RecruitingPHASE1, PHASE2INTERVENTIONAL

Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis

This study is investigating a new type of cell therapy, called YTB323, for people living with a serious form of myasthenia gravis (gMG). Myasthenia gravis is a long-term condition that causes muscle weakness. This particular study is for patients whose gMG is hard to treat with regular medicines. Scientists want to understand if YTB323 is safe to use and if it can help improve symptoms. They will also look at how the treatment works inside the body. The study is an early-stage trial, meaning it's one of the first times this treatment is being tested in people with gMG. The goal is to see if it’s a promising option that should be looked into further.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

22 Apr 2025

Estimated completion

26 Oct 2029

Generalized Myasthenia Gravis

What is this study about?

This research study is focused on a new treatment called YTB323 for people who have a condition called generalized myasthenia gravis (gMG). Myasthenia gravis is a condition that makes your muscles weak and tired, which can affect things like moving your eyes, speaking, swallowing, and even breathing. In gMG, the muscle weakness can affect many different parts of your body.

This study is specifically looking at patients whose gMG is considered 'treatment-resistant'. This means that their current medicines haven't helped enough to control their symptoms. YTB323 is a special type of treatment known as a 'CAR-T cell therapy'. This involves taking some of your own immune cells, changing them in a lab to target the disease, and then putting them back into your body to fight the condition.

The main goals of this study are to find out if YTB323 is safe for people with gMG, if it helps their symptoms get better, and how the modified cells behave in the body over time. This information is crucial for deciding if this new treatment should be developed further as a potential option for people living with this challenging condition.

Key takeaways

This study is for a new cell therapy (YTB323) for severe myasthenia gravis.
It's for people whose gMG hasn't responded well to other treatments.
The main goals are to check safety and if it helps with symptoms.
Participation involves a single treatment dose and very long-term monitoring (up to 15 years).
It's an early-stage (Phase 1/2) study, meaning it's one of the first times this treatment is being tested in humans.
Eligibility is strict to ensure patient safety and get clear results.

Who may be eligible?

To join this study, you must be between 18 and 65 years old. You need to have a confirmed diagnosis of generalized myasthenia gravis (gMG) that has been difficult to treat with other medicines. Your doctors will check this by looking at your medical history, specifically if you have certain antibodies in your blood and if your muscles don't work properly in specific tests.

Your myasthenia gravis symptoms also need to be at a certain level (not too mild, and not too severe or life-threatening) and have not improved enough despite taking at least two different types of strong medications. If you're taking steroid medications, your dose needs to be stable, and you must be willing to reduce it to a certain level before starting the main part of the study. Also, if you take medications for myasthenia gravis (like cholinesterase inhibitors), those doses also need to be stable for at least two weeks.

You cannot join if your myasthenia gravis only affects your eyes, or if your symptoms are very severe and need urgent medical attention. Other reasons you might not be able to join include having certain infections (like hepatitis B or C, or HIV), other uncontrolled illnesses that need frequent steroid treatment (like severe asthma or certain gut conditions), or a history of bone marrow or organ transplants.

Quick self-check

Are you between 18 and 65 years old?
Do you have a confirmed diagnosis of generalized myasthenia gravis (gMG)?
Have your gMG symptoms not improved enough despite trying at least two different strong medications?
Are your current medication doses for gMG stable, and are you willing to adjust your steroid dose if needed?
Do you *not* have certain infections (like HIV, hepatitis B/C) or other serious uncontrolled illnesses?
Do you *not* have myasthenia gravis that only affects your eyes or is currently very severe/life-threatening?

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

What does participation involve?

If you are eligible and agree to take part, this study will last for a very long time – up to 15 years after you receive the YTB323 treatment. Initially, you will go through screening tests to make sure the study is right for you. If you qualify, some of your blood cells will be collected in a process called 'leukapheresis'. These cells will then be sent to a lab to be specially prepared as YTB323.

After preparation, you will receive a single dose of YTB323. Throughout the study, you will have regular hospital visits for various tests, like blood tests and physical examinations, to monitor your safety, how well the treatment is working, and how the treated cells are behaving in your body. There will be 15 participants in total, with the first 3 (sentinel cohort) being closely monitored before the remaining 12 participants join. The long follow-up helps researchers understand the long-term effects of this new treatment.

Potential risks and benefits

Participating in this study might offer a potential benefit if YTB323 helps to improve your myasthenia gravis symptoms when other treatments haven't worked. However, because this is an early-stage study for a new type of treatment, there's no guarantee it will help you. Like all medical treatments, YTB323 could have side effects, which the research team will explain to you in detail. The doctors and nurses will closely monitor you for any potential problems. Remember, joining a study is always voluntary, and you have the right to withdraw at any time without affecting your medical care.

Locations (10)

Univ Cali Irvine ALS Neuromuscular
Orange, United States· Recruiting
Wake Forest Univ School of Medicine
Winston-Salem, United States· Recruiting
Houston Methodist Hospital
Houston, United States· Recruiting
Novartis Investigative Site
Bordeaux, France· Recruiting
Novartis Investigative Site
Brest, France· Recruiting
Novartis Investigative Site
Lille, France· Recruiting
Novartis Investigative Site
Chiba, Japan· Recruiting
Novartis Investigative Site
Kyoto, Japan· Recruiting
Novartis Investigative Site
Sheffield, United Kingdom· Recruiting
Novartis Investigative Site
London, United Kingdom· Recruiting

Common questions

What is generalized myasthenia gravis (gMG)?

Generalized myasthenia gravis is a long-term condition that causes weakness in muscles in different parts of your body, like those used for moving your eyes, speaking, swallowing, and breathing.

What is YTB323?

YTB323 is a new type of treatment being studied. It's a special 'cell therapy' that involves using your own changed immune cells to fight the muscle weakness in gMG.

Why is this study being done?

This study aims to find out if YTB323 is safe and effective for people with gMG whose symptoms haven't improved with other treatments. It's an early step in developing new medicines.

How long will I be involved in the study?

If you join, the study involves a very long-term follow-up that could last up to 15 years after you receive the single dose of YTB323.

Can I leave the study if I change my mind?

Yes, taking part in any study is completely your choice. You can decide to leave the study at any time without it affecting your usual medical care.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.