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Avacopan

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.

Table of Contents

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.[1] 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.[1] All three studies are interventional, meaning researchers are giving study treatment and then measuring the results.[1]

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.[1] This study is authorised and plans to enroll 25 participants.[1] 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.[1]

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.[2] It is authorised, Phase 3, and plans to enroll 130 participants.[2] 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.[2]

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.[2] 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.[2]

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.[3] It is authorised and plans to enroll 107 participants.[3] The trial includes people with antineutrophil cytoplasmic antibody-associated vasculitis, which is another full name for AAV.[3]

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.[3] The main purpose is to evaluate overall safety over time.[3]

What the trials measure

The trials focus on different endpoints, which are the main results researchers want to measure.[1][2][3] 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.[1]

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.[2] 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.[3]

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.[3] These are all important safety topics because they help show whether a treatment can be used safely over time.[3]

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.[1][2][3] The pediatric study is specific to ages 6 years to under 18 years.[1] 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.[2]

All three studies are authorised, which means they have been approved to move forward in the listed setting.[1][2][3] Together, these trials are designed to learn whether Avacopan can help different groups of people with AAV while also tracking safety carefully.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
NCT06321601 Phase 3 Active ANCA-associated vasculitis in children 6 years to under 18 years Authorised 25
2024-519620-24-01 Phase 3 ANCA-associated vasculitis with severe kidney involvement Authorised 130
2023-503184-42-00 Phase 3 Long-term safety and efficacy in ANCA-associated vasculitis Authorised 107

FAQ

  • What conditions are being studied with Avacopan? The trials study ANCA-associated vasculitis, also called AAV. One study focuses on children with active AAV, and others focus on adults with severe kidney involvement.
  • Who may take part in these Avacopan trials? One trial includes children from 6 years to under 18 years old with active AAV. Other trials include participants with AAV, including people with severe kidney disease.
  • What phases are the Avacopan trials in? The trials listed here are Phase 3 studies. Phase 3 means the treatment is being tested in larger groups to better understand benefit and safety.
  • What are the trials trying to measure? One study looks at kidney function at week 52 using estimated glomerular filtration rate, or eGFR. Another study focuses on long-term safety, including side effects, serious infections, liver problems, and other safety topics.
  • How many people are planned for these studies? The trials listed plan to enroll 25, 130, and 107 participants. This gives a total picture of both pediatric and adult research with Avacopan.

Ongoing Clinical Trials on Avacopan

  • Testing avacopan added to standard treatment in patients with ANCA-associated vasculitis with severe kidney damage

    Recruiting

    3 1 1
    Investigated drugs:
    France

  • Study of Avacopan Safety and Effectiveness in Patients with ANCA-associated Vasculitis

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark France Greece Hungary Poland +1

Glossary

  • ANCA-associated vasculitis (AAV): A disease where the body's immune system attacks blood vessels and causes inflammation. It can affect many organs, including the kidneys.
  • Active disease: This means the illness is currently causing symptoms or inflammation and is not quiet or in remission.
  • Phase 3: A later stage of clinical research in which a treatment is studied in larger groups to learn more about how well it works and how safe it is.
  • Interventional study: A study where participants receive a treatment or comparison treatment so researchers can measure the effects.
  • Estimated glomerular filtration rate (eGFR): A blood-test-based estimate of how well the kidneys are filtering waste from the blood.
  • Serious adverse event (SAE): A harmful medical event that is severe, such as one that is life-threatening, causes hospital care, or leads to major problems.
  • Treatment discontinuation: Stopping the study treatment before the planned end.
  • Refractory vasculitis: Vasculitis that does not respond well to treatment.
  • Relapse: When a disease becomes active again after improving.
  • Hematology: Blood test results that help show how the blood and blood cells are working.
  • Serum chemistry: Blood tests that measure chemicals in the blood, which can help check organ function.
  • Urinalysis: A urine test used to look for signs of disease or kidney problems.

References

  1. https://clinicaltrials.gov/study/NCT06321601
  2. https://clinicaltrials.gov/study/2024-519620-24-01
  3. https://clinicaltrials.gov/study/2023-503184-42-00