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

NCT ID: NCT03000439

Last Updated: 2025-06-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-05-10

Study Completion Date

2024-03-27

Brief Summary

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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.

Detailed Description

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Conditions

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Arthritis Juvenile Idiopathic

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Tofacitinib 5 mg BID

oral, twice daily, tablet or solution.

Group Type EXPERIMENTAL

In open-label phase: treatment with tofacitinib

Intervention Type DRUG

Treatment with investigational drug

In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Intervention Type DRUG

Treatment with investigational drug or placebo

Placebo

Group Type PLACEBO_COMPARATOR

In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Intervention Type DRUG

Treatment with investigational drug or placebo

Interventions

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In open-label phase: treatment with tofacitinib

Treatment with investigational drug

Intervention Type DRUG

In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Treatment with investigational drug or placebo

Intervention Type DRUG

Other Intervention Names

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CP-690,550 Xeljanz CP-690,550 Xeljanz

Eligibility Criteria

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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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Cohen Children's Medical Center of New York

Lake Success, New York, United States

Site Status

Cohen Children's Medical Center of New York

New Hyde Park, New York, United States

Site Status

Instituto CAICI SRL

Rosario, Santa Fe Province, Argentina

Site Status

Centro Medico Privado de Reumatologia

San Miguel de Tucumán, Tucumán Province, Argentina

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

Faculdade de Medicina da UNESP

Botucatu, São Paulo, Brazil

Site Status

UPECLIN Unidade de Pesquisa Clinica da Faculdade de Medicina da UNESP

Botucatu, São Paulo, Brazil

Site Status

SPDM - Associacao Paulista para o Desenvolvimento da Medicina

São Paulo, , Brazil

Site Status

SPDM - Associacao Paulista para o Desenvolvimento da Medicina

São Paulo, , Brazil

Site Status

Instituto da Crianca do Hospital das Clinicas da FMUSP

São Paulo, , Brazil

Site Status

Alberta Children's Hospital/University of Calgary

Calgary, Alberta, Canada

Site Status

Research Institute of McGill University Health Center, Glen site

Montreal, Quebec, Canada

Site Status

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

Site Status

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong, China

Site Status

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, China

Site Status

The Second Xiangya Hospital of Central South University

Changsha, Hunan, China

Site Status

Children's Hospital of Soochow University

Suzhou, Jiangsu, China

Site Status

Xi'an Children's Hospital

Xi'an, Shaanxi, China

Site Status

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, China

Site Status

The Children's Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Site Status

Beijing Children's Hospital, Capital Medical University/Rheumatology Department

Beijing, , China

Site Status

Hospital Metropolitano

San José, , Costa Rica

Site Status

Universitaetsklinikum Erlangen

Erlangen, Bavaria, Germany

Site Status

St. Josef-Stift Sendenhorst

Sendenhorst, , Germany

Site Status

Semmelweis Egyetem

Budapest, , Hungary

Site Status

Nirmal Hospital Pvt Ltd

Surat, Gujarat, India

Site Status

Sir Ganga Ram Hospital

Rajinder Nagar, NEW Delhi, India

Site Status

Institute of Child Health

Kolkata, West Bengal, India

Site Status

Institute of Post Graduate Medical Education and Research & SSKM Hospital

Kolkata, West Bengal, India

Site Status

Rambam Health Care Campus

Haifa, , Israel

Site Status

Meir Medical Center - Pediatric Clinic

Kfar Saba, , Israel

Site Status

Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico

Genova, Genoa, Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Milan, Italy

Site Status

Clínica de Investigacion en Reumatologia y Obesidad, S.C.

Guadalajara, Jalisco, Mexico

Site Status

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, Mexico

Site Status

Centro de Alta Especialidad en Reumatologia e Investigacion del Potosi, S.C.

San Luis Potosí City, , Mexico

Site Status

Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher

Warsaw, , Poland

Site Status

Clinic of FSBEI HE BSMU MoH RF

Ufa, Bashkortostan Republic, Russia

Site Status

Federal State Budgetary Scientific Institution "Scientific and Research Rheumatology

Moscow, , Russia

Site Status

Enhancing Care Foundation

Durban, KwaZulu-Natal, South Africa

Site Status

Panorama Medical Centre

Cape Town, Western Cape, South Africa

Site Status

Hospital Universitario La Paz

Madrid, , Spain

Site Status

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Site Status

Hacettepe University Medical Faculty

Ankara, , Turkey (Türkiye)

Site Status

Istanbul Goztepe Prof. Dr. Suleyman Yalcin City Hospital Department of Pediatric Rheumatology

Kadikoy / Istanbul, , Turkey (Türkiye)

Site Status

Communal Institution "Dnipropetrovsk Specialized Clinical Medical Center of Mother and Child n.a.

Dnipro, , Ukraine

Site Status

Municipal non-Profit Enterprise

Ivano-Frankivsk, , Ukraine

Site Status

CNE of Lviv Regional Council "Western Ukrainian Specialized Pediatric

Lviv, , Ukraine

Site Status

Vinnytsia Regional Children's Clinical Hospital

Vinnytsia, , Ukraine

Site Status

Countries

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United States Argentina Belgium Brazil Canada China Costa Rica Germany Hungary India Israel Italy Mexico Poland Russia South Africa Spain Turkey (Türkiye) Ukraine

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=A3921165

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Other Identifiers

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2017-002018-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

A3921165

Identifier Type: -

Identifier Source: org_study_id

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