AuthorisedPhase I and Phase II (Integrated)- OtherInterventional

Anti-CD 19 CAR-T cell therapy in patients with ANCA Vasculitis: A two-stage interventional, prospective, open-label, phase I/II trial in patients with active, treatment refractory, ANCA-IgG-positive vasculitis

This research study is for people in the UK aged 18 and over who have a specific type of ANCA vasculitis (ANCA-IgG-positive) that hasn't gotten better with standard treatments. The study is testing a new cell therapy called KYV-101. This treatment involves modifying your own immune cells to target the cells causing the vasculitis. Researchers want to understand how safe this new treatment is and if it can help by reducing the harmful antibodies in your blood. They'll also check how well your body tolerates the treatment and how long any benefits last. The study will follow participants for a long time, up to 15 years, to monitor their health.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Charite Universitaetsmedizin Berlin KöR

Enrolment target

Start

09 Dec 2024

ANCA-IgG-positive ANCA associated vasculitis

What is this study about?

This study is looking at a new type of treatment called KYV-101 for a serious condition known as ANCA vasculitis. ANCA vasculitis is a rare disease where your immune system mistakenly attacks your own small blood vessels, which can cause damage to various organs like the kidneys, lungs, and nerves. This study is specifically for people whose ANCA vasculitis has not improved with the usual treatments.

The KYV-101 therapy is an advanced treatment where some of your immune cells (a type of white blood cell) are taken from your body, specially changed in a lab, and then given back to you. These modified cells are designed to target and remove other cells that contribute to the vasculitis. The scientists want to find out how safe this new treatment is, what side effects it might have, and if it can successfully reduce the harmful antibodies that cause the condition.

This is a Phase I and Phase II study, which means it's still in the early stages of testing. Phase I usually focuses on safety, while Phase II starts to look at how well the treatment works. The study will involve a long-term follow-up of 15 years to make sure researchers understand all the possible effects of the treatment over time. The goal is to see if KYV-101 could be a new and effective option for people with ANCA vasculitis who currently have limited treatment choices.

Key takeaways

This study is for adults with a specific type of ANCA vasculitis that hasn't responded to previous treatments.
It's testing a new cell therapy called KYV-101, aimed at reducing harmful antibodies.
The main goals are to check the new treatment's safety and how well it works.
Participation involves regular hospital visits, blood tests, and a long-term follow-up for 15 years.
Potential benefits include a new treatment option, but risks like side effects are also possible.

Who may be eligible?

This study is looking for adults aged 18 and older who have a specific type of ANCA vasculitis called ANCA-IgG-positive vasculitis. This means doctors have found certain antibodies in your blood that are linked to the disease.

Importantly, your ANCA vasculitis must be active, meaning it's currently causing problems, and it must not have gotten better with the treatments you've received so far. This study is for those who have not responded well to other therapies.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have ANCA-IgG-positive ANCA vasculitis?
Is your vasculitis currently active?
Has your vasculitis not improved with other treatments?
Are you able to commit to long-term follow-up for up to 15 years?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the new treatment, KYV-101, which is given after you've had some other medicines like Fludarabinphosphat-GRY® and ENDOXAN to prepare your body. You'll have regular visits to the hospital or clinic for tests and check-ups. These include blood tests to measure the antibodies linked to your vasculitis, see how the new cells are working, and check for any side effects. Researchers will also assess your overall health and how your vasculitis is responding.

The study has two main parts: a safety part (Phase I) where they closely watch for any reactions in the first month, and a second part (Phase II) which expands on safety monitoring and also looks at how well the treatment is working up to 24 weeks. After these initial periods, all participants will enter a very long follow-up phase that lasts for 15 years. During this time, you'll continue to have regular check-ups to monitor your long-term health and the lasting effects of the treatment.

Potential risks and benefits

Taking part in a research study always has potential benefits and risks. A potential benefit of this study is that the new treatment, KYV-101, might help improve your ANCA vasculitis if other treatments haven't worked for you. However, it's also possible that the treatment may not help you, or it could cause side effects. Like any new treatment, there are unknown risks, but specific concerns for this type of therapy often include fever, flu-like symptoms, and temporary changes in brain function, especially in the first few weeks. All participants have the right to withdraw from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany

Common questions

What is ANCA vasculitis?

ANCA vasculitis is a rare disease where your immune system attacks your body's small blood vessels, which can damage organs like your kidneys, lungs, or nerves.

What does 'treatment refractory' mean?

It means your ANCA vasculitis hasn't gotten better even after trying standard or usual treatments.

What is KYV-101?

KYV-101 is a new type of cell therapy where your own immune cells are specially modified and then given back to you to fight the disease.

How long will I be followed in this study?

After the initial treatment and monitoring phases, you will be followed for a very long time, for a total of 15 years, to check your long-term health.

Are there other medicines involved in the study?

Yes, before receiving KYV-101, you would likely be given other medications like Fludarabinphosphat-GRY® and ENDOXAN to prepare your body.