A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA

March 19, 2024 updated by: Pfizer

EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF TOFACITINIB FOR TREATMENT OF SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (SJIA) WITH ACTIVE SYSTEMIC FEATURES IN CHILDREN AND ADOLESCENT SUBJECTS

A randomized withdrawal study in which responders to open-label treatment with tofacitinib will be randomized in a 1:1 ratio to tofacitinib or placebo in a double-blind phase. In the double-blind phase "time to sJIA flare" will be evaluated as primary endpoint and subjects will be discontinued once they experience sJIA flare. An interim analysis for efficacy and futility will be conducted when at least 20 flares have been observed. If either criterion is met, the study will be stopped. If neither criterion is met, the study will continue until the requisite number of flares are observed as determined by the number of flares included in the interim analysis and a statistical penalty for conducting the interim analysis.

Study Overview

Study Type

Interventional

Enrollment (Actual)

100

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

    • Santa FE
      • Rosario, Santa FE, Argentina, S2000PBJ
        • Instituto CAICI SRL
    • Tucuman
      • San Miguel de Tucuman, Tucuman, Argentina, T4000AXL
        • Centro Medico Privado de Reumatologia
      • Brussels, Belgium, 1020
        • Hopital Universitaire des Enfants Reine Fabiola (HUDERF)
      • Gent, Belgium, 9000
        • Universitair Ziekenhuis Gent
      • Sao Paulo, Brazil, 05409-011
        • Instituto da Crianca do Hospital das Clinicas da FMUSP
      • Sao Paulo, Brazil, 04024-002
        • SPDM - Associacao Paulista para o Desenvolvimento da Medicina
      • Sao Paulo, Brazil, 04037-002
        • SPDM - Associacao Paulista para o Desenvolvimento da Medicina
    • RIO Grande DO SUL
      • Porto Alegre, RIO Grande DO SUL, Brazil, 90035-903
        • Hospital de Clinicas de Porto Alegre
    • SAO Paulo
      • Botucatu, SAO Paulo, Brazil, 18618-970
        • UPECLIN Unidade de Pesquisa Clinica da Faculdade de Medicina da UNESP
      • Campinas, SAO Paulo, Brazil, 13083-888
        • Hospital de Clínicas da Unicamp
    • SÃO Paulo
      • Botucatu, SÃO Paulo, Brazil, 18618-686
        • Faculdade de Medicina da UNESP
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Alberta Children's Hospital/University of Calgary
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, H4A 3J1
        • McGill University Health Center, Glen Site
      • Montreal, Quebec, Canada, H4A 3J1
        • Research Institute of McGill University Health Center, Glen site
      • Quebec City, Quebec, Canada, G1V 4G2
        • CHU de Québec - Université Laval, Site CHUL
      • Beijing, China, 100045
        • Beijing Children's Hospital, Capital Medical University/Rheumatology Department
      • Shanghai, China, 201102
        • Children's Hospital of Fudan University
    • Chongqing
      • Chongqing, Chongqing, China, 401122
        • Children's Hospital of Chongqing Medical University
    • Guangdong
      • Guangzhou, Guangdong, China, 510623
        • Guangzhou Women and Children's Medical Center
    • Hubei
      • Wuhan, Hubei, China, 430030
        • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
    • Hunan
      • Changsha, Hunan, China, 410011
        • The Second Xiangya Hospital of Central South University
    • Jiangsu
      • Suzhou, Jiangsu, China, 215003
        • Children's Hospital of Soochow University
    • Shaanxi
      • Xi'an, Shaanxi, China, 710003
        • Xi'an Children's Hospital
    • Sichuan
      • Chengdu, Sichuan, China, 610073
        • Chengdu Women's and Children's central hospital
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310057
        • The Children's Hospital Zhejiang University School of Medicine
      • San Jose, Costa Rica, 10103
        • Hospital Metropolitano
      • Berlin, Germany, 13125
        • HELIOS Kliniken Berlin Buch GmbH
      • Berlin, Germany, 13125
        • HELIOS Klinikum Berlin Buch GmbH
      • Berlin, Germany, 13353
        • Charite - Universitaetsmedizin Berlin, Campus Virchow
      • Bremen, Germany, 28177
        • Gesundheit Nord gGmbH
      • Dresden, Germany, 01307
        • Universitaetsklinikum Carl Gustav Carus Dresden
      • Garmisch-partenkirchen, Germany, 82467
        • Deutsches Zentrum für Kinder- und Jugendrheumatologie
      • Sankt Augustin, Germany, 53757
        • Asklepios Klinik Sankt Augustin GmbH, Zentrum für Allgemeine Pädiatrie und Neonatologie
      • Sendenhorst, Germany, 48324
        • St. Josef-Stift Sendenhorst
    • Bayern
      • Erlangen, Bayern, Germany, 91054
        • Universitaetsklinikum Erlangen
      • Budapest, Hungary, 1094
        • Semmelweis Egyetem
    • Gujarat
      • Surat, Gujarat, India, 395002
        • Nirmal Hospital Pvt Ltd
    • NEW Delhi
      • Rajinder Nagar, NEW Delhi, India, 110060
        • Sir Ganga Ram Hospital
    • WEST Bengal
      • Kolkata, WEST Bengal, India, 700020
        • Institute of Post Graduate Medical Education and Research & SSKM Hospital
      • Kolkata, WEST Bengal, India, 700017
        • Institute of Child Health
      • Haifa, Israel, 3109601
        • Rambam Health Care Campus
      • Kfar Saba, Israel, 4428164
        • Meir Medical Center - Pediatric Clinic
      • Kfar Saba, Israel, 4428164
        • Meir Medical Center- Pharmacy
    • Genoa
