Clinical trial NCT01500551: Long-Term Safety Study Of Tofacitinib In Patients With Juvenile Idiopathic Arthritis
Official title: A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Juvenile Idiopathic Arthritis
Keywords: Arthritis
Pediatric
Long-term
JIA
CP-690,550
tofacitinib
Xeljanz
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2011-12-28
Enrollment: 302
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies.

Detailed description: This is a Phase 2/3, long term, open-label, follow-up study. Subjects will have previously participated in qualifying/index JIA studies of tofacitinib. Those who have already completed such participation and enroll outside the 14 day window following completion of the End of Study (EOS) Visit of the qualifying/index study will participate in a screening Visit to determine eligibility. A Baseline Visit will then occur within 28 days after the Screening Visit. For subjects who are completing participation in a qualifying study of tofacitinib and enrolling on the same day of the EOS Visit of the qualifying/index study, the EOS Visit of the qualifying/index study can be combined with the Screening and Baseline Visits for this study. The subjects who enroll within the 14 day window following completion of the EOS Visit of the qualifying/index study will participate in a combined Screening and Baseline Visit for this study. After the Baseline Visit, visits will occur at 1 month (1 month=30 days) and 3 months, then every 3 months thereafter as long as the subject remains in the study.

Approximately 340 participants are projected to enroll into this open label extension study after completing a qualifying/index study in the JIA program.

For subjects who entered this study from the A3921103 and A3921104 qualifying/index studies, their participation in this study ends after the first marketing approval of tofacitinib for the treatment of polyarticular course Juvenile Idiopathic Arthritis (pJIA) in any country. This study will end once the last subject, and all other subjects, who entered from index study A3921165 have completed approximately 1 year in this study, or after the first marketing approval of tofacitinib for the treatment of systemic JIA, whichever comes first.

The total duration of an individual subject's participation may vary depending upon when they enter the trial.

Eligibility criteria: Inclusion Criteria:

* Pediatric subjects with JIA aged from 2 to less than 18 years who met entry criteria for the qualifying/index study and in the opinion of the investigator have sufficient evidence of JIA disease activity to warrant use of tofacitinib as a DMARD. Subjects turning 18 years of age during participation in the qualifying/index study or subsequently will be eligible for participation in this study.
* The subject has discontinued disallowed concomitant medications for the required time prior to the first dose of study drug, as defined in Appendix 1, and is taking only those concomitant medications in doses and frequency allowed by the protocol.
* Fertile male subjects and female subjects of childbearing potential who are, in the opinion of the investigator, sexually active and at risk for pregnancy with their partner(s) must be using a highly effective method of contraception as outlined in this protocol throughout the study and for at least 28 days after the last dose of study medication.
* Subjects must have previously completed participation in a qualifying study of tofacitinib for the treatment of JIA. Subjects who have required earlier discontinuation of treatment in a qualifying study for reasons other than tofacitinib related serious adverse events may be eligible.

Exclusion Criteria:

* persistent oligoarthritis, and undifferentiated JIA.
* Infections:

  1. Chronic infections.
  2. Any infection requiring hospitalization, parenteral antimicrobial therapy or judged to be opportunistic by the investigator within the 3 months prior to the first dose of study drug.
  3. Any treated infections within 2 weeks of baseline visit.
  4. A subject known to be infected with human immunodeficiency virus (HIV), hepatitis B or hepatitis C virus.
  5. History of infected joint prosthesis with prosthesis still in situ.
* History of recurrent (more than one episode) herpes zoster or disseminated (a single episode) herpes zoster or disseminated (a single episode) herpes simplex.

