Clinical trial NCT02165345: Extension Study Evaluating the Safety and Efficacy of Subcutaneous Tocilizumab (RoActemra/Actemra) Administration in Systemic and Polyarticular-Course Juvenile Idiopathic Arthritis
Official title: Long-Term Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Tocilizumab in Patients With Polyarticular-Course and Systemic Juvenile Idiopathic Arthritis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Juvenile Idiopathic Arthritis
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2014-06-17
Enrollment: 82
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Juvenile Arthritis Disease Activity Score (JADAS-71)
timeFrame: Baseline up to 3 years
measure: Percentage of Participants With Adverse Events (AEs), Serious AEs (AEs) and AEs of Special Interest
timeFrame: Baseline up to 5 years
Secondary outcomes:
measure: Childhood Health Assessment Questionnaire (CHAQ) Score
timeFrame: Baseline up to 3 years
measure: Percentage of Participants With Protocol Defined Inactive Disease/Clinical Remission
timeFrame: Baseline up to 3 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital Research Institute
state: Arkansas
city: Chicago
country: United States
facility: University of Chicago Hospital
state: Illinois
city: Hackensack
country: United States
facility: Hackensack University Medical Center; Pediatric Rheumatology
state: New Jersey
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children'S Hospital Medical Center; Division of Rheumatology
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Fndn
state: Ohio
city: Tulsa
country: United States
facility: Healthcare Research Consultants
state: Oklahoma
city: Salt Lake City
country: United States
facility: University of Utah; Immunology/Rheumatology/Allergy
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Buenos Aires
country: Argentina
facility: Hospital Gral de Niños Pedro Elizalde
city: Cordoba
country: Argentina
facility: Hospital de Ninos de la Santisima Trinidad; Hematología
city: Westmead
country: Australia
facility: Westmead Hospital; Paediatric Rheumatology
state: New South Wales
city: Parkville
country: Australia
facility: Royal Children'S Hospital; Paediatric Rheumatology
state: Victoria
city: Sao Paulo
country: Brazil
facility: Universidade Federal de Sao Paulo - UNIFES
state: SP
city: Sao Paulo
country: Brazil
facility: Hospital das Clinicas - FMUSP
state: SP
city: Calgary
country: Canada
facility: Alberta Children'S Hospital
state: Alberta
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Le Kremlin Bicêtre
country: France
facility: CH de Bicêtre; Pediatrie Generale
city: Berlin
country: Germany
facility: Charité Campus; Virchow Klinikum Berlin
city: Freiburg
country: Germany
facility: Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Pädiatrische Infektio- u. Rheumatologie
city: Sankt Augustin
country: Germany
facility: Asklepios Klinik; Zentrum für Allgemeine Pädiatrie und Neonatologie
city: Roma
country: Italy
facility: Irccs Ospedale Pediatrico Bambin Gesu - Dip. Di Medicina
state: Lazio
city: Mexico
country: Mexico
facility: Hospital Infantil de México "Federico Gomez"; Rheumatology
city: Monterrey
country: Mexico
facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
city: Moscow
country: Russian Federation
facility: SI Sceintific children health center RAMS
city: Esplugas DE Llobregat
country: Spain
facility: Hospital Sant Joan De Deu; Servicio de Reumatologia Pediatrica
state: Barcelona
city: Madrid
country: Spain
facility: Hospital Ramon y Cajal ; Servicio de Reumatologia
city: Madrid
country: Spain
facility: Hospital de La Paz; Unidad de Reumatologia Pediatrica
city: Valencia
country: Spain
facility: Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica
city: Bristol
country: United Kingdom
facility: Bristol Royal Hospital for Children; Rheumatology Department
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's NHS Foundation Trust
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: