Start Time Optimization of Biologics in Polyarticular JIA
Condition: Polyarticular Juvenile Rheumatoid Arthritis; Arthritis, Juvenile Idiopathic
MeSH terms: Arthritis; Arthritis, Juvenile
Study type: Observational

Summary: 
      STOP-JIA is a PCORI funded prospective observational study which compared the clinical
      effectiveness and impact on patient reported outcomes of 3 Childhood Arthritis & Rheumatology
      Research Alliance (CARRA) consensus derived treatment strategies (CTPs) in new-onset
      polyarticular JIA (pJIA) patients to answer the critical question of when is the best time to
      begin biologic medications to achieve the optimal clinical and patient reported outcomes.
      Because the CARRA Registry will be used for data collection, all patients will be enrolled in
      the CARRA Registry. The standard of care treatments are chosen by the treating physician and
      patient/caregiver and are not randomized.
    

Detailed description: 
      STOP-JIA is a prospective, observational study comparing the clinical effectiveness and
      impact on patient reported outcomes of 3 different treatment strategies (CTPs) in new onset
      pJIA patients to answer the critical question of when to start biologic medications. All
      participants will be enrolled in the CARRA Registry and started on one of the CTPs, which
      will be decided by the treating physician and patient/caregiver. Subjects will be enrolled at
      one of 60 participating CARRA sites across the US and Canada. Total anticipated enrollment
      was 400 and this was completed in 9/19.

      Specific Aim 1:

      To compare the clinical effectiveness of different strategies (CTPs) for using biologic
      medications in achieving clinically inactive disease (CID) at 12 months in new-onset pJIA.
      Three common strategies that differ in the timing of biologic medication introduction will be
      compared: 1) Step-Up: disease modifying anti-rheumatic drug (DMARD) monotherapy stepping up
      by addition of a biologic medication if needed; 2) Early Combination: DMARD plus biologic
      medication at treatment onset; and 3) Biologic First: biologic medication monotherapy at
      treatment onset.

      Hypothesis 1: A significantly higher proportion of children started on a biologic medication
      at onset (CTP 2 or 3) will achieve CID after 12 months of therapy compared to standard
      therapy (CTP 1).

      Specific Aim 2:

      To compare patient and caregiver reported outcomes between the different strategies.

      Hypothesis 2: There will be statistically significant differences in patient/caregiver
      reported outcomes (PROs) between treatment strategies that can inform future patients and
      providers in selecting optimal treatments.

      The CARRA Registry will be housed at CARRA's clinical and data coordinating center, Duke
      Clinical Research Institute (DCRI). The CARRA Registry Protocol documents that the CARRA
      Registry fulfills all PCOR standards for registries. STOP-JIA will utilize data collection,
      storage, and management processes, systems requirements, and security processes already
      established for the CARRA Registry at DCRI.

      STOP-JIA used Web-based electronic CRFs (eCRFs) developed for the CARRA Registry that are
      already familiar to site personnel. The eCRF platform, RAVE, is 21CFR part11 compliant and
      meets regulatory requirements. Database and Web servers are secured by a firewall and through
      controlled physical access. eCRFs will be monitored for completeness, accuracy, and attention
      to detail throughout the study by DCRI data and site management teams using processes
      developed for the CARRA Registry and consistent with DCRI's internal SOPs. Use of electronic
      data capture will allow for immediate prompts/queries if entered values are out of expected
      ranges or there are incomplete data fields. The design of the data collection instrument will
      allow centers to record a planned assessment of a patient was missed and to enter any known
      reasons for the assessment being missed. DCRI will regularly provide reports detailing data
      completion metrics to the sites. Stakeholder engagement is also an important aspect of this
      study, and patients/caregivers as well as other stakeholders are serving as research partners
      and advisors in this study.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Children with new onset, untreated Poly JIA enrolled in the CARRA Registry.
      
Keywords: Joint Diseases; Juvenile Arthritis; treatment; biologic therapy; comparative effectiveness