Primary outcomes:
measure: Standard laboratory safety data and adverse event (AE) reports. Body weight, height and Tanner Stages will collected to assess growth and physical development.
timeFrame: up to 8 years
Secondary outcomes:
measure: Physician global evaluation of disease activity at each visit.
timeFrame: up to 8 years
measure: Number of joints with active arthritis at each visit.
timeFrame: up to 8 years
measure: Number of joints with limitation of motion at each visit.
timeFrame: up to 8 years
measure: Index of inflammation (C-reactive protein [CRP] and Erythrocyte Sedimentation Rate [ESR]) at each visit.
timeFrame: up to 8 years
measure: Parent's Assessment of Physical Function (Childhood Health Assessment Questionnaire [CHAQ]Disability Index)at each visit.
timeFrame: up to 8 years
measure: Parent's Assessment of Child's Arthritis Pain (Childhood Health Assessment Questionnaire [CHAQ] Discomfort Index, Visual Analog Scale [VAS])at each visit.
timeFrame: up to 8 years
measure: Parent's Global Assessment of Overall Wellbeing (Childhood Health Assessment Questionnaire [CHAQ] subsection, Visual Analog Scale [VAS])at each visit.
timeFrame: up to 8 years
measure: JIA American College of Rheumatology (ACR) response and occurrence of JIA ACR disease flare at each visit.
timeFrame: up to 8 years
measure: JIA ACR Clinical Inactive Disease status and Clinical Remission on Medication at each visit.
timeFrame: up to 8 years
measure: Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS) 27- CRP and JADAS 27-ESR, and occurrence of JADAS minimum disease activity and inactive disease at each visit.
timeFrame: up to 8 years
measure: In subjects with Enthesitis Related Arthritis (ERA): Change from baseline in the Tender Entheseal Assessment, Modified Schober's Test, Overall Back Pain and Nocturnal Back Pain responses at various visits.
timeFrame: up to 8 years
measure: In subjects with psoriatic arthritis (PsA): Change from baseline in body surface area (BSA) affected by psoriasis and Physician's Global Assessment (PGA) of psoriasis) at various visits.
timeFrame: up to 8 years
measure: In subjects with sJIA: "Absence of Fever", defined as absence of fever due to sJIA in the week preceding the assessment at each visit.
timeFrame: up to 8 years
measure: Eligibility of tapering defined per protocol for corticosteroids
timeFrame: up to 8 years
measure: Eligibility of tapering defined per protocol for methotrexate
timeFrame: up to 8 years
measure: Eligibility of tapering defined per protocol for leflunomide
timeFrame: Up to 8 years
measure: Eligibility of tapering defined per protocol for tofacitinib
timeFrame: Up to 8 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Loma Linda
country: United States
facility: Loma Linda University Children'S Hospital
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Clinical Trials Center
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Eye Institute
state: California
city: Loma Linda
country: United States
facility: Loma Linda University General Pediatric Clinic - Meridian
state: California
city: Loma Linda
country: United States
facility: Pediatric Speciality Team Centers of LLU Children's Hospital (Rheumatology)
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: San Bernardino
country: United States
facility: Pediatric Speciality Team Centers of LLU Children's Hospital (Rheumatology)
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital Center for Pediatric Clinical Research
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital Rheumatology Clinic
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego- Education and Office Building
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: San Diego
country: United States
facility: Rady Children's Research Pharmacy
state: California
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Washington
country: United States
facility: IDS Pharmacy
state: District of Columbia
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Center for Advanced Pediatrics
state: Georgia
city: Augusta
country: United States
facility: AU Medical Center
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: The University of Chicago Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children at IU Health
state: Indiana
city: Boston
country: United States
facility: Tufts Medical Center - Floating Hospital for Children
state: Massachusetts
city: Minneapolis
country: United States
facility: Explorer Clinic, University of Minnesota Children's Hospital
state: Minnesota
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Lake Success
country: United States
facility: Cohen Children's Medical Center of New York
state: New York
city: New York
country: United States
facility: Columbia University Medical Center-Herbert Irving Pavillion
state: New York
city: Charlotte
country: United States
facility: Pediatric Research
state: North Carolina
city: Charlotte
country: United States
facility: Levine Children's Specialty Center
state: North Carolina
city: Charlotte
country: United States
facility: Atrium Health- Investigational Drug Services
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Portland
country: United States
facility: Randall Children's Hospital at Legacy Emanuel
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Austin
country: United States
facility: Dell Children's Medical Group, Dell Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital- Clinical Care Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital- Clinical Research Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital- Investigational Pharmacy
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital- Main Hospital
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital/Baylor College of Medicine- Feigin Center
