Clinical trial NCT06560606: UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease
Official title: UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Juvenile Idiopathic Arthritis
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
Collaborators: name: Canadian Institutes of Health Research (CIHR)
name: Genome Canada
name: Genome Alberta
name: The Arthritis Society, Canada
name: ReumaNederland
name: ZonMw: The Netherlands Organisation for Health Research and Development
name: Alberta Children's Hospital
name: Ontario Genomics
First posted: 2024-08-19
Enrollment: 4100
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants will be recruited from the patient population of a UCAN study site.

Brief summary: Childhood arthritis is a chronic disabling disease. New medications called biologic therapies are now available to treat arthritis that target key biologic molecules that cause inflammation. Biologic therapies, while very effective in treating arthritis in children, may have serious side effects including infections and potentially cancers, and are very expensive and doctors don't know, which one to choose for which child. The investigators will develop tests that enable them to learn about the biology of each child's arthritis and be able to predict when and which biologic therapy to start and when to stop.

Detailed description: UCAN CAN-DU is a multicenter observational cohort study that will collect prospective data from children with arthritis. Biologic samples, clinical data and patient reported outcomes will be collected.

In addition, the study will also include a health economics component which will include a number of complementary approaches for quantifying and comparing benefits and risks that promote evidence-based, patient centered health care. This will address both the personal and societal economic burden of disease and include qualitative methods to inform the measurement of preferences, economic and simulation modelling to assess the value of biomarker testing. The socioeconomic impact of biomarker based treatment will be evaluated.

All clinical, biological and patient-derived data will be collected at an aggregation point housed and managed by High Performance Computing 4 Health (HPC4Health), a private hospital-only secure cloud-computing service within Compute Canada and physically located at SickKids/UHN. These databases and apps include biospecimen data and data collected through the eHealth platform. This will enable the study team to share and integrate data in near real-time into analytic models throughout the study course; hence providing a near real-time feedback from bench to bedside and vice versa.

The analysis of the cohorts will help define and confirm the biologic pathways predictive of disease course, treatment response and disease remission. This knowledge will then be used to develop a comprehensive clinical predictive tool to guide effective and safe treatment of childhood arthritis.

Eligibility criteria: Inclusion Criteria:

Cohort 1: - Biologic Basis of JIA

* ≤18 years\*
* Active objective arthritis suspected to be JIA or diagnosed with JIA within 6 months of enrolment
* Treatment naïve except for NSAIDs, allowed to have received NSAIDS within 6 months of diagnosis

Cohort 2 - Start Biologics

* JIA diagnosis as per ILAR criteria (all subtypes)
* ≤18 years\*
* Active arthritis
* For sJIA, active disease not necessarily with arthritis.
* Time of start, restart or switch biologic therapy: e.g. failure, insufficient/partial response or intolerance

Cohort 3 - Stop Biologics

* JIA diagnosis as per ILAR criteria (all subtypes)
* ≤18 years\*
* Inactive disease
* Discontinuing/tapering biologics for inactive disease

Cohort 4: Extreme Phenotypes

* Unexplained systemic inflammation with arthritis/arthralgia as a part of manifestations
* High suspicion of genetic contribution
* Severely affected patients with difficult to control disease (ie failure of multiple biologics)

Exclusion Criteria:

Cohort 1 :

* Arthritis explained by another diagnosis
* Joint injections as previous treatment less than 4 weeks prior to enrollment

Cohort 2:

* Arthritis explained by any other cause
* Start on biologics as an indication for uveitis only

Cohort 3:

- Tapering scheme \> 12 months to complete biologics stop

Cohort 4:

- Arthritis explained by another diagnosis

Primary outcomes:
measure: Prospectively collect essential clinical data elements from children with new onset JIA
timeFrame: Up to 24 months
measure: Evaluate clinical outcomes associated with the use of therapeutic agents in children with JIA
timeFrame: Up to 24 months
measure: Evaluate clinical outcomes associated with the de-prescribing of therapeutic agents in children with JIA
timeFrame: Up to 24 months
measure: Prospectively collect essential clinical data elements from children with extreme phenotypes of JIA.
timeFrame: Up to 12 months
measure: Prospectively collect essential biological data elements from children with new onset JIA
timeFrame: Up to 24 months
measure: Evaluate biological outcomes associated with the use of therapeutic agents in children with JIA
timeFrame: Up to 24 months
measure: Evaluate biological outcomes associated with the de-prescribing of therapeutic agents in children with JIA
timeFrame: Up to 24 months
measure: Prospectively collect essential biological data elements from children with extreme phenotypes of JIA
timeFrame: Up to 12 months
measure: Prospectively collect essential socioeconomic data elements from children with new onset JIA
timeFrame: Up to 12 months
measure: Evaluate the socioeconomic impact associated with the use of therapeutic agents in children with JIA
timeFrame: Up to 12 months
measure: Evaluate the socioeconomic impact associated with the de-prescribing of therapeutic agents in children with JIA
timeFrame: Up to 24 months
measure: Prospectively collect essential socioeconomic data elements from children with extreme phenotypes of JIA
timeFrame: Up to 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Alberta Children's Hospital - University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
state: Alberta
city: Vancouver
country: Canada
facility: BC Children's Hospital
state: British Columbia
city: Winnipeg
country: Canada
facility: Children's Hospital Health Science Centre Winnipeg
state: Manitoba
city: St John's
country: Canada
facility: Janeway Children's Hospital and Rehabilitation Centre
state: Newfoundland and Labrador
city: Halifax
country: Canada
facility: IWK Health Centre
state: Nova Scotia
city: Hamilton
country: Canada
facility: McMaster Children's Hospital
state: Ontario
city: London
country: Canada
facility: Children's Hospital, London Health Sciences Centre
state: Ontario
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Montréal
country: Canada
facility: Montréal Children's Hospital
state: Quebec
city: Saskatoon
country: Canada
facility: University of Saskatchewan
state: Saskatchewan
city: Nijmegen
country: Netherlands
facility: Amalia Children's Hospital, Radboudumc
state: Gelderland
city: Boxmeer
country: Netherlands
facility: Sint Maartenskinderkliniek
state: North Brabant
city: Amsterdam
country: Netherlands
facility: Emma Children's Hospital, Amsterdam UMC
state: North Holland
city: Leiden
country: Netherlands
facility: Willem-Alexander Children's Hospital, LUMC
state: South Holland
city: Rotterdam
country: Netherlands
facility: Sophia Children's Hospital, EMC
state: South Holland
city: Groningen
country: Netherlands
facility: Beatrix Children's Hospital, UMCG
city: Utrecht
country: Netherlands
facility: Wilhelmina Children's Hospital, UMCU
Study officials:
affiliation: The Hospital for Sick Children (SickKids), University of Toronto
name: Rae SM Yeung, MD, PhD, FRCPC
role: PRINCIPAL_INVESTIGATOR
affiliation: Wilhelmina Children's Hospital, University Medical Center Utrecht
name: Nico Wulffraat, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Alberta Children's Hospital and Cumming School of Medicine, University of Calgary
name: Susa Benseler, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Wilhelmina Children's Hospital, University Medical Center Utrecht
name: Joost Swart, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Wilhelmina Children's Hospital, University Medical Center Utrecht
name: Bas Vastert, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: