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Study of iptacopan effectiveness in achieving remission in patients with active 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 study focuses on patients with ANCA-associated vasculitis, a rare condition where inflammation affects blood vessels. This disease occurs when the body's immune system mistakenly attacks and damages small blood vessels, which can affect various organs in the body. The study will evaluate a new medication called iptacopan (also known as LNP023) compared to standard treatments currently used for this condition.

The purpose of this research is to determine if iptacopan can help patients achieve and maintain a state where the disease is under control (remission). The medication comes in the form of hard gelatin capsules that are taken by mouth. Some participants will receive iptacopan, while others will receive a placebo along with standard care treatments. The study will last for 48 weeks, during which participants will have regular check-ups to monitor their health and how well the treatment is working.

Throughout the study, doctors will assess various aspects of the disease activity, including how well the kidneys are functioning and whether there are signs of blood in the urine. The medication being tested aims to work differently from existing treatments by targeting a specific part of the immune system that is involved in causing the blood vessel inflammation.

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

    Initial evaluation and medication start

    You will begin treatment with iptacopan (study medication) or placebo capsules taken by mouth

    You will also receive rituximab (RTX) and glucocorticoids (GC) as standard treatment

    Your kidney function and urine will be tested to establish baseline measurements

  2. Step 2

    First 24 weeks of treatment

    Regular monitoring of your condition using the BVAS score (a measure of disease activity)

    Blood and urine tests to check kidney function

    Monitoring for any signs of disease relapse

  3. Step 3

    24-week evaluation

    Assessment to determine if you have achieved complete remission

    Evaluation of kidney function through blood and urine tests

    Review of any symptoms or side effects

  4. Step 4

    Weeks 24-48 continuation phase

    Continued treatment with study medication or placebo

    Regular monitoring for any signs of disease relapse

    Ongoing kidney function assessments

  5. Step 5

    48-week final evaluation

    Final assessment of sustained remission status

    Complete evaluation of kidney function

    Final review of disease activity using BVAS score

Who can join the trial?

9 criteria

  • Age requirement: Must be 18 years or older at the time of screening
  • Must sign an informed consent form before participating in the study
  • Must have either newly diagnosed or relapsed GPA (Granulomatosis with Polyangiitis) or MPA (Microscopic Polyangiitis) that requires treatment with rituximab and glucocorticoids as determined by the doctor
  • Must have a BVAS (Birmingham Vasculitis Activity Score) showing either:
    • At least 1 major symptom, or
    • At least 3 minor symptoms, or
    • At least 2 kidney-related symptoms
    • Must have either:
      • A positive blood test for anti-PR3 (anti-proteinase 3) or anti-MPO (anti-myeloperoxidase) antibodies during screening, or
      • Previous documented evidence of a positive test for these antibodies
      • Both men and women can participate

Who cannot join the trial?

15 criteria

  • History of severe allergic reactions to medications
  • Pregnancy or breastfeeding
  • Active or chronic infections, including hepatitis B, hepatitis C, or HIV
  • Severe liver disease (poor liver function)
  • Severe kidney disease requiring dialysis
  • History of cancer within the past 5 years (except for successfully treated skin cancer)
  • Major surgery within 8 weeks before starting the study
  • Participation in other clinical trials within the past 30 days
  • Uncontrolled high blood pressure
  • Mental health conditions that could interfere with study participation
  • Use of prohibited medications that cannot be stopped
  • History of substance abuse within the past year
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • Unable to follow study procedures or attend scheduled visits
  • History of blood clotting disorders
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Investigated drugs

  • Iptacopan

    (also known as LNP023) is a medication being studied for treating ANCA-associated vasculitis, which is a condition where blood vessels become inflamed. This medication works by blocking a specific part of the immune system called the complement pathway. It is designed to reduce inflammation and help patients achieve sustained remission of their disease.

  • Standard of Care (SOC)

    refers to the currently approved and commonly used treatments for ANCA-associated vasculitis. This typically includes medications that suppress the immune system to reduce inflammation and prevent damage to blood vessels. The standard treatments are being used as a comparison to evaluate how well iptacopan works.

What is already known about the treatment

Iptacopan (LNP023) - An oral medication being investigated for the treatment of ANCA-associated vasculitis, a rare autoimmune condition affecting blood vessels. This drug works by inhibiting complement factor B, a key protein in the alternative complement pathway of the immune system, thereby reducing inflammation and vessel damage. Iptacopan is currently being evaluated in clinical trials as a potential alternative to standard immunosuppressive treatments, with the aim of achieving sustained disease remission. As a small-molecule drug taken by mouth, it represents a new approach in treating vasculitis, belonging to the class of complement pathway inhibitors.

Investigated diseases

ANCA-Associated Vasculitis - A rare autoimmune condition where the body's immune system attacks and damages small blood vessels throughout the body. The disease occurs when certain antibodies called ANCA (antineutrophil cytoplasmic antibodies) mistakenly target neutrophils, a type of white blood cell, leading to inflammation of blood vessels. This inflammation can affect multiple organs, including the kidneys, lungs, skin, and nerves. The condition typically develops gradually, with symptoms varying depending on which blood vessels and organs are affected. Common signs include fatigue, fever, weight loss, and various organ-specific symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-510525-15-00Protocol codeCLNP023R12201Estimated enrolment78 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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