Brief Summary

Evaluate efficacy, safety and tolerability of tofacitinib in pediatric JIA patients.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network

Enrollment

225

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Jun 2016

Typical duration for phase_3

Geographic Reach

14 countries

103 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.9 years study duration

First Submitted

Initial submission to the registry

August 14, 2015

Completed

3 months until next milestone

First Posted

Study publicly available on registry

October 30, 2015

Completed

7 months until next milestone

Study Start

First participant enrolled

June 10, 2016

Completed

2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 16, 2019

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 16, 2019

Completed

9 months until next milestone

Results Posted

Study results publicly available

February 21, 2020

Completed

Last Updated

April 13, 2020

Status Verified

March 1, 2020

Enrollment Period

2.9 years

First QC Date

August 14, 2015

Results QC Date

February 6, 2020

Last Update Submit

March 30, 2020

Conditions

Juvenile Idiopathic Arthritis

Keywords

ArthritisPediatricLong-termJIApolyarticularCP-690,550tofacitinibXeljanz

Outcome Measures

Primary Outcomes (1)

Double Blind Phase: Percentage of Participants With Disease Flare According to Pediatric Rheumatology Collaborative Study Group/Pediatric Rheumatology International Trials Organization (PRCSG/PRINTO) Disease Flare Criteria at Week 44
According to PRCSG/PRINTO, disease flare defined as worsening of \>=30 percent(%) in \>=3 of 6 variables of JIA core set, with no more than 1 variable improving by \>=30%. Six core variables: 1) Number of joints with active arthritis (joint with swelling/in absence of swelling, limited range of motion accompanied by pain/tenderness), 2)Number of joints with limited range of motion, 3) Physician global evaluation of disease activity (assessed on a Visual Analog Scale\[VAS\] of 0\[no activity\] to 10\[maximum activity\]), 4) Parent/legal guardian/participant global assessment of overall well-being (assessed on VAS of 0\[very well\] to 10\[very poor\], 5) Childhood Health Assessment Questionnaire- Disability Index (CHAQ-DI): 30 questions in 8 domains, each question answered on scale of 0=without difficulty to 3=unable to do; scores of each domain were averaged to derive total CHAQ-DI score,which ranges from 0 (minimum dysfunction) to 3 (most severe dysfunction);6) Erythrocyte Sedimentation Rate(ESR).
Week 44

Secondary Outcomes (71)

Double Blind Phase: Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 50 Response at Week 44
Week 44
Double Blind Phase: Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 30 Response at Week 44
Week 44
Double Blind Phase: Percentage of Participants With Juvenile Idiopathic Arthritis (JIA) American College of Rheumatology (ACR) 70 Response at Week 44
Week 44
Double Blind Phase: JIA ACR Core Variable- Change From Double-Blind Baseline in Childhood Health Assessment Questionnaire- Disability Index (CHAQ-DI) Total Score at Week 44
Baseline, Week 44
Open-Label Phase: Percentage of Participants With Disease Flare According to Pediatric Rheumatology Collaborative Study Group/Pediatric Rheumatology International Trials Organization (PRCSG/PRINTO) Disease Flare Criteria at Week 2, 4, 8, 12 and 18
Weeks 2, 4, 8, 12 and 18
+66 more secondary outcomes

Study Arms (2)

CP-690,550

EXPERIMENTAL

Treatment arm: Tofacitinib tablets or solution, according to subjects' body weights

Drug: CP-690,550 (tofacitinib)

Placebo

PLACEBO COMPARATOR

Control arm: matching placebo tablets or solution for tofacitinib

Other: placebo

Interventions

CP-690,550 (tofacitinib)DRUG

During the open label run in phase, all subjects will receive active tofacitinib oral tablets or oral solution twice daily (BID) orally, at a dosage based on the subject's body weight as specified below. During the double blind, placebo controlled phase, subjects will receive either active tofacitinib oral tablets/oral solution or matching placebo oral tablets/oral solution, twice daily (BID), at a dosage specified below. Body Weight (Dosage in tablet \[BID\] or solution \[BID\]): 5\<7kg (2mg or 2mL); 7\<10kg(2.5mg or 2. mL); 10 \<15kg (3mg or 3mL); 15\<25kg (3.5mg or 3.5mL); 25\<40kg (4mg or 4mL); 40kg (5 mg or 5 ml). Oral solution (1 mg/mL) is used for subjects \<40 kg. Oral tablets (5 mg) are used for subjects \>=40 kg.

Also known as: Matching placebo to tofacitinib (same tablet or solution as the active arm).

