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

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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Arthritis Juvenile Idiopathic Drug: In open-label phase: treatment with tofacitinib Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : May 10, 2018
Actual Primary Completion Date : March 27, 2024
Actual Study Completion Date : March 27, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Arthritis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Tofacitinib 5 mg BID
oral, twice daily, tablet or solution.
 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
Placebo Comparator: Placebo 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


Outcome Measures
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Primary Outcome Measures :
  1. Time to flare [ Time Frame: Up to 82 weeks after randomization ]
    Time to sJIA disease flare in the double-blind phase


Secondary Outcome Measures :
  1. 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.

  2. Achievement of corticosteroid tapering at the end of the open-label phase [ Time Frame: 12 to 40 weeks ]
    Rate of successful corticosteroid tapering.

  3. 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 ]
  4. 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 ]
  5. 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 ]
  6. CRP ≤ 10 mg/L at every visit of the open label phase. [ Time Frame: 12 to 40 weeks ]
  7. "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. ]
  8. Time to first Adapted JIA ACR 30 response in Part 1 of the open label phase. [ Time Frame: 12 to 40 weeks ]
  9. 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. ]
  10. 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. ]
  11. 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. ]
  12. 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. ]
  13. 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. ]
  14. 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. ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03000439


Locations
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Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03000439    
Other Study ID Numbers: A3921165
2017-002018-29 ( EudraCT Number )
First Posted: December 22, 2016    Key Record Dates
Last Update Posted: April 19, 2024
Last Verified: April 2024
Individual Participant Data (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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pfizer:
systemic Juvenile Idiopathic Arthritis
Xeljanz
Tofacitinib
sJIA
CP-690 550
Additional relevant MeSH terms:
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Arthritis
Joint Diseases
Musculoskeletal Diseases
Tofacitinib
 Janus Kinase Inhibitors
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action