Juvenile Idiopathic ArthritisNCT02524340
Patient Centered Adaptive Treatment Strategies Using Bayesian Causal Inference
An observational study of Juvenile Idiopathic Arthritis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The best treatment plan for Juvenile Idiopathic Arthritis (JIA) is often complicated. Patients and clinicians often don't know what is the best treatment strategy for a given patient at a given time. Read more about this study
- Who is it for?
- Ages 1 year to 19 years
- Conditions the study is for
- Juvenile Idiopathic Arthritis
- How many people
- 465 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2015to Jan 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children less or equal to 19 years of age that have been diagnosed with ployarticular course of juvenile idiopathic arthritis.
Participants should meet the following inclusion criteria:
- Age ≤19 years at baseline
- Diagnosed with ployarticular course of juvenile idiopathic arthritis (pcJIA) follow the operational case definition of pcJIA presented in Table 2 of the journal article published by Ringold et al. (2014).
- Meet new patient definition, i.e. diagnosed with pcJIA no more than 6 month at the first clinical encounter captured in the database
- Taken DMARDs no more than 9 months after diagnosed with pcJIA
Participants will be excluded if they meet the following exclusion criteria:
- Systematic JIA patients according to the ILAR code
- Patients with comorbidities of inflammatory bowel disease (IBD), celiac disease, and trisomy 21.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Patient Centered Adaptive Treatment Strategies for Juvenile Idiopathic Arthritis Using Bayesian Causal Inference
The best treatment plan for Juvenile Idiopathic Arthritis (JIA) is often complicated. Patients and clinicians often don't know what is the best treatment strategy for a given patient at a given time. The purpose of this study is to develop a method to analyze data in situations where the treatment and disease state change over time. The researchers will develop a web-based package that will use the methods developed in this study. The package will be easy to use and allow dissemination of the methods to the public.
Read the detailed description
During routine clinical care, patients and physicians are often confronted with the following questions: "Given my (my child's) responses to the previous treatments, what is the best treatment option for me (my child)?" (by a patient/parent) and "What treatment should we recommend to patients who fail to respond to the first (or second) line of treatment?" (by a physician). Both questions are at the heart of patient centered outcomes research and clinical care, yet answers to these questions are seriously hindered by the lack of adequate analytic methods that appropriately take into account the fact that treatments, as well as the determinants of a treatment decision, vary over time during the course of the disease. Case in point: despite many medication options, polyarticular Juvenile Idiopathic Arthritis (pJIA) is often refractory, and requires better adaptive treatment strategies (ATS). Three ATS were recommended by a panel of experts for pJIA patients, but they need adequate analysis methods to evaluate and identify better ATS using observational data. Motivated by our patient-centered questions, and rigorously designed to evaluate the clinical effectiveness of patient centered adaptive treatment strategies (PCATS), the proposed method development will directly address: "development and dissemination of methods for adequate analysis of data in cases where the treatment/exposure varies over time", an area of interest identified by PCORI.
Accomplishing the proposed study will provide much needed double robust Bayesian causal inference methods that take the challenges of analyzing large registry and electronic health records including model uncertainty, large dimensional covariates and the unmeasured confounders, into account. A web-based userfriendly analytic computational package will be developed to allow easy application of the proposed methods. These developments will: 1) immediately offer methods and computational tools for evaluating clinical effectiveness and informing optimal ATS, 2) in the near future, enable shared-decision making tools for identifying optimal PCATS at the point-of-care, and 3) eventually enable a rapid learning system that will facilitate optimal PCATS. This study will have an immediate impact to children and stakeholders of JIA and a long-term broad impact to many chronically ill patients. Successful completion of this project will significantly move PCORI closer to its mission of helping "people make better informed healthcare decisions and improve healthcare delivery and outcomes".
- Conditions
- Juvenile Idiopathic Arthritis
- Keywords
- JIA, Arthritis, pcJIA, PCATS
- Medical subject headings
- Arthritis, Juvenile, Arthritis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Juvenile Idiopathic Arthritis | Children diagnosed with Juvenile Idiopathic Arthritis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical response measured by clinical Juvenile Arthritis Disease Activity Score (cJADAS)
Clinical trials outcome measurement based on 3 measures that are collected during office visits. The three measures are: active joint count, physician global assessment, and parent global evaluation.
