RecruitingPHASE3INTERVENTIONAL

Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis

Researchers are looking into a new treatment called Descartes-08 for adults with a type of myasthenia gravis where their immune system attacks nerve connections. This treatment involves using your own cells, which are specially modified and then given back to you. The study, called AURORA, aims to find out if Descartes-08 is safe and effective in improving symptoms. Some people will receive Descartes-08, while others will get a dummy treatment (placebo). The study is divided into two parts, lasting about 6 to 8 months in total for those who qualify for both. This is an important step in potentially finding new ways to manage myasthenia gravis.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Cartesian Therapeutics

Enrolment target

100

Start

06 May 2025

Estimated completion

30 Sep 2027

Myasthaenia Gravis

What is this study about?

Myasthenia gravis is a condition where your immune system mistakenly attacks healthy parts of your body, specifically the connections between your nerves and muscles. This can lead to muscle weakness, which can affect things like your eyes, face, throat, and limbs. Current treatments aim to manage these symptoms, but researchers are always looking for new and better ways to help.

This study is investigating a new type of treatment called Descartes-08, which uses something called "mRNA CAR T-cell therapy." In simple terms, this means we take some of your own immune cells (T-cells), teach them to recognise and fight the specific immune cells that cause myasthenia gravis, and then give them back to you. The idea is to calm down the faulty immune response that's causing the muscle weakness.

The AURORA study is a "Phase 3" trial. This means it's one of the final stages of testing, where the treatment has already shown some promise in earlier studies and we now need to see if it's safe and works well for a larger group of people. Some participants will receive Descartes-08, and others will receive a placebo (an inactive substance that looks like the treatment). This helps us accurately compare the new treatment's effects. The study will look closely at how safe the treatment is and if it helps improve daily life for people with generalized myasthenia gravis.

Key takeaways

This study is testing a new cell-based therapy (Descartes-08) for generalized myasthenia gravis.
It aims to see if the treatment is safe and effective in adults.
Participation involves regular clinic visits, blood tests, and assessments.
Some participants will receive the active cell therapy, others a dummy treatment (placebo).
The study lasts approximately 6 to 14 months depending on eligibility for follow-up.
You can withdraw from the study at any time without affecting your health care.

Who may be eligible?

To join this study, you need to be at least 18 years old and have a specific type of myasthenia gravis that affects many parts of your body (generalized myasthenia gravis), and your symptoms must be at a certain level. You also need to have specific antibodies in your blood that show your immune system is attacking nerve connections, and this must have been confirmed recently.

There are also some important points about your current medications. If you're taking medicines to suppress your immune system, the dose needs to have been stable for at least 8 weeks before starting the study. If you take steroids, your daily dose can't be more than 40mg of prednisone (or its equivalent) and must also have been stable for at least 8 weeks. You also need to be able to attend all study appointments.

Some things would mean you couldn't take part. This includes having other serious ongoing health issues that aren't well controlled, being diagnosed with myasthenia gravis less than a year ago, or having another nerve and muscle disease. You also can't be pregnant or breastfeeding. There are also specific rules about when you last had certain other treatments for myasthenia gravis, like IVIG, plasma exchange, or rituximab, as these might prevent you from joining right away.

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 at least 18 years old?
Do you have generalized myasthenia gravis?
Has your myasthenia gravis been diagnosed for at least 12 months?
Do you have specific antibodies (AChR autoantibodies) in your blood?
If you take immune-suppressing drugs or steroids, has your dose been stable for at least 8 weeks?
Are you able to attend all study appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study involves several visits to the clinic. The first part of the study will last about 6 months, and if you're suitable, you could then continue into a second part for another 8 months. During these visits, you'll have regular check-ups, blood tests, and assessments to see how you're feeling and how the treatment might be affecting your myasthenia gravis. You'll either receive the investigational treatment, Descartes-08, or a placebo, which looks like the treatment but contains no active medicine. The study team will explain all the procedures in detail before you agree to participate. You can ask questions at any time, and you always have the right to withdraw from the study.

Potential risks and benefits

Taking part in a study like this means you might be one of the first to receive a new treatment that could potentially help your myasthenia gravis symptoms. However, like all new medicines, Descartes-08 might have side effects that we don't fully know yet, or it might not work for everyone. There's also a chance you could receive the placebo and not the active treatment. The study team will monitor you very closely for any side effects and will explain all known risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (34)

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

A40
Verified postcode
Tucson, United States· Recruiting
A13
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Carlsbad, United States· Recruiting
A46
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Los Angeles, United States· Recruiting
A14
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Orange, United States· Recruiting
A21
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Aurora, United States· Recruiting
A50
Verified postcode
Washington D.C., United States· Recruiting
A48
Verified postcode
Maitland, United States· Recruiting
A10
Verified postcode
Tampa, United States· Recruiting
A53
Verified postcode
O'Fallon, United States· Recruiting
A20
Verified postcode
Fairway, United States· Recruiting
A16
Verified postcode
Lexington, United States· Recruiting
A38
Verified postcode
Boston, United States· Recruiting

Common questions

What is Descartes-08?

It's a new, experimental treatment for myasthenia gravis that uses your own specially modified immune cells to calm down the disease activity.

What is a placebo?

A placebo is a dummy treatment that looks like the real medicine but contains no active ingredients. It helps researchers fairly compare the effects of the new treatment.

How long will I be in the study?

The first part of the study lasts around 6 months. If you’re eligible for the second part, it could be another 8 months, totaling about 14 months.

Will I know if I'm getting the real treatment or the placebo?

No, during the study, neither you nor the study doctors will know whether you're receiving Descartes-08 or the placebo. This helps ensure fair results.

Can I continue with my usual medications for myasthenia gravis?

You might be able to, but there are specific rules. For example, dosages of certain immune-suppressing drugs must have been stable for at least 8 weeks before joining.

How to find out more

Cartesian Clinical Trials

trials@cartesiantx.com 617-231-8102 Official trial record

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