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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial investigates a treatment for Antineutrophil Cytoplasmic Antibody (ANCA)-associated Vasculitis, a rare condition where blood vessels become inflamed. The main medication being studied is avacopan (also known as AMG 569), which will be compared to placebo. The study aims to evaluate the long-term safety and effectiveness of avacopan in patients with this condition.

Participants in the study may also receive standard treatments for vasculitis, including rituximab, cyclophosphamide, azathioprine, mycophenolate mofetil, methotrexate, and glucocorticoids. These medications help control inflammation and suppress the immune system to manage the disease. The treatment period will last for 60 months, during which patients will take either avacopan or placebo along with their other medications.

Throughout the study, doctors will monitor patients' health, checking for any side effects and measuring how well the treatment works. They will pay special attention to liver function, allergic reactions, and infections. They will also track improvements in kidney function and overall quality of life. This information will help determine if avacopan is safe and effective for long-term use in treating this type of vasculitis.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment and group assignment

    You will be assigned to one of three treatment groups after joining the clinical trial for ANCA-associated vasculitis (AAV).

    Your kidney function will be measured through an eGFR test (a blood test that checks how well your kidneys are working).

    Your current disease activity will be evaluated using the BVAS scale (a method to measure vasculitis symptoms).

  2. Step 2

    Treatment initiation

    You will receive either avacopan (Tavneos 10 mg capsules) or a placebo (inactive substance) to take by mouth.

    Your treatment will include either cyclophosphamide (given orally or through an IV) or rituximab (given through an IV) as part of the standard therapy.

    Additional medications may include mycophenolate mofetil, azathioprine, or methotrexate, depending on your assigned treatment group.

  3. Step 3

    Regular monitoring

    Your health will be monitored through regular blood and urine tests.

    Doctors will check for any side effects or health changes.

    Your kidney function will be regularly assessed.

    You will complete quality of life questionnaires (SF-36 v2 and EQ 5D-5L) to evaluate how you are feeling.

  4. Step 4

    Long-term follow-up

    The study will continue for 60 months (5 years).

    Your disease activity will be monitored for any signs of relapse.

    Damage from vasculitis will be tracked using a special scale (VDI).

    The amount of medications you need will be recorded throughout the study.

Who can join the trial?

6 criteria

  • You must provide informed consent before any study procedures begin
  • You must be 18 years of age or older (or above the legal age in your country if it's higher than 18)
  • You must have either newly diagnosed or relapsing GPA or MPA (types of blood vessel inflammation) requiring treatment with cyclophosphamide or rituximab
  • You must have tested positive (now or in the past) for anti-PR3 or anti-MPO antibodies (specific blood proteins related to the condition)
  • You must have at least one of these conditions: - One major symptom on the BVAS scale (disease activity measurement scale) - Three non-major symptoms on the BVAS scale - Both protein in urine and blood in urine
  • Your kidney function must be at least 15 mL/min/1.73 m2 (measured by a specific kidney function test called CKD-EPI)

Who cannot join the trial?

15 criteria

  • Patients under 18 years old cannot participate
  • Pregnant or breastfeeding women are not eligible
  • People with severe liver disease (where the liver is not functioning properly)
  • Patients with active infections
  • Those who have received live vaccines within 4 weeks before the study
  • People with uncontrolled high blood pressure
  • Patients with severe kidney disease (where the kidneys are not functioning properly)
  • Those with active cancer or cancer treatment in the past 5 years
  • People with history of severe allergic reactions to similar medications
  • Patients participating in other clinical trials
  • Those with significant heart problems
  • People who cannot follow study procedures or attend scheduled visits
  • Patients with uncontrolled diabetes (high blood sugar levels)
  • Those with history of drug or alcohol abuse within the past 6 months
  • People with mental conditions that could interfere with the study participation
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Investigated drugs

Avacopan is a medication used in the treatment of ANCA-associated vasculitis, which is a rare autoimmune condition affecting blood vessels. This medication works by blocking a specific protein involved in inflammation, helping to reduce the symptoms of vasculitis. It is taken orally and represents an alternative to traditional treatments for this condition. Note: This is a Phase 4 clinical trial studying the long-term effects of the medication, which means the drug has already been approved for use but is being studied further to understand its long-term safety and effectiveness.

What is already known about the treatment

Avacopan – An orally administered medication specifically designed for treating antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), a rare autoimmune condition affecting blood vessels. This drug works by selectively blocking the C5a receptor, which plays a crucial role in the inflammatory process that damages blood vessels in AAV patients. As a complement inhibitor class medication, avacopan represents a targeted approach to treating vasculitis, offering an alternative to traditional broad-spectrum immunosuppressive therapies. The drug has demonstrated effectiveness in clinical trials and is currently being studied for long-term safety and efficacy in Phase 4 trials, marking its established presence in the treatment landscape for AAV.

Investigated diseases

Antineutrophil cytoplasmic antibody-associated vasculitis (AAV) - A rare autoimmune condition where the body's immune system attacks its own blood vessels, causing inflammation and damage. The disease can affect small and medium-sized blood vessels throughout the body, particularly in the kidneys, lungs, skin, and nerves. AAV develops when harmful antibodies called ANCAs (antineutrophil cytoplasmic antibodies) mistakenly target certain white blood cells, leading to blood vessel inflammation. The condition typically progresses gradually, with symptoms varying depending on which organs are affected. The inflammation can cause the blood vessel walls to thicken, reducing blood flow to various organs and tissues.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-503184-42-00Protocol code20220159Estimated enrolment107 patientsSponsorAmgen Inc.

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