A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA Clinical Trial Results and Information

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

ClinicalTrials.gov processed this data on July 9, 2024. Link to the current ClinicalTrials.gov record.

Recruitment Status

COMPLETED (See Contacts and Locations)
Verified July 2024 by Pfizer

Sponsor

Pfizer

Information Provided by (Responsible Party)

Pfizer

Clinicaltrials.gov Identifier

NCT03000439
Other Study ID Numbers: A3921165
First Submitted: November 9, 2016
First Posted: December 22, 2016
Last Update Posted: July 11, 2024
Last Verified: July 2024
History of Changes

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Study Description

Not Provided

Condition or Disease	Intervention/Treatment
Arthritis Juvenile Idiopathic	Drug: In open-label phase: treatment with tofacitinib Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Study Design

Study Type	Interventional
Actual Enrollment	100 participants
Design Allocation	Randomized
Interventional Model	Parallel Assignment
Masking	Quadruple
Primary Purpose	Treatment
Official Title	EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF TOFACITINIB FOR TREATMENT OF SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (SJIA) WITH ACTIVE SYSTEMIC FEATURES IN CHILDREN AND ADOLESCENT SUBJECTS
Study Start Date	May 10, 2018
Actual Primary Completion Date	March 27, 2024
Actual Study Completion Date	March 27, 2024

Groups and Cohorts

Group/ Cohort	Intervention/ Treatment
Tofacitinib 5 mg BID oral, twice daily, tablet or solution.	Drug: In open-label phase: treatment with tofacitinib Treatment with investigational drug Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio
Placebo	Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Outcome Measures

Primary Outcome Measures

Time to flare [Up to 82 weeks after randomization]
Time to sJIA disease flare in the double-blind phase

Secondary Outcome Measures

Occurrence of disease flare in double-blind phase [Up to 82 weeks after randomization]
disease flare frequency by visit in the double-blind phase.
Achievement of corticosteroid tapering at the end of the open-label phase [12 to 40 weeks]
Rate of successful corticosteroid tapering.
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]
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]
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]
CRP ≤ 10 mg/L at every visit of the open label phase. [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. [Up to 82 weeks after randomization.]
Time to first Adapted JIA ACR 30 response in Part 1 of the open label phase. [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. [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. [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. [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. [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. [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. [Up to 82 weeks after randomization.]

Eligibility Criteria

Ages Eligible for Study	2 Years to 17 Years (Child)
Sexes Eligible for Study	All
Accepts Healthy Volunteers	No
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.

Contacts and Locations

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