Clinical trial NCT01399281: Pharmacovigilance in Juvenile Idiopathic Arthritis Patients Treated With Biologic Agents and/or Methotrexate
Official title: Pharmacovigilance in Juvenile Idiopathic Arthritis Patients (Pharmachild) Treated With Biologic Agents and/or Methotrexate. A PRINTO/PRES Registry
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Juvenile Idiopathic Arthritis
Keywords: juvenile idiopathic arthritis, biologics, methotrexate
Lead sponsor: Istituto Giannina Gaslini
Sponsor class: OTHER
Collaborators: name: Members of the PRINTO network (PRINTO at www.printo.it)
First posted: 2011-07-21
Enrollment: 9000
Sex: ALL
Minimum age (years): 6
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Male and female JIA patients selected among the PRINTO centres as part of the routine standard of care

Brief summary: BACKGROUND: Juvenile idiopathic arthritis (JIA) is the most common chronic paediatric rheumatic disease (PRD) and an important cause of short and long-term disability. Although none of the available drugs for JIA has a curative potential, prognosis has greatly improved as a result of substantial progress in disease management. The therapeutic treatment of children with JIA encompasses the use of NSAIDs and intra-articular steroid injections. In those patients not responding to NSAIDs, methotrexate (MTX) has become the disease modifying anti-rheumatic drug (DMARD) of first choice worldwide. For children not responding to MTX, biologic agents recently have become treatment options.

PATIENTS AND METHODS: 3-10 year observation study related to children with JIA undergoing treatment with MTX or biologic agents with the following objectives:

1. To create a long-term observational registry of a large population of prevalent and incident cases.
2. Use the accumulating data in the registry to conduct (i) a pharmacovigilance/safety study (primary endpoint) and (ii) estimate effectiveness (frequency and magnitude of response, disease activity over time inhibition or slowing of joint erosions and other radiological evidence of disease progression,), and (iii) estimate adherence to the various treatment regimens. Data from the registry will be used to compare safety and effectiveness profiles amongst the patient cohorts.
3. To identify clinical and laboratory predictors of safety, response to therapy, including remission This project has retrospective (first 3 years) and prospective components (up to 10 years) and will be conducted by the participating centres of the more than 50 countries belonging to the Paediatric Rheumatology INternational Trials Organisation (PRINTO certified ISO 9001-2008, www.printo.it), or the Pediatric Rheumatology European Society (PRES at www.pres.org.uk). The main role of these organisations is to provide a scientific basis for current treatments of paediatric rheumatic diseases.

The overall hypothesis to be tested is:

• Biologic agents ± MTX agents are able to maintain an acceptable safety profile in the long term in children with different JIA categories while achieving clinical remission and prevent/stop joint erosion development over time.

The overall aims are to establish the long term safety of biologic agents and MTX, and their relative effectiveness in children with JIA who need treatment with second line agents.

Detailed description: Study Design This is a 3-10 year, international, multicentre, observational, safety and efficacy (response, joint erosion, damage, and treatment adherence) study aimed at collecting prospective safety, tolerability, efficacy, and treatment adherence information on JIA subjects exposed to any biologic agents and MTX, according to local standard of practice.

This is a non-interventional study, where the medicinal products are prescribed as per the investigator's decision. The assignment of the subject to a particular therapeutic strategy is not decided in advance by the study protocol, but falls within current practice and the prescription of the medicine is clearly separated from the decision to include the subject in the study. No additional diagnostic or monitoring procedures shall be applied to the subjects and epidemiological methods will be used for the analysis of collected data.

Duration and treatment will be as per investigator's decision. The nature and frequency of subjects' visits to the investigator's site will be determined only by the investigator, according to his/her judgment on the basis of the clinical evolution of the subject.

The duration of the study is expected to be at least 3 years from initiation of the first site and may be continued beyond if adequate funding is be available.

Eligibility criteria: Inclusion Criteria:

* Signed written informed consent by subjects and /or parent or legally acceptable representative;
* JIA (any ILAR category);
* Subjects receiving biologic agents ± MTX, MTX alone, or NSAIDs and/or steroid injections only as per physician discretion.

Exclusion Criteria:

* Contraindications to biologic agents and/or MTX treatment

Primary outcomes:
measure: Safety in patients with JIA on biologics/MTX from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Long-term efficacy in patients with JIA on biologics/MTX from 3 to 10 years from drug initiation
timeFrame: 3-10 years
Secondary outcomes:
measure: Predictors of safety from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Risk factor from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Number of children in treatment with biologics from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Wrist joint erosion from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Assess the reasons for stopping drug treatment from 3 to 10 years from drug initiation
timeFrame: 3-10 years
measure: Efficacy from 3 to 10 years from drug initiation
timeFrame: 3-10 years
Other outcomes:
Locations:
city: Genoa
country: Italy
facility: IRCCS G. Gaslini
Study officials:
affiliation: Istituto G. Gaslini/ PRINTO
name: Nicola Ruperto, MD, MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: All PRINTO members who will receive ethics committee approval
name: PRINTO Member
role: STUDY_DIRECTOR
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