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Study on Avacopan with Rituximab or Cyclophosphamide for Children Aged 6 to 17 with Active ANCA-associated Vasculitis

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a condition called ANCA-associated vasculitis (AAV), which is a type of inflammation of the blood vessels. The study aims to evaluate the effectiveness and safety of a medication called avacopan when used in combination with other treatments such as rituximab or cyclophosphamide. These treatments are used to manage the symptoms of AAV in children aged 6 to under 18 years who have active disease.

The purpose of the study is to explore how well avacopan works in helping children achieve remission, which means the disease is not active. The study will also look at how the body processes avacopan and monitor any side effects. Participants will receive avacopan along with either rituximab or cyclophosphamide, depending on their treatment plan. The study will last for about a year, during which the participants' health and response to the treatment will be closely monitored.

Throughout the study, researchers will assess the participants' progress by checking if they achieve remission at different points, specifically at 26 weeks and 52 weeks. Remission is defined as having no active disease symptoms and a low dose of steroids. The study will also track the levels of avacopan in the blood and any side effects that may occur. This research aims to provide valuable information on the potential benefits of avacopan for children with ANCA-associated vasculitis.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Introduction to the study

    Upon joining the study, the patient will receive an overview of the trial's purpose, which is to evaluate the effectiveness and safety of avacopan in combination with either rituximab or cyclophosphamide for treating ANCA-associated vasculitis (AAV) in children aged 6 to under 18 years.

    The patient will be informed about the study's open-label and uncontrolled design, meaning all participants will receive the same treatment without a comparison group.

  2. Step 2

    Treatment initiation

    The patient will begin treatment with avacopan, administered orally. The specific dosage and frequency will be determined by the study protocol and the patient's individual needs.

    In addition to avacopan, the patient will receive either rituximab or cyclophosphamide. Rituximab is given intravenously, while cyclophosphamide is also administered intravenously. The choice between these medications will depend on the patient's condition and the study's requirements.

  3. Step 3

    Monitoring and assessments

    Throughout the study, the patient will undergo regular monitoring to assess the treatment's effectiveness and safety. This includes measuring plasma concentrations of avacopan from day 1 through the end of the study.

    The patient's disease activity will be evaluated using the Pediatric Vasculitis Activity Score (PVAS) to determine remission status at weeks 26 and 52.

  4. Step 4

    Evaluation of outcomes

    The primary outcomes include the proportion of patients achieving disease remission at week 26 and sustained remission at week 52, based on PVAS scores.

    Secondary outcomes involve assessing treatment-emergent adverse events, taste and acceptability of the medication, and additional disease remission evaluations using the Birmingham Vasculitis Activity Score (BVAS).

  5. Step 5

    Completion of the study

    The study is expected to conclude by July 19, 2030. Upon completion, the patient will receive a summary of their participation and any relevant findings related to their treatment.

Who can join the trial?

7 criteria

  • The patient or their legally authorized representative must provide written permission to participate in the study. If the patient is too young or unable to give permission, their representative must do so, and the patient must agree in writing if required by local rules.
  • The study is open to both boys and girls who are between 6 and less than 18 years old.
  • The patient must have a clinical diagnosis of either granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). These are specific types of conditions related to blood vessel inflammation.
  • The patient must have a new or returning case of ANCA-associated Vasculitis (AAV) and test positive for specific antibodies called anti-PR3 or anti-MPO.
  • The patient must show certain symptoms on a scoring system called the Pediatric Vasculitis Activity Score (PVAS). This includes having at least one major symptom, or at least three non-major symptoms, or at least two symptoms related to kidney issues like proteinuria (protein in urine) and hematuria (blood in urine). The patient must be receiving or need treatment that includes rituximab or cyclophosphamide.
  • The patient must have an estimated glomerular filtration rate (eGFR) of at least 15 mL/minute/1.73 m². This is a measure of how well the kidneys are working.
  • The patient must weigh at least 15 kg (about 33 pounds) on the first day of the study.

Who cannot join the trial?

6 criteria

  • Patients who are not diagnosed with ANCA-associated Vasculitis (AAV) cannot participate. This is a specific condition where the body's immune system attacks its own blood vessels.
  • Patients who are not within the specified age range for the study cannot participate. The study is looking for a specific age group.
  • Patients who are not willing or able to follow the study procedures cannot participate. This means they need to be able to attend appointments and take any required medication.
  • Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
  • Patients who have other serious health conditions that might interfere with the study cannot participate. This is to make sure the study results are accurate and not affected by other health issues.
  • Patients who are currently participating in another clinical trial cannot participate. This is to avoid any interference between different study treatments.
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Investigated drugs

  • Avacopan

    is a medication being studied for its effectiveness in treating children with a condition called ANCA-associated vasculitis. This condition involves inflammation of the blood vessels. Avacopan works by blocking a specific protein in the body that contributes to inflammation, potentially reducing symptoms and preventing further damage to the blood vessels.

  • Rituximab

    is a medication used in this study as part of a treatment regimen for ANCA-associated vasculitis. It is a type of drug known as a monoclonal antibody, which targets and helps to reduce certain immune cells that are involved in causing inflammation and damage in this condition.

  • Cyclophosphamide

    is another medication included in the study's treatment regimen. It is a chemotherapy drug that works by suppressing the immune system, which can help to control the inflammation and prevent further damage to the blood vessels in patients with ANCA-associated vasculitis.

What is already known about the treatment

Avacopan – Avacopan is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness in treating ANCA-associated vasculitis (AAV), a condition characterized by inflammation of blood vessels. The medication works by inhibiting the complement C5a receptor, which plays a role in the immune system's inflammatory response. Avacopan is classified as a complement inhibitor and is being explored for its potential to reduce inflammation and improve outcomes in patients with AAV.

Investigated diseases

ANCA-associated Vasculitis (AAV) – This is a group of diseases characterized by inflammation of small blood vessels, which can lead to damage in various organs. The inflammation is associated with the presence of specific antibodies called anti-neutrophil cytoplasmic antibodies (ANCA). AAV can affect organs such as the kidneys, lungs, and skin, causing symptoms like fatigue, weight loss, and muscle pain. As the disease progresses, it may lead to more severe symptoms, including kidney dysfunction and respiratory issues. The course of the disease can vary, with periods of activity and remission.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2024-514779-16-00Protocol code20230070Estimated enrolment25 patientsSponsorAmgen Inc.

sourced from the EU Clinical Trials Register and site verification

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