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Study of KYV-101 Therapy for Patients with Refractory Generalized Myasthenia Gravis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called refractory generalized myasthenia gravis. This is a chronic autoimmune disease where the body's immune system mistakenly attacks the communication between nerves and muscles, leading to muscle weakness. The study is testing a new treatment called KYV-101, which is a type of cell therapy. Specifically, it uses a technique called Chimeric Antigen Receptor T-Cell (CAR T) therapy. This involves using the patient's own immune cells, which are modified to better recognize and attack certain targets in the body.

The purpose of the study is to understand how safe and tolerable KYV-101 is for patients, as well as how effective it might be in treating the disease. Participants in the study will receive the treatment through an intravenous infusion, which means the medicine is given directly into a vein. The study will follow participants over a period of time to monitor their health and any changes in their condition. This includes checking for any side effects and measuring the strength and function of their muscles.

Throughout the study, researchers will collect information on various health markers, such as the presence of specific antibodies and the levels of certain cells in the blood. This will help them understand how the treatment is working and its impact on the disease. The study aims to provide valuable insights into the potential benefits of KYV-101 for people living with refractory generalized myasthenia gravis.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Enrollment
The study involves individuals diagnosed with refractory generalized myasthenia gravis, specifically those with autoantibodies to AChR and MuSK, classified as Myasthenia Gravis Foundation of America (MGFA) Class IIB-IV.
Step 2
Treatment initiation
The treatment involves the administration of KYV-101, a suspension given through an intravenous infusion. This is a type of therapy known as anti-CD19 chimeric antigen receptor T-cell (CAR T) therapy.
Step 3
Monitoring and assessment
The primary focus is on the safety and tolerability of the treatment, as well as its effectiveness. This includes monitoring the incidence and severity of any adverse events and laboratory abnormalities.
The MG-ADL (Myasthenia Gravis Activities of Daily Living) score is assessed at 24 weeks to evaluate the impact on daily activities.
Step 4
Secondary evaluations
Secondary assessments include the QMG (Quantitative Myasthenia Gravis) score and MGC (Myasthenia Gravis Composite) score at 12, 24, and 52 weeks.
Changes in specific antibodies, such as anti-AChR, anti-MuSK, and anti-LRP4, are measured over time.
Other evaluations include CAR-positive T-cell counts, CAR transgene levels, B-cell counts, and systemic cytokine concentrations.
The presence of anti-KYV-101 antibodies is also monitored.
Quality of life and fatigue are assessed using tools like MGQOL15r, MGFA-PIS, Neuro-QOL Fatigue Scale, and EQ-5D.
Step 5
Trial duration
The estimated end date for the trial is December 31, 2027, with recruitment starting on July 31, 2024.

Who can join the trial?

6 criteria

The patient must have a diagnosis of **myasthenia gravis (MG)**, which is a condition that causes muscle weakness.
The patient must have **autoantibodies** to **AChR** and **MuSK**. Autoantibodies are proteins made by the immune system that mistakenly attack the body's own tissues. AChR and MuSK are specific targets in myasthenia gravis.
The patient must be classified as **Myasthenia Gravis Foundation of America (MGFA) Class IIB-IV**. This classification helps to describe the severity of the condition, with Class IIB-IV indicating moderate to severe symptoms.
The patient must be between the ages of 18 and 64 years old.
Both male and female patients are eligible to participate.
The study may include individuals who are considered part of a **vulnerable population**, which means they might need extra protection or care.

Who cannot join the trial?

5 criteria

Patients who have a different condition than **refractory generalized myasthenia gravis**. This is a specific type of muscle weakness that doesn't respond well to treatment.
Patients who are not within the specified age range for the study.
Patients who are not part of the specific clinical trial group being studied.
Patients who are not male or female, as the study includes both genders.
Patients who are considered part of a vulnerable population, which means they might need special protection or care.

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Verified sites

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Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
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Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Munich, Germany
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Medizinische Hochschule Hannover
Hanover, Germany

Trial locations

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Investigated drugs

KYV-101 is a type of therapy that uses your own immune cells to fight disease. In this trial, it is used to treat people with a condition called generalized myasthenia gravis, which affects the muscles. The therapy involves taking your immune cells, modifying them to better target the disease, and then putting them back into your body. This is done to help your immune system recognize and attack the cells causing the problem.

What is already known about the treatment

KYV-101 – KYV-101 is an investigational therapy administered as an infusion of autologous T-cells that have been genetically modified to express a chimeric antigen receptor (CAR) targeting the CD19 protein. It is currently being studied in clinical trials for its safety, tolerability, and effectiveness in treating refractory generalized myasthenia gravis, a condition where the immune system attacks the communication between nerves and muscles. The therapy works by enabling the modified T-cells to recognize and destroy cells expressing the CD19 antigen, which is involved in the disease process. KYV-101 falls under the pharmacological classification of CAR T-cell therapies, a type of immunotherapy designed to enhance the body's immune response against specific targets.

Investigated diseases

Refractory Generalized Myasthenia Gravis – This is a chronic autoimmune disorder where the body's immune system mistakenly attacks the communication between nerves and muscles, leading to muscle weakness. In the generalized form, it affects multiple muscle groups, including those responsible for eye movement, facial expression, chewing, talking, and swallowing. The term "refractory" indicates that the condition does not respond well to standard treatments. Symptoms can fluctuate, often worsening with activity and improving with rest. Over time, the disease can lead to significant muscle fatigue and weakness, impacting daily activities. The progression varies among individuals, with some experiencing periods of remission.