Clinical trial NCT06838143: Ilaris NIS in Korea
Official title: Real Life Non-interventional Study on Safety and Effectiveness of Ilaris® (Canakinumab) 150 mg for Subcutaneous Injection in Hereditary Periodic Fever Syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) Patients and sJIA Patients (REASSURE)
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Hereditary Periodic Fever Syndromes
Cryopyrin-associated Periodic Syndromes (CAPS)
Colchicine Resistance Familial Mediterranean Fever (crFMF)
TNF Receptor Associated Periodic Syndrome (TRAPS)
Hyper-IgD Syndrome / Mevalonate Kinase Deficiency (HIDS/MKD)
Systemic Juvenile Idiopathic Arthritis (sJIA)
Keywords: Hereditary Periodic Fever Syndromes
Cryopyrin-associated periodic syndromes (CAPS)
colchicine resistance familial Mediterranean fever (crFMF)
TNF receptor associated periodic syndrome (TRAPS)
Hyper-IgD syndrome / Mevalonate kinase deficiency (HIDS/MKD)
Systemic juvenile idiopathic arthritis (sJIA)
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-02-20
Enrollment: 16
Sex: ALL
Minimum age (years): 2
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: ≥2 year and \<19 year-old pediatric and ≥19 year-old adult hereditary periodic fever syndrome patients and ≥2 year and \<19 year-old sJIA patients receiving Ilaris for the treatment of CAPS, crFMF, TRAPS, HIDS/MKD and sJIA

Brief summary: This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes, Cryopyrin-associated periodic syndromes (CAPS), colchicine resistance familial Mediterranean fever (crFMF), TNF receptor associated periodic syndrome (TRAPS), Hyper-IgD syndrome / Mevalonate kinase deficiency (HIDS/MKD) or Systemic juvenile idiopathic arthritis (sJIA).

Detailed description: This is a prospective observational, multicenter, uncontrolled, open-label non-interventional study in ≥2 year and \<19 year-old pediatric and ≥19 year-old adult hereditary periodic fever syndrome patients and ≥2 year and \<19 year-old sJIA patients receiving Ilaris for the treatment of CAPS, crFMF, TRAPS, HIDS/MKD and sJIA, respectively, partially using retrospective observation to collect and evaluate data on the safety and effectiveness of Ilaris in patients receiving this drug in a clinical setting for any of these indications. The whole study period is up to 4 years, consisting of a 2-year enrollment period and 2-year observation period.

As all pediatric and adult hereditary periodic fever syndrome patients and all sJIA patients receiving Ilaris for approved indications will be enrolled, this study has no fixed sample size.

For subjects who started Ilaris before enrolling in this study, the safety and effectiveness baseline and early period data will be retrospectively collected.

Eligibility criteria: Inclusion Criteria:

1. Written informed consent/assent of the patient or their legal representative/parent (≥2 year and \<19 year-old pediatric patient) for voluntarily participating in this study
2. Age: ≥2 year and \<19 year-old pediatric and ≥19 year-old adult hereditary periodic fever syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) patients and ≥2 year and \<19 year-old sJIA patients
3. Patient who have an agreement to be treated or who have already started treatment with Ilaris in accordance with the approved label information

Exclusion Criteria:

1. Patients receiving Ilaris treatment for autoimmune disease other than CAPS, crFMF, TRAPS, HIDS/MKD or sJIA
2. Patients participating in an interventional clinical trial which would have an impact on routine clinical treatment

Primary outcomes:
measure: Incidence of adverse events and serious adverse events
timeFrame: Up to 104 weeks from Ilaris treatment
Secondary outcomes:
measure: Proportion of complete responders
timeFrame: Up to 16 weeks from Liars treatment
measure: Proportion of participants with Physician Global Assessment of Disease Activity (PGA) score <2
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Proportion of patients with C- reactive protein (CRP) serological response
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Proportions of patients with serum amyloid A (SAA) normalization
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Proportion of patients classified in each severity level in the physician's severity assessment of key disease -specific signs and symptoms
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Proportions of patients classified in each severity level in symptoms likely to significantly affect affect physical functioning and vital prognosis
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Percent change from baseline in Health related quality of life (HRQOL) measured by Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Percent change from baseline in Health related quality of life (HRQOL) measured by 36-item Short Form Health Survey (SF-36)
timeFrame: Up to 104 weeks from Ilaris treatment
measure: Percent change from baseline in Health related quality of life (HRQOL) measured by Work Productivity and Activity Impairment Specific Health Problem v2.0 (WPAI-SHP)
timeFrame: Up to 104 weeks from Ilaris treatment
Other outcomes:
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