In short
Clinical trials are investigating Avacopan in people with ANCA-associated vasculitis, including adults with severe kidney disease and children with active disease. These studies look at how well Avacopan works, how safe it is, and how the body handles it when used with standard treatment.
Key points
- Clinical trials of Avacopan are studying ANCA-associated vasculitis, a disease that causes inflammation of blood vessels. The listed studies include children with active disease and adults with severe kidney involvement. All of the trials shown are Phase 3 studies, which means they are testing the treatment in larger groups. One trial looks at kidney function at week 52, while another focuses on long-term safety. The trial data also show careful monitoring of serious side effects, infections, liver problems, and other safety measures.
Clinical trial overview
Avacopan is being studied in ANCA-associated vasculitis (AAV), which is an inflammatory disease that can affect blood vessels and organs such as the kidneys. The available trials are all Phase 3 studies, so they are later-stage trials designed to learn more about benefit and safety in larger groups. All three studies are interventional, meaning researchers are giving study treatment and then measuring the results.
Pediatric study in active AAV
One trial, NCT06321601, is studying Avacopan in children from 6 years to under 18 years of age who have active AAV. This study is authorised and plans to enroll 25 participants. The trial title says Avacopan is being studied in combination with a rituximab- or cyclophosphamide-containing regimen, which means it is being added to a treatment plan that already includes one of these medicines.
Study in severe kidney involvement
Another trial, 2024-519620-24-01, studies Avacopan added to standard-of-care therapy in ANCA-associated vasculitis with severe kidney involvement. It is authorised, Phase 3, and plans to enroll 130 participants. The brief summary says the goal is to show improvement in kidney function at week 52 in patients with severe forms of AAV-associated rapidly progressive glomerulonephritis, which is a fast-worsening kidney inflammation.
This study uses the kidney measure estimated glomerular filtration rate (eGFR), and the target is eGFR 30 mL/min/1.73m² or higher at week 52. The primary outcome also requires that this result is reached without stopping treatment because of a serious adverse event or needing treatment changes for refractory vasculitis or relapse.
Long-term safety and efficacy study
The third study, 2023-503184-42-00, is a Phase 3 trial looking at the long-term safety and efficacy of Avacopan in participants with AAV. It is authorised and plans to enroll 107 participants. The trial includes people with antineutrophil cytoplasmic antibody-associated vasculitis, which is another full name for AAV.
This study allows several background treatments in the intervention list, including methotrexate, mycophenolic acid, azathioprine, cyclophosphamide, rituximab, and placebo for AMG 569, while Avacopan is listed as Tavneos 10 mg hard capsules. The main purpose is to evaluate overall safety over time.
What the trials measure
The trials focus on different endpoints, which are the main results researchers want to measure. In the pediatric trial, the brief summary says the study is exploring efficacy, and the title also mentions pharmacokinetics, which means how the body takes in and handles the treatment.
In the kidney study, the main endpoint is the proportion of patients who reach eGFR 30 mL/min/1.73m² or higher at week 52 without treatment stopping or major treatment changes. In the long-term study, the main focus is safety, including treatment-emergent adverse events, special safety events, serious adverse events, withdrawals because of side effects, deaths, and changes in vital signs and lab tests.
The long-term safety study also watches for hepatic events and drug-induced liver injury, serious hypersensitivity reactions, serious infections, creatinine phosphokinase increases, malignancy, and major cardiovascular events. These are all important safety topics because they help show whether a treatment can be used safely over time.
Who can participate
Based on the trial data, the target groups are children with active AAV and adults with AAV, including people with severe kidney involvement. The pediatric study is specific to ages 6 years to under 18 years. The kidney study focuses on people who already have very low kidney function at entry, and the brief summary describes inclusion with eGFR 0 to 29 mL/min/1.73m².
All three studies are authorised, which means they have been approved to move forward in the listed setting. Together, these trials are designed to learn whether Avacopan can help different groups of people with AAV while also tracking safety carefully.