Unknown Cancer NIHR-supported project Digestion, Kidneys & Other Organs

A randomized, controlled study to evaluate LNP023 (iptacopan) in patients with active ANCA-associated vasculitis

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust Cambridge Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Ongoing

In plain English

AI plain-English summary

Patients with ANCA-associated vasculitis will receive a new oral drug, iptacopan, alongside standard treatment to see if it can control their disease without requiring repeated rituximab infusions. This matters because ANCA-associated vasculitis is a rare autoimmune disease where the body attacks its own small blood vessels, causing organ damage. Current treatment relies on rituximab and high-dose glucocorticoids, which carry serious side effects and often fail to prevent relapses. The study targets the complement alternative pathway, a part of the immune system that becomes overactive in this disease. Iptacopan blocks a protein called factor B, potentially stopping tissue damage at its source. If successful, this research could offer patients a daily pill that induces and maintains remission, reducing their dependence on steroids and repeated rituximab cycles. That would mean fewer hospital visits, less toxicity from long-term immunosuppression, and better long-term disease control. The study enrolls 78 adults with newly diagnosed or relapsed disease, tracking them for 54 weeks to compare iptacopan against placebo, both given with rituximab and a rapid steroid taper.

View original technical description
This study is designed to find out if iptacopan at a dose of 200 mg twice daily is safe and effective when used together with rituximab and quick reduction of glucocorticoids (GC) dose, can help patients with ANCA-associated vasculitis to become better (induce remission) and keep the disease controlled (maintain remission) without rituximab redosing.ANCA-associated vasculitis (AAV) is a group of disease that makes your body attack healthy cells by mistake and is characterized by damage to small blood vessels. The two most common types of AAV are granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). AAV is affected by uncontrolled activation of an important system in the body called the complement alternative pathway (AP). The “AP” usually provides protection against infections.Iptacopan is a new oral medication being developed in AAV, which blocks the activity of a protein called factor B which is part of the “AP”. By preventing activation of AP, it is expected to reduce AAV symptoms including tissue damage.This is a randomized study that will last for a duration of up to 54 weeks. Participants will receive iptacopan or placebo on top of rituximab and quick reduction of glucocorticoid dose for the treatment of newly diagnosed or relapsed (return of the disease) active GPA or MPA. The study consists of 4 periods: screening period (up to 2 weeks), induction period (24 weeks), maintenance period (24 weeks), and safety follow-up period (4 weeks). Approximately 78 participants aged 18 years or older with newly diagnosed or relapsed active GPA or MPA who require treatment with rituximab and glucocorticoid will be enrolled in the study.

Researchers

Rachel Jones (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

Lupus CLNP023K12201 Iptacopan Novartis
A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy
An open-label, multi-center, phase 2 basket study to assess efficacy, safety and pharmacokinetics of iptacopan (LNP023) in participants with autoimmune benignhematological disorders
An adaptive, randomized, double-blind, parallel group, placebo-controlled, multicenter phase 2 trial to evaluate the efficacy, safety and tolerability of LNP023 plus standard-of-care with and without oral corticosteroids in patients with active lupus nephritis Class III-IV, +/- V
A randomised controlled trial comparing rituximab to standard immunosuppression as maintenance therapy in ANCA associated vasculitis.

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