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

March 2, 2018 updated by: Dr. Nicola Ruperto, Istituto Giannina Gaslini

Pharmacovigilance in Juvenile Idiopathic Arthritis Patients (Pharmachild) Treated With Biologic Agents and/or Methotrexate. A PRINTO/PRES Registry

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.

Study Overview

Status

Unknown

Conditions

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.

Study Type

Observational

Enrollment (Anticipated)

9000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Genoa, Italy, 16147
        • Recruiting
        • IRCCS G. Gaslini
        • Principal Investigator:
          • Nicola Ruperto, MD, MPH

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Male and female JIA patients selected among the PRINTO centres as part of the routine standard of care

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Biologics alone or methotrexate alone
This group mainly refer to children with polyarticular course JIA treated with methotrexate ± biologics,
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
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety in patients with JIA on biologics/MTX from 3 to 10 years from drug initiation
Time Frame: 3-10 years
To compare the long term incidence rates of emergent moderate, severe adverse events (AEs) and serious A (SAE) observed in paediatric subjects with JIA. This will include but not be limited to, malignancies, opportunistic infections, autoimmune events, cardiovascular events, central nervous system involvement (e.g. optic neuritis, demyelinating disease), infertility, gastrointestinal bleeding, macrophage activation syndrome (MAS).
3-10 years
Long-term efficacy in patients with JIA on biologics/MTX from 3 to 10 years from drug initiation
Time Frame: 3-10 years
To assess the long-term efficacy (magnitude of response, prevention or slowing of joint erosion and damage, and treatment adherence) of biologic agents and MTX for the treatment of children and adolescents with different categories of JIA.
3-10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Predictors of safety from 3 to 10 years from drug initiation
Time Frame: 3-10 years
Identify predictors of safety (clinical or experimental, magnitude of response, remission)
3-10 years
Risk factor from 3 to 10 years from drug initiation
Time Frame: 3-10 years
Assess potential risk factors (e.g. concomitant medications or diseases, medical history etc), which may modify the safety profile of biologic agents and MTX;
3-10 years
Number of children in treatment with biologics from 3 to 10 years from drug initiation
Time Frame: 3-10 years
To assess the number of children in which a biologic agent is added to the treatment
3-10 years
Wrist joint erosion from 3 to 10 years from drug initiation
Time Frame: 3-10 years
Evaluate the progression of wrist joint erosion over time and abnormal growth/maturation in JIA subjects presenting a wrist involvement
3-10 years
Assess the reasons for stopping drug treatment from 3 to 10 years from drug initiation
Time Frame: 3-10 years
• To assess the reasons for stopping drug treatment
3-10 years
Efficacy from 3 to 10 years from drug initiation
Time Frame: 3-10 years
• To evaluate efficacy in terms, in the different JIA categories, of individual JIA core set variables, and the ACR Paediatric 30, 50, 70, 90, 100 criteria for improvement, and the achievement of clinical remission on and off medication as well as the occurrence of disease flare during biologic agents and MTX treatment course and after drug discontinuation, and the attainment of a status of minimal disease actvity (MDA)
3-10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Istituto Giannina Gaslini

Collaborators

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

Investigators

  • Principal Investigator: Nicola Ruperto, MD, MPH, Istituto G. Gaslini/ PRINTO
  • Study Director: PRINTO Member, All PRINTO members who will receive ethics committee approval

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2011

Primary Completion (Anticipated)

December 1, 2021

Study Completion (Anticipated)

December 1, 2021

Study Registration Dates

First Submitted

July 19, 2011

First Submitted That Met QC Criteria

July 20, 2011

First Posted (Estimate)

July 21, 2011

Study Record Updates

Last Update Posted (Actual)

March 5, 2018

Last Update Submitted That Met QC Criteria

March 2, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EU 260353

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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