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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03000439
Other study ID # A3921165
Secondary ID 2017-002018-29
Status Completed
Phase Phase 3
First received
Last updated
Start date May 10, 2018
Est. completion date March 27, 2024

Study information

Verified date April 2024
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Completed
Enrollment 100
Est. completion date March 27, 2024
Est. primary completion date March 27, 2024
Accepts healthy volunteers No
Gender All
Age group 2 Years to 17 Years
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
In open-label phase: treatment with tofacitinib
Treatment with investigational drug
In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio
Treatment with investigational drug or placebo

Locations

Country Name City State
Argentina Instituto CAICI SRL Rosario Santa FE
Argentina Centro Medico Privado de Reumatologia San Miguel de Tucuman Tucuman
Belgium Hopital Universitaire des Enfants Reine Fabiola (HUDERF) Brussels
Belgium Universitair Ziekenhuis Gent Gent
Brazil Faculdade de Medicina da UNESP Botucatu SÃO Paulo
Brazil UPECLIN Unidade de Pesquisa Clinica da Faculdade de Medicina da UNESP Botucatu SAO Paulo
Brazil Hospital de Clinicas da UNICAMP Campinas SAO Paulo
Brazil Hospital de Clínicas de Porto Alegre Porto Alegre RIO Grande DO SUL
Brazil Instituto da Crianca do Hospital das Clinicas da FMUSP Sao Paulo
Brazil SPDM - Associacao Paulista para o Desenvolvimento da Medicina Sao Paulo
Brazil SPDM - Associacao Paulista para o Desenvolvimento da Medicina Sao Paulo
Canada Alberta Children's Hospital/University of Calgary Calgary Alberta
Canada McGill University Health Center, Glen Site Montreal Quebec
Canada Research Institute of McGill University Health Center, Glen site Montreal Quebec
Canada CHU de Québec - Université Laval, Site CHUL Quebec City Quebec
Canada The Hospital for Sick Children Toronto Ontario
China Beijing Children's Hospital, Capital Medical University/Rheumatology Department Beijing
China The Second Xiangya Hospital of Central South University Changsha Hunan
China Chengdu Women's and Children's Central Hospital Chengdu Sichuan
China Children's Hospital of Chongqing Medical University Chongqing Chongqing
China Guangzhou Women and Children's Medical Center Guangzhou Guangdong
China The Children's Hospital Zhejiang University School of Medicine Hangzhou Zhejiang
China Children's Hospital of Fudan University Shanghai
China Children's Hospital of Soochow University Suzhou Jiangsu
China Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology Wuhan Hubei
China Xi'an Children's Hospital Xi'an Shaanxi
Costa Rica Hospital Metropolitano San Jose
Germany Charite - Universitaetsmedizin Berlin, Campus Virchow Berlin
Germany HELIOS Kliniken Berlin Buch GmbH Berlin
Germany HELIOS Klinikum Berlin Buch GmbH Berlin
Germany Gesundheit Nord gGmbH Bremen
Germany Universitaetsklinikum Carl Gustav Carus Dresden Dresden
Germany Universitaetsklinikum Erlangen Erlangen Bayern
Germany Deutsches Zentrum für Kinder- und Jugendrheumatologie Garmisch-partenkirchen
Germany Asklepios Klinik Sankt Augustin GmbH, Zentrum für Allgemeine Pädiatrie und Neonatologie Sankt Augustin
Germany St. Josef-Stift Sendenhorst Sendenhorst
Hungary Semmelweis Egyetem Budapest
India Institute of Child Health Kolkata WEST Bengal
India Institute of Post Graduate Medical Education and Research & SSKM Hospital Kolkata WEST Bengal
India Sir Ganga Ram Hospital Rajinder Nagar NEW Delhi
India Nirmal Hospital Pvt Ltd Surat Gujarat
Israel Rambam Health Care Campus Haifa
Israel Meir Medical Center - Pediatric Clinic Kfar Saba
Israel Meir Medical Center- Pharmacy Kfar Saba
Italy Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico Genova Genoa
Italy Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano Milan
Mexico Clínica de Investigacion en Reumatologia y Obesidad, S.C. Guadalajara Jalisco
Mexico Hospital Universitario "Dr. Jose Eleuterio Gonzalez" Monterrey Nuevo LEON
Mexico Centro de Alta Especialidad en Reumatologia e Investigacion del Potosi, S.C. San Luis Potosi
Poland Wojewodzki Specjalistyczny Szpital Dzieciecy im. Sw. Ludwika w Krakowie Krakow
Poland SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi Lodz
Poland Centrum Pediatrii im. Jana Pawla II w Sosnowcu Sp. z o.o. Sosnowiec
Poland WIP Warsaw IBD Point Profesor Kierkus Warsaw
Poland Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher Warszawa
Russian Federation Federal State Budgetary Scientific Institution "Scientific and Research Rheumatology Moscow
Russian Federation Clinic of FSBEI HE BSMU MoH RF Ufa Republic OF Bashkortostan
Slovakia Narodny ustav detskych chorob, Detska klinika LF UK a NUDCH Bratislava
Slovakia Detska Fakultna nemocnica Kosice Kosice
Slovakia Narodny ustav reumatickych chorob Piestany
