Ilaris NIS in Korea (REASSURE)

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)

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).

Study Overview

• Status: Recruiting
• Conditions: Cryopyrin-associated Periodic Syndromes (CAPS); Systemic Juvenile Idiopathic Arthritis (sJIA); Hereditary Periodic Fever Syndromes; Colchicine Resistance Familial Mediterranean Fever (crFMF); TNF Receptor Associated Periodic Syndrome (TRAPS); Hyper-IgD Syndrome / Mevalonate Kinase Deficiency (HIDS/MKD)
• Intervention / Treatment: Biological: Ilaris

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.

• Study Type: Observational
• Enrollment (Estimated): 25

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• South Korea
  • Seoul, South Korea, 03722
    • Recruiting
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Ilaris; Patients treated with Ilaris in a clinical setting	Biological: Ilaris; Prospective observational study. There is no treatment allocation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events and serious adverse events	Adverse events and serious adverse events in hereditary periodic fever syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) and sJIA patients treated with Ilaris.	Up to 104 weeks from Ilaris treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of complete responders	Defining patients as responders when they are assessed as having achieved complete response at 4 weeks of treatment and reach 16 weeks of treatment without flare for crFMF, TRAPS, HIDS/MKD and sJIA patients or complete response at 8 weeks of treatment and reach 16 weeks of treatment without flare for CAPS patients. Complete response will be evaluated in patients with data of clinical response as measured by Physician Global Assessment of Disease Activity (PGA) (5-point scale of none, minor, mild, moderate and severe) Complete response is defined as meeting both of the following criteria.; PGA ≤ minor (PGA < 2) (clinical response); CRP ≤10 mg/L (= 1 mg/dL), or reduction by ≥70% from the start of treatment (serological response) Flare is defined as meeting both of the following criteria.; PGA ≥ mild (PGA ≥ 2) (clinical flare); CRP >30 mg/L (= 3 mg/dL) (serological flare)	Up to 16 weeks from Liars treatment
Proportion of participants with Physician Global Assessment of Disease Activity (PGA) score <2	Participants assessed by physician on Physician's Global Assessment measured on a 5--point scale for disease activity as: 0 = None/absent; 1 = Minor; 2 = Mild; 3 = Moderate; 4 = Severe.	Up to 104 weeks from Ilaris treatment
Proportion of patients with C- reactive protein (CRP) serological response	Proportion of patients with a C-reactive protein (CRP) of ≤ 10 mg/L or a ≥ 70% decrease from the start of treatment.	Up to 104 weeks from Ilaris treatment
Proportions of patients with serum amyloid A (SAA) normalization	Proportions of patients with serum amyloid A (SAA) normalization defined as SAA ≤ 10 mg/L.	Up to 104 weeks from Ilaris treatment
Proportion of patients classified in each severity level in the physician's severity assessment of key disease -specific signs and symptoms	Physician's severity assessment of key disease-specific signs/symptoms uses the following 5- point scale. 0 = none; = minor; = mild; = moderate; = severe Key signs/symptoms to be assessed differ from disease to disease under study, which are as follows. CAPS: hearing impairment, visual impairment, renal impairment, and joint dysfunction, skin rash, injection site reaction crFMF: chest pain, abdominal pain, arthralgia/arthritis, skin rash TRAPS: skin rash, musculoskeletal pain, abdominal pain, eye manifestations HIDS (MKD): lymphadenopathy, aphthous ulcers, abdominal pain sJIA: arthritis, generalized lymphadenopathy, rheumatoid rash, hepatosplenomegaly or splenomegaly, serositis	Up to 104 weeks from Ilaris treatment
Proportions of patients classified in each severity level in symptoms likely to significantly affect affect physical functioning and vital prognosis	Symptoms that may significantly affect physical functioning and vital prognosis will be evaluated using the following 5-point scale. 0 = none; = minor; = mild; = moderate; = severe Evaluation of auditory disorder, visual disorder and renal impairment, and status of central nervous system disorder	Up to 104 weeks from Ilaris treatment
Percent change from baseline in Health related quality of life (HRQOL) measured by Child Health Questionnaire-Parent Form 50 (CHQ-PF50)	The CHQ is a generic self-administered instrument designed to capture the physical, emotional, and social components of health status of children. It comprises 15 health concepts (range, 0 to 100) exploring either physical and psychosocial domains. Higher scores in the scales indicate better HRQoL.	Up to 104 weeks from Ilaris treatment
Percent change from baseline in Health related quality of life (HRQOL) measured by 36-item Short Form Health Survey (SF-36)	The SF-36 health survey is a subjective measure of health-related QoL and consists of 36 questions relating to eight domains: physical functioning, social functioning, physical role functioning, emotional role functioning, vitality, mental health, pain, and general health. Scores of each domain varies from 0 to 100 points, and a higher score represents better QoL	Up to 104 weeks from Ilaris treatment
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)	The WPAI-SHP contains six items relating to health problems interfering with work or daily activities over the previous week, the WPASI-SHP yields four scores, absenteeism, presenteeism, work productivity loss, and activity impairment, which can range from 0 (no impact on work/activities) to 100 (total incapacity).	Up to 104 weeks from Ilaris treatment

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 29, 2025
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): September 30, 2028

Study Registration Dates

• First Submitted: February 14, 2025
• First Submitted That Met QC Criteria: February 14, 2025
• First Posted (Actual): February 20, 2025

Study Record Updates

• Last Update Posted (Actual): May 19, 2026
• Last Update Submitted That Met QC Criteria: May 15, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Cryopyrin-associated periodic syndromes (CAPS); Hereditary Periodic Fever Syndromes; 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)
• Additional Relevant MeSH Terms: Chronic Inducible Urticaria; Chronic Urticaria; Cold Urticaria; Musculoskeletal Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Chronic Disease; Disease Attributes; Arthritis; Joint Diseases; Rheumatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Hematologic Diseases; Skin Diseases; Immunoproliferative Disorders; Urticaria; Skin Diseases, Vascular; Blood Protein Disorders; Skin Diseases, Genetic; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Hypergammaglobulinemia; Peroxisomal Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Nutritional and Metabolic Diseases; Skin and Connective Tissue Diseases; Hemic and Lymphatic Diseases; Arthritis, Juvenile; Hereditary Autoinflammatory Diseases; Cryopyrin-Associated Periodic Syndromes; Mevalonate Kinase Deficiency; Periodic fever, familial, autosomal dominant; canakinumab
• Other Study ID Numbers: CACZ885NKR01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO