Table of Contents

• Trial overview
• Conditions being studied
• Trial designs and phases
• Who can take part
• Main outcomes being measured
• Key trial examples

Trial overview

These studies are testing Anakinra in many different patient groups, including children, adults, and critically ill patients.^[1][2] The trials are mainly asking whether Anakinra can help with disease control, recovery, inflammation, and survival in specific conditions.^[3]

The trial data show both early and later research, from Phase 1 safety studies to larger Phase 3 comparison trials and one Phase 4 study.^[1][2] Several trials are authorised, and some are completed.^[1]

Conditions being studied

Anakinra is being studied in many inflammatory and immune-related conditions, including systemic JIA (also called Still’s disease), recurrent pericarditis, Familial Mediterranean Fever, Kawasaki disease, and severe non-classified inflammatory disease.^{[1][4][5][6][7]}

The trials also include serious hospital conditions such as sepsis, acute respiratory distress syndrome, myocarditis, intracerebral haemorrhage, inflammatory dilated cardiomyopathy, and post-acute COVID syndrome.^{[2][3][8][9][10]}

Other studies are testing Anakinra in cystic fibrosis, gout flare in people with kidney disease or kidney transplantation, pancreatic transplant recipients, and pediatric acute myocarditis.^{[11][12][13][14]}

Trial designs and phases

Most of the Anakinra trials are interventional, which means the researchers give a treatment and measure the results.^[1] The studies include randomised trials, placebo-controlled trials, double-blind trials, and open-label trials.^[2][4][5]

Several trials are in Phase 2, where the main focus is on early evidence of benefit and continued safety checks.^{[2][8][9][10][11][13][14][15]}

There are also multiple Phase 3 studies, which are larger trials that compare Anakinra with another treatment or with standard care.^{[1][4][5][6][7][12]}

One study is listed as Phase 4, meaning it is a later study looking at treatment use in a real-world type setting after earlier research has already been done.^[3]

Who can take part

Participant groups differ a lot from one trial to another.^[1] Some studies include children only, such as trials in systemic JIA, recurrent pericarditis, and pediatric acute myocarditis.^[1][4][5]

Other studies include adults, such as the cystic fibrosis trial and the gout trial in people with kidney disease, transplantation, or dialysis.^[11][12]

Some trials focus on very specific groups, such as healthy volunteers in a human endotoxemia study, children with cancer and sepsis, or patients in intensive care units.^[2][15][14]

Main outcomes being measured

The trials measure different outcomes depending on the disease. Common outcomes include overall survival, pain reduction, fever control, heart function, lung function, inflammation markers, and relapse prevention.^{[2][4][5][8][9][10][11][12][13]}

Some studies use more specific measures, such as the number of injections needed to keep disease inactive, the percentage of children with recovered left ventricle ejection fraction, or the number of ventilation-free days.^[1][5][10]

Several trials also include safety measures such as adverse events, dose-limiting toxicity, and changes in laboratory values.^[6][7][13]

Key trial examples

In the ESTIS trial in children with systemic JIA, researchers are studying whether IL-18-guided decisions can reduce the number of Anakinra injections needed to reach and maintain clinically inactive disease during the first year of treatment.^[1]

In the PRECISION study, Anakinra is being tested in people with post-acute COVID syndrome to see whether it improves clinical and immunological status over 4 to 8 weeks using a composite score called the Score of PACS progression reversal.^[3]

In the pediatric recurrent pericarditis trial, Anakinra is compared with steroids to see whether it gives a complete response by day 7 and prevents relapse by month 3.^[4]

In pediatric acute myocarditis, the trial compares Anakinra with placebo on top of standard care and measures recovery of left ventricle ejection fraction at 3 days.^[5]

In the gout trial, Anakinra is compared with prednisone in people with advanced kidney disease, kidney transplantation, or dialysis, and the main outcome is pain change after treatment starts.^[12]

In the Kawasaki disease study, researchers are comparing Anakinra with a second intravenous immunoglobulin infusion in children who did not respond to the first standard infusion, with fever control as the main outcome.^[13]