South Africa Panorama Medical Centre Cape Town Western CAPE
South Africa Enhancing Care Foundation Durban Kwazulu-natal
South Africa Enhancing Care Foundation Durban Kwazulu-natal
South Africa Emmed Research Pretoria Gauteng
Spain Hospital Universitario La Paz Madrid
Spain Hospital Universitario Ramon y Cajal Madrid
Spain Hospital Universitario y Politecnico La Fe Valencia
Turkey Hacettepe University Medical Faculty Ankara
Turkey Istanbul University Cerrahpasa - Cerrahpasa Tip Fakultesi Clinical Research Center Fatih / Istanbul
Turkey Umraniye Training and Research Hospital Istanbul
Turkey Istanbul Goztepe Prof. Dr. Suleyman Yalcin City Hospital Department of Pediatric Rheumatology Kadikoy / Istanbul
Turkey Erciyes Universitesi Tip Fakultesi Hastanesi Kayseri
Ukraine Communal Institution "Dnipropetrovsk Specialized Clinical Medical Center of Mother and Child n.a. Dnipro
Ukraine Municipal non-Profit Enterprise Ivano-Frankivsk
Ukraine CNE of Lviv Regional Council "Western Ukrainian Specialized Pediatric Lviv
Ukraine Vinnytsia Regional Children's Clinical Hospital Vinnytsia
United Kingdom NHS Greater Glasgow and Clyde, Royal Hospital for Children Glasgow
United States AU Medical Center Augusta Georgia
United States Augusta University Augusta Georgia
United States UNC Children's Hospital Chapel Hill North Carolina
United States UNC Clinical & Translational Research Center Chapel Hill North Carolina
United States Ann & Robert H Lurie Children's Hospital of Chicago Chicago Illinois
United States Lurie Rheumatology Offices Chicago Illinois
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States Cincinnati Children's Hospital Medical Center Investigational Drug Service Cincinnati Ohio
United States Hackensack University Medical Center Hackensack New Jersey
United States Cohen Children's Medical Center of New York Lake Success New York
United States Children's Hospital Los Angeles Los Angeles California
United States PPD Sample Management Department (PK Laboratory) Middleton Wisconsin
United States Cohen Children's Medical Center of New York New Hyde Park New York
United States Hospital for Special Surgery New York New York
United States University of Oklahoma Health Sciences Center Oklahoma City Oklahoma
United States AdventHealth Orlando Florida
United States AdventHealth Pediatric Outpatient Procedures and Sedation Orlando Florida
United States AHMG Pediatric Rheumatology and Immunology Orlando Florida
United States Phoenix Children's Hospital Phoenix Arizona
United States Randall Children's Hospital at Legacy Emanuel Portland Oregon
United States UNC Children's Raleigh Raleigh North Carolina
United States All Children's Hospital Specialty Physicians Saint Petersburg Florida
United States Children's National Medical Center Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Argentina,  Belgium,  Brazil,  Canada,  China,  Costa Rica,  Germany,  Hungary,  India,  Israel,  Italy,  Mexico,  Poland,  Russian Federation,  Slovakia,  South Africa,  Spain,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Time to flare Time to sJIA disease flare in the double-blind phase Up to 82 weeks after randomization
Secondary Occurrence of disease flare in double-blind phase disease flare frequency by visit in the double-blind phase. Up to 82 weeks after randomization
Secondary Achievement of corticosteroid tapering at the end of the open-label phase Rate of successful corticosteroid tapering. 12 to 40 weeks
Secondary 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 12 to 40 weeks
Secondary Adapted sJIA ACR 30/50/70/90/100 response at every visit from Day 7 onward in the open label and double blind phase. Up to 82 weeks after randomization
Secondary Fever (Temp >38 Degrees Celsius) attributed to sJIA at Day 3, Day 7 and Day 14 of the open label phase. Day 3, Day 7, Day 14
Secondary CRP = 10 mg/L at every visit of the open label phase. 12 to 40 weeks
Secondary "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. Up to 82 weeks after randomization.
Secondary Time to first Adapted JIA ACR 30 response in Part 1 of the open label phase. 12 to 40 weeks
Secondary 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. Up to 82 weeks after randomization.
Secondary Change from baseline in each JIA ACR core variable at every visit from Day 7 onward in the open label and double blind phase. Up to 82 weeks after randomization.
Secondary 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. Up to 82 weeks after randomization.
Secondary Change from baseline in Child Health Assessment Questionnaire (CHAQ) at every visit from Day 7 onward in the open label and double blind phase. Up to 82 weeks after randomization.
Secondary 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. Up to 82 weeks after randomization.
Secondary 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. Up to 82 weeks after randomization.