state: Texas
city: Salt Lake City
country: United States
facility: Intermountain - Primary Children's Hospital
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Rosario
country: Argentina
facility: Instituto CAICI SRL
state: Santa FE
city: San Miguel de Tucuman
country: Argentina
facility: Centro Medico Privado de Reumatologia
state: Tucuman
city: Caba
country: Argentina
facility: Hospital Britanico de Buenos Aires
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
state: New South Wales
city: Parkville
country: Australia
facility: The Royal Children's Hospital
state: Victoria
city: Gent
country: Belgium
facility: UZ Gent
city: Leuven
country: Belgium
facility: UZ Leuven - Gasthuisberg
city: Salvador
country: Brazil
facility: SER - Serviços Especializados em Reumatologia
state: Bahia
city: Juiz de Fora
country: Brazil
facility: CMIP - Centro Mineiro de Pesquisa Ltda
state: Minas Gerais
city: Curitiba
country: Brazil
facility: Hospital Pequeno Príncipe Clinical Research Office
state: Parana
city: Botucatu
country: Brazil
facility: Faculdade de Medicina da UNESP
state: SAO Paulo
city: Rio de Janeiro
country: Brazil
facility: Instituto de Puericultura e Pediatria Martagao Gesteira (IPPMG)
city: Sao Paulo
country: Brazil
facility: SPDM - Associacao Paulista para o Desenvolvimento da Medicina
city: Sao Paulo
country: Brazil
facility: Instituto da Crianca do Hospital das Clinicas da FMUSP
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: Montreal
country: Canada
facility: McGill University Health Center, Glen Site
state: Quebec
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Hangzhou
country: China
facility: The Children's Hospital Zhejiang University School of Medicine
state: Zhejiang
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University/Rheumatology Department
city: Bad Bramstedt
country: Germany
facility: PRI - Pediatric Rheumatology Research Institute GmbH
city: Hamburg
country: Germany
facility: Hamburger Zentrum fur Kinder und Jugendrheumatologie
city: Sankt Augustin
country: Germany
facility: Asklepios Klinik Sankt Augustin GmbH
city: Sendenhorst
country: Germany
facility: St. Josef-Stift Sendenhorst
city: Surat
country: India
facility: Nirmal Hospital Pvt Ltd.
state: Gujarat
city: Kolkata
country: India
facility: Institute of Child Health
state: WEST Bengal
city: Kolkata
country: India
facility: Institute of Post Graduate Medical Education and Research & SSKM Hospital
state: WEST Bengal
city: New Delhi
country: India
facility: Sir Ganga Ram Hospital
city: Haifa
country: Israel
facility: Rambam Health Care Campus
city: Kfar Saba
country: Israel
facility: Meir Medical Center - Pediatric Clinic
city: Ramat Gan
country: Israel
facility: Chaim Sheba M.C Tel hashomer
city: Guadalajara
country: Mexico
facility: Clínica de Investigacion en Reumatologia y Obesidad, S.C.
state: Jalisco
city: Monterrey
country: Mexico
facility: Hospital Universitario "Dr. Jose Eleuterio Gonzalez"
state: Nuevo LEON
city: San Luis De Potosí
country: Mexico
facility: Centro de Alta Especialidad de Reumatología e Investigación del Potosí, S.C.
city: San Luis Potosi
country: Mexico
facility: Hospital Central "Dr. Ignacio Morones Prieto"
city: San Luis Potosi
country: Mexico
facility: Unidad de Investigaciones Reumatologicas A.C.
city: Bydgoszcz
country: Poland
facility: Wojewodzki Szpital Dzieciecy im. J. Brudzinskiego
city: Krakow
country: Poland
facility: Wojewodzki Specjalistyczny Szpital Dzieciecy im. Sw. Ludwika w Krakowie
city: Lodz
country: Poland
facility: Klinika Kardiologii i Reumatologii Dzieciecej
city: Warszawa
country: Poland
facility: Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher
city: Ufa
country: Russian Federation
facility: FSBEI HE BSMU MoH RF
state: Republic OF Bashkortostan
city: Ufa
country: Russian Federation
facility: Clinic of FSBEI HE BSMU MoH RF
state: Republic OF Bashkortostan
city: Moscow
country: Russian Federation
facility: Federal State Budgetary Scientific Institution
city: Moscow
country: Russian Federation
facility: FSAEI HE I.M. Sechenov First MSMU of Minzdrav of Russia (Sechenovskiy University)
city: Moscow
country: Russian Federation
facility: FSAEI HE I.M. Sechenov First MSMU of Minzdrav of Russia (Sechenovskiy University),
city: Moscow
country: Russian Federation
facility: FSAI "NMRCCH" of MOH Russia
city: Saint-Petersburg
country: Russian Federation
facility: FSBEI HE "St. Petersburg State Pediatric Medical University" of the Ministry of Healthcare
city: Tolyatti
country: Russian Federation
facility: State Budgetary Healthcare Institution of Samara Region "Tolyatti City Clinical Hospital #5"
city: Piestany
country: Slovakia
facility: Narodny ustav reumatickych chorob
city: Panorama
country: South Africa
facility: Panorama Medical Centre
state: Cape Town
city: Durban
country: South Africa
facility: Durban International Clinical Research Site, Enhancing Care Foundation
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico La Fe
city: Ankara
country: Turkey
facility: Hacettepe University Medical Faculty
city: Istanbul
country: Turkey
facility: Umraniye Training and Research Hospital
city: Kadikoy / Istanbul
country: Turkey
facility: Istanbul Goztepe Prof. Dr. Suleyman Yalcin Sehir Hastanesi Cocuk Romatoloji Bolumu
city: Dnipro
country: Ukraine
facility: Communal Institution "Dnipropetrovsk Specialized Clinical Medical Center of Mother and Child n.a.
city: Ivano-Frankivsk
country: Ukraine
facility: Ivano-Frankivsk Regional Children's Clinical Hospital
city: Vinnytsia
country: Ukraine
facility: Communal Non-profit Enterprise
city: Birmingham
country: United Kingdom
facility: Birmingham Woman's and Children's NHS Foundation Trust
state: WEST Midlands
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: