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

• ClinicalTrials.gov Identifier: NCT03000439
• Recruitment Status: Completed
• First Posted: December 22, 2016
• Last Update Posted: April 19, 2024
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Tracking Information

• First Submitted Date: November 9, 2016
• First Posted Date: December 22, 2016
• Last Update Posted Date: April 19, 2024
• Actual Study Start Date: May 10, 2018
• Actual Primary Completion Date: March 27, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 19, 2016)

Time to flare [ Time Frame: Up to 82 weeks after randomization ]

Time to sJIA disease flare in the double-blind phase

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 19, 2016)

• Occurrence of disease flare in double-blind phase [ Time Frame: Up to 82 weeks after randomization ]
  disease flare frequency by visit in the double-blind phase.
• Achievement of corticosteroid tapering at the end of the open-label phase [ Time Frame: 12 to 40 weeks ]
  Rate of successful corticosteroid tapering.
• 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 [ Time Frame: 12 to 40 weeks ]
• Adapted sJIA ACR 30/50/70/90/100 response at every visit from Day 7 onward in the open label and double blind phase. [ Time Frame: Up to 82 weeks after randomization ]
• Fever (Temp >38 Degrees Celsius) attributed to sJIA at Day 3, Day 7 and Day 14 of the open label phase. [ Time Frame: Day 3, Day 7, Day 14 ]
• CRP ≤ 10 mg/L at every visit of the open label phase. [ Time Frame: 12 to 40 weeks ]
• "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. [ Time Frame: Up to 82 weeks after randomization. ]
• Time to first Adapted JIA ACR 30 response in Part 1 of the open label phase. [ Time Frame: 12 to 40 weeks ]
• 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. [ Time Frame: Up to 82 weeks after randomization. ]
• Change from baseline in each JIA ACR core variable at every visit from Day 7 onward in the open label and double blind phase. [ Time Frame: Up to 82 weeks after randomization. ]
• 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. [ Time Frame: Up to 82 weeks after randomization. ]
• Change from baseline in Child Health Assessment Questionnaire (CHAQ) at every visit from Day 7 onward in the open label and double blind phase. [ Time Frame: Up to 82 weeks after randomization. ]
• 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. [ Time Frame: Up to 82 weeks after randomization. ]
• 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. [ Time Frame: Up to 82 weeks after randomization. ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Arthritis Juvenile Idiopathic

Intervention

• Drug: In open-label phase: treatment with tofacitinib
  Treatment with investigational drug
  Other Names:

  • CP-690,550
  • Xeljanz
• Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio
  Treatment with investigational drug or placebo
  Other Names:

  • CP-690,550
  • Xeljanz

Study Arms

• Experimental: Tofacitinib 5 mg BID
  oral, twice daily, tablet or solution.
  Interventions:

  • Drug: In open-label phase: treatment with tofacitinib
  • Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio
• Placebo Comparator: Placebo
  Intervention: Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 19, 2016): 100
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: March 27, 2024
• Actual Primary Completion Date: March 27, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 17 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Argentina, Belgium, Brazil, Canada, China, Costa Rica, Germany, Hungary, India, Israel, Italy, Mexico, Poland, Russian Federation, Slovakia, South Africa, Spain, Turkey, Ukraine, United Kingdom, United States
• Removed Location Countries: Chile, Netherlands

Administrative Information

• NCT Number: NCT03000439
• Other Study ID Numbers: A3921165; 2017-002018-29 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
• URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

• Current Responsible Party: Pfizer
• Original Responsible Party: Same as current
• Current Study Sponsor: Pfizer
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

• PRS Account: Pfizer
• Verification Date: April 2024