CP-690,550

placeboOTHER

matching placebo tablet or solution for tofacitinib

Also known as: Placebo for tofacitinib

Placebo

Eligibility Criteria

Age2 Years - 17 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Male or female aged 2 to \<18 years.
Must meet International League Against Rheumatism (ILAR) JIA diagnostic criteria for one of the following categories with active disease for at least 6 weeks:
Extended oligoarthritis;
Polyarthritis (RF+);
Polyarthritis (RF-);
Systemic JIA with active arthritis but without active systemic features in the prior 6 months and at the time of enrollment;
Psoriatic arthritis;
Enthesitis related arthritis. Subjects with polyarticular course JIA (ie, extended oligoarthritis, polyarthritis RF+, polyarthritis RF , systemic JIA with active arthritis but without active systemic features) must have a minimum of 5 active joints (an active joint is defined as a joint with swelling or, in the absence of swelling, limited range of motion accompanied by either pain on motion or tenderness) at screening and baseline to be eligible for study entry.
Subjects with psoriatic or enthesitis related arthritis must have a minimum of 3 active joints (an active joint is defined as a joint with swelling or, in the absence of swelling, limited range of motion accompanied by either pain on motion or tenderness) at screening and baseline to be eligible for study entry.
Treatment with stable doses of a Non Steroidal Anti inflammatory Drug (NSAID) and/or a stable dose of an oral glucocorticoid, and/or a stable dose of methotrexate is permitted.
For subjects receiving an oral glucocorticoid: Glucocorticoids may be administered at a maximum dose of 0.2 mg of prednisone equivalent per kilogram per day or 10 mg per day for ≥ 2 weeks before baseline, whichever is lower.
For subjects receiving methotrexate (MTX) treatment: MTX may be administered either orally or parenterally at doses not to exceed 25 mg/wk or 20 mg/m2/week (whichever is lower); participants must have taken MTX for 3 months and be at a stable dose for at least 6 weeks before baseline. Subjects taking MTX must be taking folic acid or folinic acid in accordance with local standards.
For subjects with psoriatic arthritis, the following topical treatments for psoriasis are allowed: non medicated emollients for use over the whole body; topical steroids including hydrocortisone and hydrocortisone acetate ≤1% for the palms, soles, face, and intertriginous areas only; tar, salicylic acid preparations, and shampoos free of corticosteroids are permitted only for the scalp
Inadequate response or intolerance to at least one Disease Modifying Anti Rheumatic Drug (DMARD), which may include MTX or biologic agents; in the case of ERA and psoriatic arthritis, inadequate response to Non Steroidal Anti Inflammatory Drugs (NSAIDs).
No evidence or history of untreated or inadequately treated active or latent tuberculosis (TB) infection as evidenced by the following:
+8 more criteria

You may not qualify if:

Subjects with any of the following characteristics/conditions will not be included in the study:
Previous JIA treatment with tofacitinib.
Systemic JIA (sJIA) with active systemic features (including subjects with characteristic sJIA fever and rash or serositis within 6 months of enrollment).
Persistent oligoarthritis.
Undifferentiated JIA.
Infections:
Chronic infections;
Any infection requiring hospitalization, parenteral antimicrobial therapy or judged to be opportunistic by the investigator within the 6 months prior to the first dose of study drug;
Any treated infections within 2 weeks of Baseline visit;
A subject know to be infected with Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C;
History of infected joint prosthesis with prosthesis still in situ.
History of recurrent (more than one episode) herpes zoster or disseminated (at least one episode) herpes zoster, or disseminated (at least one episode) herpes simplex.
Active uveitis (according to SUN criteria) within 3 months of enrollment.
Blood dyscrasias, including:
Hemoglobin \<10 g/dL or Hematocrit \<33%;
+23 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pfizerlead

Study Sites (103)

Arkansas Children's Hospital - Attention: Jill Hernandez

Little Rock, Arkansas, 72202, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Loma Linda University Children's Hospital

Loma Linda, California, 92354, United States

Location

Loma Linda University Clinical Trial Center

Loma Linda, California, 92354, United States

Location

Loma Linda University Eye Institute

Loma Linda, California, 92354, United States

Location

Loma Linda University General Pediatric Clinic - Meridian

Loma Linda, California, 92354, United States

Location

Pediatric Specialty Team Centers of LLU Children's Hospital

Loma Linda, California, 92408, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Pediatric Specialty Team Centers of LU Children's Hospital

San Bernardino, California, 92408, United States

Location

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

Location

Rady Children's Hospital Center for Pediatric Clinical Research

San Diego, California, 92123, United States

Location

Rady Children's Hospital Education and Office Building

San Diego, California, 92123, United States

Location

Rady Children's Hospital Research Pharmacy

San Diego, California, 92123, United States

Location

Rady Children's Hospital Rheumatology Clinic

San Diego, California, 92123, United States

Location

Connecticut Children's Medical Center -Pharmacy

Hartford, Connecticut, 06106, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

IDS Pharmacy Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Children's Healthcare of Atlanta-Pediatric Research Center

Atlanta, Georgia, 30322, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Children's Specialty Services

Atlanta, Georgia, 30322, United States

Location

Augusta University Health Pharmacy

Augusta, Georgia, 30912, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

The University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

IU Health Investigational Drug Services

Indianapolis, Indiana, 46202, United States

Location

Riley Hospital for Children at IU Health

Indianapolis, Indiana, 46202, United States

Location

Tufts Medical Center Floating Hospital for Children

Boston, Massachusetts, 02111, United States

Location

Journey Clinic: Center for Children with Cancer and Blood Diseases, Uni. of Minnesota Medical Centre

Minneapolis, Minnesota, 55454, United States

Location

Pediatric Specialty Care Explorer Clinic, University of Minnesota, Medical Center

Minneapolis, Minnesota, 55454, United States

Location

University of Minnesota Medical Center, Fairview IDS Pharmacy

Minneapolis, Minnesota, 55455, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Cohen Children's Medical Center of NY

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

Columbia University Medical Center - Herbert Irving Pavilion

New York, New York, 10032, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Pharmacy Department

The Bronx, New York, 10467, United States

Location

Children's Specialty Center

Charlotte, North Carolina, 28203, United States

Location

Carolinas HealthCare System IDS Pharmacy

Charlotte, North Carolina, 28204, United States

Location

Pediatric Research

Charlotte, North Carolina, 28207, United States

Location

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Legacy Emanuel Medical Center - Inpatient Pharmacy

Portland, Oregon, 97227, United States

Location

Randall Children's Hospital at Legacy Emanuel

Portland, Oregon, 97227, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States

Location

Specially for Children, Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States

Location

Texas Children's Hospital - Clinical Care center