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

NCT03000439 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: A39211652017-002018-29
• Study Type: interventional
• Target: 100
• Locations: 19 countries
• Sites: 52

Brief 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.

Conditions

Arthritis Juvenile Idiopathic

Keywords

systemic Juvenile Idiopathic Arthritis; Xeljanz; Tofacitinib; sJIA; CP-690 550

Outcome Measures

Primary Outcomes (1)

• Time to Systemic Juvenile Idiopathic Arthritis (sJIA) Disease Flare: Double-Blind Phase

  Time to the disease flare=the number of days from randomization to flare in the DB phase and calculated as date of disease flare minus (-) date of randomization plus (+) 1. sJIA Flare=as at least one of the following criteria: Recurrence of fever \>38 degree Celsius (C)/100.4 degree Fahrenheit (F) on 2 or more consecutive days) was considered due to SJIA activity. Worsening of 30 percent (%) or more in three or more of the six variables included: Number of joints with active arthritis and limited range of motion, disease activity, parent child evaluation of overall well-being, functional ability, childhood health assessment questionnaire ( CHAQ disability index), erythrocyte sedimentation rate (ESR) millimeter/ hour (mm/hr), of the JIA core set with no more than one variable of the JIA core set improving by 30% compared to the day of randomization into the withdrawal phase. 95% Confidence Interval (CI) based on Brookmeyer and Crowley Method.

  From randomization up to 248 weeks

Secondary Outcomes (64)

• Probability of Occurrence of sJIA Disease Flare at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52: Double-Blind Phase

  DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52

• Percentage of Participants Who Achieved Successful Corticosteroid Tapering: at the End of Open-label Phase Part 2

  From end of OL Part 1 to up to 24 weeks in OL Part 2

• Percentage of Participants Who Achieved Corticosteroid Dose of <= 0.2 mg/kg/Day or 10 mg/Day: at the End of Open-label Phase Part 2

  From end of OL Part 1 to up to 24 weeks in OL Part 2

• Percentage of Participants With Adapted JIA ACR 30/50/70/90/100 Response at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase

  DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52

• Percentage of Participants With Adapted JIA American College of Rheumatology (ACR) 30/50/70/90/100 Response at Part 1 Day 7, Weeks 2, 4, 8, 12, and 16: Open-Label Phase Part 1

  Part 1 Day 7, Part 1 Weeks 2, 4, 8, 12, and 16

Study Arms (2)

Tofacitinib 5 mg BID

EXPERIMENTAL

oral, twice daily, tablet or solution.

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

Placebo

PLACEBO COMPARATOR

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

Interventions

In open-label phase: treatment with tofacitinib DRUG

Treatment with investigational drug

Also known as: CP-690,550, Xeljanz

Tofacitinib 5 mg BID

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

Treatment with investigational drug or placebo

Also known as: CP-690,550, Xeljanz

Placebo; Tofacitinib 5 mg BID

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (52)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Cohen Children's Medical Center of New York

Lake Success, New York, 11042, United States

Location

Cohen Children's Medical Center of New York

New Hyde Park, New York, 11040, United States

Location

Instituto CAICI SRL

Rosario, Santa Fe Province, S2000PBJ, Argentina

Location

Centro Medico Privado de Reumatologia

San Miguel de Tucumán, Tucumán Province, T4000AXL, Argentina

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

Faculdade de Medicina da UNESP

Botucatu, São Paulo, 18618-686, Brazil

Location

UPECLIN Unidade de Pesquisa Clinica da Faculdade de Medicina da UNESP

Botucatu, São Paulo, 18618-970, Brazil

Location

SPDM - Associacao Paulista para o Desenvolvimento da Medicina

São Paulo, 04024-002, Brazil

Location

SPDM - Associacao Paulista para o Desenvolvimento da Medicina

São Paulo, 04037-002, Brazil

Location

Instituto da Crianca do Hospital das Clinicas da FMUSP

São Paulo, 05409-011, Brazil

Location

Alberta Children's Hospital/University of Calgary

Calgary, Alberta, T3B 6A8, Canada

Location

Research Institute of McGill University Health Center, Glen site

Montreal, Quebec, H4A 3J1, Canada

Location

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, 401122, China

Location

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong, 510623, China

Location

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

Wuhan, Hubei, 430030, China

Location

The Second Xiangya Hospital of Central South University

Changsha, Hunan, 410011, China

Location

Children's Hospital of Soochow University

Suzhou, Jiangsu, 215003, China

Location

Xi'an Children's Hospital

Xi'an, Shaanxi, 710003, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610073, China

Location

The Children's Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310057, China

Location

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

Beijing, 100045, China

Location

Hospital Metropolitano

San José, 10103, Costa Rica

Location

Universitaetsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

Location

St. Josef-Stift Sendenhorst

Sendenhorst, 48324, Germany

Location

Semmelweis Egyetem

Budapest, 1094, Hungary

Location

Nirmal Hospital Pvt Ltd

Surat, Gujarat, 395002, India

Location

Sir Ganga Ram Hospital

Rajinder Nagar, NEW Delhi, 110060, India

Location

Institute of Child Health

Kolkata, West Bengal, 700017, India

Location

Institute of Post Graduate Medical Education and Research & SSKM Hospital

Kolkata, West Bengal, 700020, India

Location

Rambam Health Care Campus

Haifa, 3109601, Israel

Location

Meir Medical Center - Pediatric Clinic

Kfar Saba, 4428164, Israel

Location

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

Genova, Genoa, 16147, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Milan, 20122, Italy

Location

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

Guadalajara, Jalisco, 44650, Mexico

Location

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, 64460, Mexico

Location

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

San Luis Potosí City, 78213, Mexico

Location

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

Warsaw, 02-637, Poland

Location

Clinic of FSBEI HE BSMU MoH RF

Ufa, Bashkortostan Republic, 450083, Russia

Location

Federal State Budgetary Scientific Institution "Scientific and Research Rheumatology

Moscow, 115522, Russia

Location

Enhancing Care Foundation

Durban, KwaZulu-Natal, 4302, South Africa

Location

Panorama Medical Centre

Cape Town, Western Cape, 7500, South Africa

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Universitario y Politecnico La Fe

Valencia, 46026, Spain

Location

Hacettepe University Medical Faculty

Ankara, 06100, Turkey (Türkiye)

Location

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

Kadikoy / Istanbul, 34722, Turkey (Türkiye)

Location

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

Dnipro, 49006, Ukraine

Location

Municipal non-Profit Enterprise

Ivano-Frankivsk, 76014, Ukraine

Location

CNE of Lviv Regional Council "Western Ukrainian Specialized Pediatric

Lviv, 79035, Ukraine

Location

Vinnytsia Regional Children's Clinical Hospital

Vinnytsia, 21000, Ukraine

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Arthritis, Juvenile

Interventions

tofacitinib

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

8007181021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2016
• First Posted: December 22, 2016
• Study Start: May 10, 2018
• Primary Completion: March 27, 2024
• Study Completion: March 27, 2024
• Last Updated: June 3, 2025
• Results First Posted: June 3, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share

Locations

BE (1); IN (4); IT (2); ME (3); PL (1); CA (2); US (5); DE (2); CO (1); TU (2); CN (9); IL (2); UA (4); BR (5); HU (1); ZA (2); RU (2); ES (2); AR (2)