Measured: 1 years
Secondary outcomes (1)
Quality of Life as measured by the Pediatric Quality of Life Inventory (PedsQL) survey
PedsQL is a survey that measures the health-related quality of life in both healthy children and children with acute or chronic diseases.
Measured: 1 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2015 (estimated)
- Study startSep 2015
- Primary completionJan 2019
- Study completionJan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Juvenile Idiopathic ArthritisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 465 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute
- Responsible party
- Sponsor
- Investigators
- Bin Huang, PhD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02524340
- Sponsor's protocol ID
- PCATS for JIA
- Status last verified
- Dec 2018
- First submitted
- 12 Aug 2015
- Last updated
- 23 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Data will be shared with PCORI.
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient Centered Adaptive Treatment Strategies for Juvenile Idiopathic Arthritis Using Bayesian Causal Inference
- ClinicalTrials.gov ID
- NCT02524340
- Acronym / sponsor ID
- PCATS for JIA
- Conditions
- Juvenile Idiopathic Arthritis
- Keywords
- JIA, Arthritis, pcJIA, PCATS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 465 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute
- Study start
- Sep 2015
- Primary completion
- Jan 2019 (actual)
- Study completion
- Jan 2019 (actual)
- First posted
- 14 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The best treatment plan for Juvenile Idiopathic Arthritis (JIA) is often complicated. Patients and clinicians often don't know what is the best treatment strategy for a given patient at a given time. The purpose of this study is to develop a method to analyze data in situations where the treatment and disease state change over time. The researchers will develop a web-based package that will use the methods developed in this study. The package will be easy to use and allow dissemination of the methods to the public.
Detailed description
During routine clinical care, patients and physicians are often confronted with the following questions: "Given my (my child's) responses to the previous treatments, what is the best treatment option for me (my child)?" (by a patient/parent) and "What treatment should we recommend to patients who fail to respond to the first (or second) line of treatment?" (by a physician). Both questions are at the heart of patient centered outcomes research and clinical care, yet answers to these questions are seriously hindered by the lack of adequate analytic methods that appropriately take into account the fact that treatments, as well as the determinants of a treatment decision, vary over time during the course of the disease. Case in point: despite many medication options, polyarticular Juvenile Idiopathic Arthritis (pJIA) is often refractory, and requires better adaptive treatment strategies (ATS). Three ATS were recommended by a panel of experts for pJIA patients, but they need adequate analysis methods to evaluate and identify better ATS using observational data. Motivated by our patient-centered questions, and rigorously designed to evaluate the clinical effectiveness of patient centered adaptive treatment strategies (PCATS), the proposed method development will directly address: "development and dissemination of methods for adequate analysis of data in cases where the treatment/exposure varies over time", an area of interest identified by PCORI.
Accomplishing the proposed study will provide much needed double robust Bayesian causal inference methods that take the challenges of analyzing large registry and electronic health records including model uncertainty, large dimensional covariates and the unmeasured confounders, into account. A web-based userfriendly analytic computational package will be developed to allow easy application of the proposed methods. These developments will: 1) immediately offer methods and computational tools for evaluating clinical effectiveness and informing optimal ATS, 2) in the near future, enable shared-decision making tools for identifying optimal PCATS at the point-of-care, and 3) eventually enable a rapid learning system that will facilitate optimal PCATS. This study will have an immediate impact to children and stakeholders of JIA and a long-term broad impact to many chronically ill patients. Successful completion of this project will significantly move PCORI closer to its mission of helping "people make better informed healthcare decisions and improve healthcare delivery and outcomes".
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Juvenile Idiopathic Arthritis | – | –Children diagnosed with Juvenile Idiopathic Arthritis |
Who may qualify
- Ages
- 1 year to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children less or equal to 19 years of age that have been diagnosed with ployarticular course of juvenile idiopathic arthritis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Clinical response measured by clinical Juvenile Arthritis Disease Activity Score (cJADAS)
Time frame: 1 years
Clinical trials outcome measurement based on 3 measures that are collected during office visits. The three measures are: active joint count, physician global assessment, and parent global evaluation.
1 secondary outcome
Quality of Life as measured by the Pediatric Quality of Life Inventory (PedsQL) survey
Time frame: 1 Years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2019.
- Individual participant dataSharing planned: Yes. Data will be shared with PCORI.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bin Huang, PhD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Juvenile Idiopathic Arthritis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical response measured by clinical Juvenile Arthritis Disease Activity Score (cJADAS) (time frame: 1 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2019.