      • Genova, Genoa, Italy, 16147
        • Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
    • Milan
      • Milano, Milan, Italy, 20122
        • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
      • San Luis Potosi, Mexico, 78213
        • Centro de Alta Especialidad en Reumatologia e Investigacion del Potosi, S.C.
    • Jalisco
      • Guadalajara, Jalisco, Mexico, 44650
        • Clínica de Investigacion en Reumatologia y Obesidad, S.C.
    • Nuevo LEON
      • Monterrey, Nuevo LEON, Mexico, 64460
        • Hospital Universitario "Dr. Jose Eleuterio Gonzalez"
      • Krakow, Poland, 31-503
        • Wojewódzki Specjalistyczny Szpital Dzieciecy im. Sw. Ludwika w Krakowie
      • Lodz, Poland, 91-738
        • SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi
      • Sosnowiec, Poland, 41-218
        • Centrum Pediatrii im. Jana Pawla II w Sosnowcu Sp. z o.o.
      • Warsaw, Poland, 00-728
        • WIP Warsaw IBD Point Profesor Kierkus
      • Warszawa, Poland, 02-637
        • Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher
      • Moscow, Russian Federation, 115522
        • Federal State Budgetary Scientific Institution "Scientific and Research Rheumatology
    • Republic OF Bashkortostan
      • Ufa, Republic OF Bashkortostan, Russian Federation, 450083
        • Clinic of FSBEI HE BSMU MoH RF
      • Bratislava, Slovakia, 833 40
        • Narodny ustav detskych chorob, Detska klinika LF UK a NUDCH
      • Kosice, Slovakia, 04011
        • Detska Fakultna nemocnica Kosice
      • Piestany, Slovakia, 921 12
        • Narodny ustav reumatickych chorob
    • Gauteng
      • Pretoria, Gauteng, South Africa, 0002
        • Emmed Research
    • Kwazulu-natal
      • Durban, Kwazulu-natal, South Africa, 4301
        • Enhancing Care Foundation
      • Durban, Kwazulu-natal, South Africa, 4302
        • Enhancing Care Foundation
    • Western CAPE
      • Cape Town, Western CAPE, South Africa, 7500
        • Panorama Medical Centre
      • Madrid, Spain, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz
      • Valencia, Spain, 46026
        • Hospital Universitario y Politecnico La Fe
      • Ankara, Turkey, 06100
        • Hacettepe University Medical Faculty
      • Fatih / Istanbul, Turkey, 34098
        • Istanbul University Cerrahpasa - Cerrahpasa Tip Fakultesi Clinical Research Center
      • Istanbul, Turkey, 34764
        • Umraniye Training and Research Hospital
      • Kadikoy / Istanbul, Turkey, 34722
        • Istanbul Goztepe Prof. Dr. Suleyman Yalcin City Hospital Department of Pediatric Rheumatology
      • Kayseri, Turkey, 38039
        • Erciyes Universitesi Tip Fakultesi Hastanesi
      • Dnipro, Ukraine, 49006
        • Communal Institution "Dnipropetrovsk Specialized Clinical Medical Center of Mother and Child n.a.
      • Ivano-Frankivsk, Ukraine, 76014
        • Municipal non-Profit Enterprise
      • Lviv, Ukraine, 79035
        • CNE of Lviv Regional Council "Western Ukrainian Specialized Pediatric
      • Vinnytsia, Ukraine, 21000
        • Vinnytsia Regional Children's Clinical Hospital
      • Glasgow, United Kingdom, G51 4TF
        • NHS Greater Glasgow and Clyde, Royal Hospital for Children
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Orlando, Florida, United States, 32803
        • AdventHealth
      • Orlando, Florida, United States, 32804
        • AdventHealth Pediatric Outpatient Procedures and Sedation
      • Orlando, Florida, United States, 32804
        • AHMG Pediatric Rheumatology and Immunology
      • Saint Petersburg, Florida, United States, 33701
        • All Children's Hospital Specialty Physicians
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University
      • Augusta, Georgia, United States, 30912
        • AU Medical Center
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H Lurie Children's Hospital of Chicago
      • Chicago, Illinois, United States, 60611
        • Lurie Rheumatology Offices
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • Lake Success, New York, United States, 11042
        • Cohen Children's Medical Center of New York
      • New Hyde Park, New York, United States, 11040
        • Cohen Children's Medical Center of New York
      • New York, New York, United States, 10021
        • Hospital for Special Surgery
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • UNC Children's Hospital
      • Chapel Hill, North Carolina, United States, 27599
        • UNC Clinical & Translational Research Center
      • Raleigh, North Carolina, United States, 27607
        • UNC Children's Raleigh
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center Investigational Drug Service
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma Health Sciences Center
    • Oregon
      • Portland, Oregon, United States, 97227
        • Randall Children's Hospital at Legacy Emanuel
    • Wisconsin
      • Middleton, Wisconsin, United States, 53562
        • PPD Sample Management Department (PK Laboratory)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • active sJIA disease according to ILAR criteria before screening and at baseline (Day 1);
  • Treatment with stable doses of methotrexate (MTX) ≤25 mg/week or ≤20 mg/m2/week, whichever is lower, is permitted;
  • Treatment with a stable dose of oral prednisone ≤1 mg/kg/day up to a maximum of 30 mg/day, or equivalent, for at least 1 week before the first study drug dose is permitted.

