Pharmacovigilance in Juvenile Idiopathic Arthritis Patients Treated With Biologic Agents and/or Methotrexate

NCT ID: NCT01399281

Last Updated: 2018-03-05

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 9000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2011-12-31
• Study Completion Date: 2021-12-31

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.

Conditions

Juvenile Idiopathic Arthritis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Biologics alone or methotrexate alone

This group mainly refer to children with polyarticular course JIA treated with methotrexate ± biologics,

No interventions assigned to this group

Biologics and MTX

JIA treated with a combination of biologic and MTX (including any other add on therapy e.g. cyclosporine, leflunomide etc). This group mainly refer to children with polyarticular course JIA treated with MTX ± biologics

No interventions assigned to this group

NSAIDs and/or steroid injections alone

This group refers to children with mostly oligoarticular persistent course who are usually NOT treated with second line agents and have a more benign course.

No interventions assigned to this group

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

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Members of the PRINTO network (PRINTO at www.printo.it)

UNKNOWN

Sponsor Role: collaborator

Istituto Giannina Gaslini

OTHER

Sponsor Role: lead

Responsible Party

Dr. Nicola Ruperto

MD, MPH

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Nicola Ruperto, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Istituto G. Gaslini/ PRINTO

PRINTO Member

Role: STUDY_DIRECTOR

All PRINTO members who will receive ethics committee approval

Locations

IRCCS G. Gaslini

Genoa, , Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Nicola Ruperto, MD, MPH

Role: CONTACT

nicolaruperto@gaslini.org

+39-010-382854

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Related Links

http://www.printo.it

Official website of the Paediatric Rheumatology International Trials Organisation (PRINTO)

Other Identifiers

EU 260353

Identifier Type: -

Identifier Source: org_study_id