Type: Interventional
• Enrollment (Actual): 82
• Phase: Phase 1

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, C1270AAN
    • Hospital Gral de Niños Pedro Elizalde
  • Cordoba, Argentina, X5000XAF
    • Hospital de Ninos de la Santisima Trinidad; Hematología
• Australia
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital; Paediatric Rheumatology
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Royal Children'S Hospital; Paediatric Rheumatology
• Brazil
  • SP
    • Sao Paulo, SP, Brazil, 04026-000
      • Universidade Federal de Sao Paulo - UNIFES
    • Sao Paulo, SP, Brazil, 05403-900
      • Hospital das Clinicas - FMUSP
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T3B 6A8
      • Alberta Children's Hospital
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
• France
  • Le Kremlin Bicêtre, France, 94275
    • CH de Bicêtre; Pediatrie Generale
• Germany
  • Berlin, Germany, 13353
    • Charité Campus; Virchow Klinikum Berlin
  • Freiburg, Germany, 79106
    • Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Pädiatrische Infektio- u. Rheumatologie
  • Sankt Augustin, Germany, 53757
    • Asklepios Klinik; Zentrum für Allgemeine Pädiatrie und Neonatologie
• Italy
  • Lazio
    • Roma, Lazio, Italy, 00165
      • Irccs Ospedale Pediatrico Bambin Gesu - Dip. Di Medicina
• Mexico
  • Mexico, Mexico, 06720
    • Hospital Infantil de México "Federico Gomez"; Rheumatology
  • Monterrey, Mexico, 64460
    • Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
• Russian Federation
  • Moscow, Russian Federation, 119991
    • SI Sceintific children health center RAMS
• Spain
  • Madrid, Spain, 28034
    • Hospital Ramon y Cajal ; Servicio de Reumatologia
  • Madrid, Spain, 28046
    • Hospital de La Paz; Unidad de Reumatologia Pediatrica
  • Valencia, Spain, 46026
    • Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica
  • Barcelona
    • Esplugas DE Llobregat, Barcelona, Spain, 08950
      • Hospital Sant Joan De Deu; Servicio de Reumatologia Pediatrica
• United Kingdom
  • Bristol, United Kingdom, BS2 8BJ
    • Bristol Royal Hospital for Children; Rheumatology Department
  • Liverpool, United Kingdom, L12 2AP
    • Alder Hey Children's NHS Foundation Trust
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital Research Institute
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Hospital
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack University Medical Center; Pediatric Rheumatology
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Duke University
  • Ohio
    • Cincinnati, Ohio, United States, 45229-3039
      • Cincinnati Children'S Hospital Medical Center; Division of Rheumatology
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Fndn
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74135
      • Healthcare Research Consultants
  • Utah
    • Salt Lake City, Utah, United States, 84109
      • University of Utah; Immunology/Rheumatology/Allergy
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Completion of either of the JIGSAW studies, study WA28117 (for participants with pJIA) or study WA28118 (for participants with sJIA)
• Adequate disease control with the use of SC tocilizumab(TCZ)(comparable to the use of IV TCZ, if received prior to enrollment in the JIGSAW study), per clinical judgment of the investigator
• For participants of reproductive potential: agreement to remain abstinent or use of effective contraception as defined by the study protocol

Exclusion Criteria:

• Prior discontinuation of SC tocilizumab because of inadequate clinical response during participation in a JIGSAW study
• Poorly controlled disease (in opinion of treating physician) despite treatment with SC tocilizumab in the JIGSAW study
• Prior discontinuation of intravenous tocilizumab because of inadequate clinical response or safety events (including hypersensitivity)
• Therapy with biologic agents (except tocilizumab) in the period between completion of the JIGSAW study and screening for the current study
• Concurrent treatment with disease-modifying anti-rheumatic drugs (DMARDs) (including methotrexate), nonsteroidal anti-inflammatory drugs (NSAIDs), and oral corticosteroids is permitted at the discretion of the investigator
• Use of live or attenuated vaccines and immunosuppressants, such as cyclosporine and cyclophosphamide
• Any significant concurrent medical or surgical conditions or findings that would jeopardize the participant's safety or ability to complete the study, including but not limited to disease of the nervous, renal, hepatic, cardiac, pulmonary, gastric, or endocrine system or any infection
• History of alcohol, drug, or chemical abuse within 6 months prior to screening
• History of atypical tuberculosis (TB) or active TB requiring treatment within 2 years prior to screening
• Known human immunodeficiency virus infection or other acquired forms of immune compromise or inborn conditions characterized by a compromised immune system
• Clinical signs or symptoms of acute or chronic viral hepatitis or chronic autoimmune hepatitis arising since enrollment in the JIGSAW study
• History of concurrent serious gastrointestinal disorders, such as ulcer or inflammatory bowel disease, Crohn's disease, ulcerative colitis, or other symptomatic lower gastrointestinal conditions
• History of or current cancer or lymphoma
• Uncontrolled diabetes mellitus with elevated glycosylated hemoglobin (HbA1c), defined with the use of age-specific standards
• Any abnormal laboratory values, an elevation of hepatic transaminases ([aspartate aminotransferase [AST] or alanine aminotransferase [ALT]), lowering of neutrophil count, or thrombocytopenia attributed to tocilizumab use by investigator at screening, the participant may be enrolled; however, the initial tocilizumab dose may be delayed to adhere to the protocol risk mitigation strategy or per the investigator's clinical judgment
• Prior stem cell transplant at any time

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tocilizumab; Participants will receive tocilizumab until the commercial availability of the drug or up to 5 years, whichever is earlier.	Drug: Tocilizumab; 162 mg tocilizumab will be administered by SC injection at the following intervals: pJIA participants less than (<) 30 kilograms (kg): every 3 weeks; pJIA participants greater than or equal to (>=) 30 kg: every 2 weeks; sJIA participants <30 kg: every 2 weeks; sJIA participants >/= 30 kg: once weekly; Other Names: RoActemra, Actemra

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Juvenile Arthritis Disease Activity Score (JADAS-71)	Baseline up to 3 years
Percentage of Participants With Adverse Events (AEs), Serious AEs (AEs) and AEs of Special Interest	Baseline up to 5 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Childhood Health Assessment Questionnaire (CHAQ) Score	Baseline up to 3 years
Percentage of Participants With Protocol Defined Inactive Disease/Clinical Remission	Baseline up to 3 years

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (Actual): July 16, 2014
• Primary Completion (Actual): November 24, 2021
• Study Completion (Actual): November 24, 2021

Study Registration Dates

• First Submitted: June 13, 2014
• First Submitted That Met QC Criteria: June 16, 2014
• First Posted (Estimate): June 17, 2014

Study Record Updates

• Last Update Posted (Actual): February 23, 2022
• Last Update Submitted That Met QC Criteria: February 22, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Arthritis; Arthritis, Juvenile
• Other Study ID Numbers: WA29231; 2013-005212-98 (EudraCT Number)