anakinra for Systemic juvenile idiopathic arthritis (sJIA) | Still's disease

Inclusion criteria

• {"criterion_text":"- Open label lead-in (observational part): 1. Children and adolescents diagnosed with sJIA (ILAR 2004 classification criteria);"}
• {"criterion_text":"- 2. Both male and female patients, aged 8 months - 16 years (anakinra is approved in children aged 8 months and older who suffer from CAPS, and as per definition, JIA has an onset before the age of 16);"}
• {"criterion_text":"- 3. Parents or legal guardian (and the subject when age is appropriate) who are willing to sign the consent/assent forms."}
• {"criterion_text":"- Intervention part (tapering and stop phase): 1. patients treated with rIL-1RA as first line therapy showing an initial beneficial response (no fever on day 7) to rIL-1RA monotherapy (concomitant NSAID allowed);"}
• {"criterion_text":"- 2. Achieving at least an ACRPed90 response without fever around point 90 days after start of therapy on rIL-1RA mono therapy (concomitant NSAID allowed)."}

Exclusion criteria

• {"criterion_text":"- Open label lead-in (observational part): 1. An onset of Macrophage Activation Syndrome (MAS) simultaneously with sJIA or after the diagnosis of sJIA will lead to exclusion of a (potential) subject from participation in this study;"}
• {"criterion_text":"- 2. Previous systemically administered corticosteroid treatment within 6 weeks before diagnosis and enrollment."}
• {"criterion_text":"- 3. Known exclusion criteria for the use of rIL-1RA (renal failure, with a creatinin clearance rate of < 30 ml/min or neutropenia with neutrophil counts of < 1,5 * 10e9/L)."}
• {"criterion_text":"- Intervention part (tapering and stop phase): 1. An onset of Macrophage Activation Syndrome (MAS) after the diagnosis of sJIA will lead to exclusion of a (potential) subject from participation in this study;"}
• {"criterion_text":"- 2. Patients with a relapse of sJIA in the open label lead-in phase of the study will be excluded for the tapering and stop phase, and will switch treatment to concomitant corticosteroid treatment and/or other biological therapy (Tocilizumab or Canakinumab) upon the decision of the treating physician."}

Primary endpoints

• {"endpoint_text":"- * The total (mean/median) number of injections of anakinra per patient necessary to achieve and maintain clinically inactive disease during the first year of treatment.","definition_or_measurement_approach":"Measured as the total (mean/median) number of anakinra injections per patient required to achieve and maintain clinically inactive disease during the first year of treatment."}

Secondary endpoints

• {"endpoint_text":"- * The number of patients with *clinically inactive disease* without medication at time point 1 year.","definition_or_measurement_approach":"Count of patients who meet criteria for clinically inactive disease and are off medication at 1 year."}
• {"endpoint_text":"- * The total number of disease flares during or after tapering and stop of therapy in the first year;","definition_or_measurement_approach":"Total count of disease flare events occurring during or after tapering/stop of therapy within the first year."}
• {"endpoint_text":"- * The number of patients with remission off medication at time point 2 years;","definition_or_measurement_approach":"Count of patients in remission off medication at 2 years."}
• {"endpoint_text":"- * The number of patients needing to switch treatment because of treatment failure during the first year (to calculate reduction in treatment costs)","definition_or_measurement_approach":"Count of patients who switch treatment due to treatment failure within the first year."}
• {"endpoint_text":"- * The number of (serious) adverse events in the first year.","definition_or_measurement_approach":"Count of adverse events and serious adverse events occurring during the first year."}

Netherlands

Earliest CTIS Part Ii Submission Date

02-12-2024

Latest Decision Or Authorization Date

13-12-2024

Processing Time Days

11

Number Of Sites

5

Number Of Participants

68

Primary sponsor

Full Name

Universitair Medisch Centrum Utrecht

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands