Study of iptacopan effectiveness in achieving remission in patients with active ANCA-associated vasculitis

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What is this study about?

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.

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 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 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 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 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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Klinikum der Stadt Ludwigshafen am Rhein gGmbH Ludwigshafen Am Rhein Germany
Algemeen Ziekenhuis Delta Roeselare Belgium
University Of Debrecen Debrecen Hungary
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Rigshospitalet Copenhagen Denmark
Hopital Beaujon Clichy France
medius KLINIKEN gGmbH Kirchheim Unter Teck Germany
University Of Szeged Szeged Hungary
Orszagos Mozgasszervi Intezet Budapest Hungary
Unsfcuhhiwnk Mxxnyzf Cikqxgk Gefsucucd Groningen The Netherlands
Hwbvgv Hibzrilb Herlev Denmark
Cwrboa Hcanwdqxcjc Ushujzhbpvfsl Dr Dbfzm Dijon France
Amqdbhpjyl Pmwtxniu Hnwsgpvf Dy Mjkmsimlg Marseille France
Ayldgs Uzlbfpfngy Hywtzjjz Aarhus Denmark
Krqnhfxe dyj Uryjoehxkudf Mpoyblim Ame Munich Germany
Cygefz Hwxuqwqpwfj Rjstabzy Dfycocbedtifyt Angers France
Fltcvmoyw Pnmo Lg Imsfdszcucxqr Branzhcca Doa Hkknzldz Ulvhzkvpfejpi Lr Pny Madrid Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
15.11.2024
Belgium Belgium
Not recruiting
15.11.2024
Czechia Czechia
Not recruiting
15.11.2024
Denmark Denmark
Not recruiting
15.11.2024
France France
Not recruiting
15.11.2024
Germany Germany
Not recruiting
15.11.2024
Hungary Hungary
Not recruiting
15.11.2024
Spain Spain
Not recruiting
15.11.2024
The Netherlands The Netherlands
Not recruiting
15.11.2024

Trial locations

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.

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 ID:
2023-510525-15-00
Protocol code:
CLNP023R12201
NCT ID:
NCT06388941
Trial Phase:
Therapeutic exploratory (Phase II)

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