Study of KYV-101, Fludarabine Phosphate, and Cyclophosphamide Monohydrate for Patients with Treatment-Resistant ANCA Vasculitis

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What is this study about?

This clinical trial is focused on studying a condition called ANCA-associated vasculitis, which is a type of inflammation of the blood vessels. The trial will explore a new treatment called Anti-CD19 CAR-T cell therapy. This therapy involves using specially modified cells, known as T-cells, to target and treat the disease. The study will also involve the use of other medications, including KYV-101, Fludarabine phosphate, and Cyclophosphamide monohydrate. These medications are used to prepare the body for the CAR-T cell therapy and help manage the disease.

The purpose of the study is to assess the safety and effectiveness of the Anti-CD19 CAR-T cell therapy in patients with active, treatment-resistant ANCA-associated vasculitis. Participants in the study will receive the treatment and be monitored over a period of time to see how their condition responds. The study will be conducted in two phases, with the first phase focusing on safety and the second phase looking at both safety and how well the treatment works. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, participants will undergo regular check-ups and tests to monitor their health and the progress of the treatment. The study aims to provide valuable information on the potential benefits and risks of using Anti-CD19 CAR-T cell therapy for treating ANCA-associated vasculitis. This research could lead to new treatment options for patients with this challenging condition.

1 joining the trial

Upon joining the trial, you will be required to sign an informed consent form. This document confirms that you understand the trial and agree to participate voluntarily.

2 initial health checks

Before starting the treatment, several health checks will be conducted. These include tests to ensure there is no active infection, a negative pregnancy test for female participants, and assessments like brain MRI, EEG, echocardiogram, and lung function tests to ensure safety during the trial.

3 leukapheresis

Leukapheresis is a procedure where white blood cells are collected from your blood. This step is necessary for the production of the anti-CD19 CAR T cells that will be used in your treatment.

4 lymphodepletion

Before receiving the CAR T cell therapy, you will undergo lymphodepletion. This involves receiving medications to reduce the number of certain immune cells in your body, preparing it for the CAR T cell infusion. The medications used are fludarabine phosphate and cyclophosphamide monohydrate, administered intravenously.

5 CAR T cell infusion

You will receive the anti-CD19 CAR T cell therapy through an intravenous infusion. This is the main treatment phase of the trial.

6 monitoring and follow-up

After the infusion, you will be closely monitored for any side effects or reactions. Regular follow-up visits will be scheduled to assess your health and the effectiveness of the treatment. This includes monitoring for any signs of cytokine release syndrome or neurotoxicity, which are potential side effects.

7 long-term follow-up

The trial includes a long-term follow-up phase lasting up to 15 years. This is to ensure the long-term safety and effectiveness of the treatment. Data will be collected and registered in a medical registry with your consent.

Who Can Join the Study?

Understand and voluntarily sign an informed consent form.
Be a male or female aged between 18 and 75 years at the time of consent.
Be able to attend study visits and follow the study protocol.
Have an active disease, which means having a certain level of disease activity as measured by a specific score (BVAS ≥ 3) at screening.
Have an insufficient response or intolerance to certain treatments, including glucocorticoids and at least one of the following: rituximab, mycophenolate mofetil, azathioprine, methotrexate, cyclophosphamide, or avacopan.
Meet one of the following criteria:
- Have granulomatosis with polyangiitis (GPA) and detectable anti-PR3 antibodies at screening.
- Have microscopic polyangiitis (MPA) and detectable anti-MPO antibodies at screening.
Have no evidence of a clinically significant active infection before certain procedures in the study.
Have no clinically relevant abnormal findings on brain MRI, EEG, echocardiogram, or lung function test that would put you at risk during the study.
Have a negative urine pregnancy test if you are a female patient.
Have a kidney function test result (eGFR) greater than 30 ml/min/m2 before certain procedures.
Have no acute central neurological toxicity before certain procedures.
Receive notification of successful CAR T cell production by the manufacturer before certain procedures.
Male participants, unless surgically sterile, must agree to use two acceptable methods of contraception (e.g., spermicide and condom) during the trial and for 12 months after receiving the study treatment.
Females of childbearing potential must have a negative urine pregnancy test at screening and agree to use a highly effective contraceptive method starting from the time of signing the informed consent form and for 12 months after receiving the study treatment.
Have an updated vaccination record according to the recommendations for immunocompromised patients.

Who Cannot Join the Study?

Patients who do not have ANCA-IgG-positive ANCA associated vasculitis cannot participate. This is a specific type of disease that affects blood vessels.
Patients who are not experiencing active symptoms of the disease cannot participate. This means the disease must currently be causing problems.
Patients whose disease is not treatment-refractory cannot participate. This means the disease must not be responding to standard treatments.
Patients who are not within the specified age range cannot participate. The study is open to certain age groups only.
Patients who are not part of the specified clinical trial group cannot participate. This refers to specific categories of patients the study is focusing on.
Patients who are not considered part of a vulnerable population cannot participate. This refers to groups of people who might need special protection in research.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not yet recruiting	01.01.2025

Trial locations

Investigated drugs:

Anti-CD19 CAR T cell therapy is a type of treatment that uses the patient’s own immune cells to fight disease. In this therapy, a special type of white blood cell called a T cell is taken from the patient’s blood. These T cells are then modified in a laboratory to recognize and attack cells that have a specific protein called CD19 on their surface. Once the T cells are ready, they are infused back into the patient’s body. This therapy is being tested to see if it can help patients with a condition called ANCA vasculitis, which is a disease where the immune system mistakenly attacks the body’s own blood vessels. The goal of this therapy is to reduce the activity of the disease and improve the patient’s symptoms.

ANCA-associated vasculitis – ANCA-associated vasculitis is a group of diseases characterized by inflammation of small to medium-sized blood vessels. This inflammation is caused by the presence of anti-neutrophil cytoplasmic antibodies (ANCA) in the blood. The disease can affect various organs, including the kidneys, lungs, and skin, leading to a range of symptoms such as fatigue, weight loss, and muscle pain. As the disease progresses, it can cause damage to the affected organs, leading to more severe symptoms like kidney dysfunction or respiratory issues. The inflammation can lead to the formation of granulomas, which are small areas of inflammation that can further damage tissues. Over time, the disease may cause chronic damage to the blood vessels and organs involved.

Trial ID:

2024-517303-36-00

Protocol code:

CCM-RNT-202402

NCT ID:

NCT06590545

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study of KYV-101, Fludarabine Phosphate, and Cyclophosphamide Monohydrate for Patients with Treatment-Resistant ANCA Vasculitis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?