Exclusion Criteria:

  • Previous juvenile idiopathic arthritis (JIA) treatment with tofacitinib.
  • Current symptoms or findings of myocarditis, endocarditis or more than minimal pericardial effusion associated with systemic juvenile idiopathic arthritis (sJIA). Current symptoms or findings of more than minimal pleuritis with sJIA.
  • Current infection or serious infection within 3 months of study enrollment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Treatment with investigational drug or placebo
Other Names:
  • Xeljanz
  • CP-690,550
Experimental: Tofacitinib 5 mg BID
oral, twice daily, tablet or solution.
Treatment with investigational drug
Other Names:
  • Xeljanz
  • CP-690,550
Treatment with investigational drug or placebo
Other Names:
  • Xeljanz
  • CP-690,550

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to flare
Time Frame: Up to 82 weeks after randomization
Time to sJIA disease flare in the double-blind phase
Up to 82 weeks after randomization

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurrence of disease flare in double-blind phase
Time Frame: Up to 82 weeks after randomization
disease flare frequency by visit in the double-blind phase.
Up to 82 weeks after randomization
Achievement of corticosteroid tapering at the end of the open-label phase
Time Frame: 12 to 40 weeks
Rate of successful corticosteroid tapering.
12 to 40 weeks
Achievement of a corticosteroid dose of 0.2 mg/kg/day or 10 mg/day (whichever is lower) at the end of the open label treatment period
Time Frame: 12 to 40 weeks
12 to 40 weeks
Adapted sJIA ACR 30/50/70/90/100 response at every visit from Day 7 onward in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization
Up to 82 weeks after randomization
Fever (Temp >38 Degrees Celsius) attributed to sJIA at Day 3, Day 7 and Day 14 of the open label phase.
Time Frame: Day 3, Day 7, Day 14
Day 3, Day 7, Day 14
CRP ≤ 10 mg/L at every visit of the open label phase.
Time Frame: 12 to 40 weeks
12 to 40 weeks
"Absence of fever", defined as absence of fever due to sJIA in the week preceding the assessment at every visit from Day 7 onward in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Time to first Adapted JIA ACR 30 response in Part 1 of the open label phase.
Time Frame: 12 to 40 weeks
12 to 40 weeks
Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS 27) at every visit from Day 7 onward in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Change from baseline in each JIA ACR core variable at every visit from Day 7 onward in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Change from baseline in Child Health Questionnaire (CHQ) responses at the end of Part 1 and Part 2 of the open label phase, at randomization and every 3 months thereafter.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Change from baseline in Child Health Assessment Questionnaire (CHAQ) at every visit from Day 7 onward in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Occurrence of inactive disease status and minimal disease activity clinical remission at every visit from Day 7 onward (JADAS 27) in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.
Occurrence of inactive disease status and clinical remission at every visit from Day 7 onward (JIA ACR) in the open label and double blind phase.
Time Frame: Up to 82 weeks after randomization.
Up to 82 weeks after randomization.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Investigators

  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 10, 2018

Primary Completion (Estimated)

March 27, 2024

Study Completion (Estimated)

March 27, 2024

Study Registration Dates

First Submitted

November 9, 2016

First Submitted That Met QC Criteria

December 19, 2016

First Posted (Estimated)

December 22, 2016

Study Record Updates

Last Update Posted (Actual)

March 20, 2024

Last Update Submitted That Met QC Criteria

March 19, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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