Arthritis Juvenile Idiopathic Clinical Trial
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
| Verified date | April 2024 |
| Source | Pfizer |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| 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. |
| 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 |
| Lead Sponsor | Collaborator |
|---|---|
| Pfizer |
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,
| 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. |