Active not recruitingPHASE2INTERVENTIONAL

Iptacopan in Patients With ANCA Associated Vasculitis

This study is investigating a new medication called iptacopan for people who have ANCA-associated vasculitis, a rare auto-immune condition where the body's immune system mistakenly attacks blood vessels. We want to find out if iptacopan, given alongside existing treatments, can effectively help patients achieve and maintain remission (when the disease is under control). Researchers will also check if this new medicine is safe and well-tolerated. The study will look into how iptacopan might affect kidney function, reduce the need for strong steroids, and improve patients' overall quality of life. This is a Phase 2 trial, meaning it's an earlier stage of drug development to gather more information on effectiveness and safety.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

05 Aug 2024

Estimated completion

30 Dec 2026

Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis

What is this study about?

This study is focused on ANCA-associated vasculitis (AAV), a serious condition where your body's immune system accidentally attacks your small blood vessels. This can lead to inflammation and damage in various parts of the body, often affecting the kidneys, lungs, and nerves. There are two main types of AAV: Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA). Standard treatments usually involve strong medicines to calm the immune system, but these can have side effects.

The main goal of this study is to test a new medication called iptacopan. Researchers want to see if adding iptacopan to the current standard care, which includes a medicine called rituximab, can help people with GPA or MPA get into remission (meaning the disease is inactive) and stay in remission for longer. They also want to understand if iptacopan can improve kidney function, reduce relapses (when the disease flares up again), lessen the side effects usually associated with long-term steroid use, and ultimately improve a patient's quality of life.

This is a randomised study, which means some participants will receive iptacopan and others a placebo (a dummy drug with no active ingredients), alongside their standard treatment. This allows researchers to compare the effects of iptacopan more accurately. By carefully studying patients over time, we hope to learn if iptacopan could become a valuable new treatment option to help people manage their ANCA-associated vasculitis more effectively and with fewer side effects.

Key takeaways

Tests a new medicine, iptacopan, for ANCA-associated vasculitis.
Aims to see if iptacopan helps people achieve and maintain remission.
Compares iptacopan to a placebo, alongside standard rituximab treatment.
Will look at effects on kidney function, side effects, and quality of life.
This is a Phase 2 study, focused on early effectiveness and safety.

Who may be eligible?

To join this study, you would need to have been recently diagnosed with ANCA-associated vasculitis (either GPA or MPA) or have had a return of your symptoms (a relapse). Your doctor must also think that treatment with rituximab and steroids is right for you at this time. Your symptoms would also need to be active enough to warrant treatment, which is measured by a specific scoring system. You would also need to have a positive test for specific antibodies (anti-proteinase 3 or anti-myeloperoxidase) in your blood, either recently or in your medical history.

There are also reasons why you might not be able to join. For example, if you have other serious conditions that cause similar symptoms, like certain lupus or rheumatoid vasculitis, this study might not be suitable. Also, if you have very severe kidney problems, have recently undergone a specific blood filtering treatment (plasma exchange), or are in intensive care with severe bleeding in your lungs, you wouldn't be able to take part.

Quick self-check

Have you been recently diagnosed with ANCA-associated vasculitis (GPA or MPA) or had a recent relapse?
Does your doctor think you need treatment with rituximab and steroids?
Do you have a positive blood test for specific ANCA antibodies?
Do you have good enough kidney function (not severely impaired)?
Have you not had a plasma exchange procedure recently?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you would receive either the new medicine, iptacopan, or a placebo, in addition to your regular treatment with rituximab. You would have regular visits to the clinic for medical check-ups, blood tests, and to fill in questionnaires about your health and how you're feeling. These assessments help the study team monitor your condition, check for any changes, and see how well the treatment is working. The study team will explain the full schedule of visits and tests during an informed consent discussion. The total duration of your participation in the study will also be clearly outlined.

Potential risks and benefits

Participating in a study like this means you might benefit from closely monitored care and potentially receive a new treatment that could improve your condition. However, there's no guarantee the new medicine will work for you, and like all medicines, iptacopan may have side effects that are not yet fully known. The placebo group receives no active study drug, but still benefits from careful monitoring. All medical procedures carry some risks, such as discomfort from blood tests. You have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (45)

Arizona Arthritis and Rheumatology Research PLLC
Mesa, United States
Mayo Clinic Rochester
Rochester, United States
Northwell Health
New York, United States
Novartis Investigative Site
CABA, Argentina
Novartis Investigative Site
La Plata, Argentina
Novartis Investigative Site
CABA, Argentina
Novartis Investigative Site
Concord, Australia
Novartis Investigative Site
Clayton, Australia
Novartis Investigative Site
Innsbruck, Austria
Novartis Investigative Site
Graz, Austria
Novartis Investigative Site
Vienna, Austria
Novartis Investigative Site
Leuven, Belgium

+33 more sites — see the official record for the full list.

Common questions

What is ANCA-associated vasculitis?

It's a rare condition where your immune system attacks blood vessels, causing inflammation and damage, often in the kidneys or lungs.

What is iptacopan?

It's a new medicine being tested to see if it can help control ANCA-associated vasculitis when used with current treatments.

What does 'randomised study' mean?

It means you'll be randomly assigned to either receive iptacopan or a dummy drug (placebo) along with your regular treatment, so we can compare the effects fairly.

Will I still get my usual treatment?

Yes, all participants will receive the standard treatment for ANCA-associated vasculitis, which includes rituximab.

How long will the study last?

The study team will tell you the exact duration of your participation during the initial discussion.