RecruitingPHASE2INTERVENTIONAL

Phase 2 Study of Rapcabtagene Autoleucel in Myositis

This study is investigating a new type of cell therapy, called rapcabtagene autoleucel, for people with severe inflammatory muscle diseases, also known as myositis. These are conditions where your body's immune system mistakenly attacks its own muscles, causing weakness and damage. The main goal is to find out if this new therapy is both safe and effective for patients who haven't responded well to other treatments. Participants will either receive the new cell therapy or a standard treatment. If those on the standard treatment aren't improving, they might get the chance to try the new therapy later. There will also be a long-term check-up period for those who receive the new therapy.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

123

Start

17 Dec 2024

Estimated completion

29 Nov 2032

Idiopathic Inflammatory Myopathies

What is this study about?

You might have heard of conditions where your body's defence system, called the immune system, mistakenly attacks healthy parts of your body. When this happens to your muscles, it's called inflammatory myositis. It can cause muscle weakness, pain, and make everyday tasks very difficult. Sometimes, standard treatments don't work well enough, or the disease keeps coming back.

This study is looking at a new and advanced treatment called rapcabtagene autoleucel. This involves using your own specially modified immune cells to fight the disease. Researchers want to see if this new approach can help people with severe forms of inflammatory myositis who haven't found relief with other medicines. They are also carefully checking to make sure it's safe to use.

By taking part in this study, you would help doctors understand if this new cell therapy could become a future treatment option for others living with this challenging condition. Comparing it to existing treatments will help them learn a lot about how well it works and any potential side effects.

Key takeaways

This is a study for people with severe inflammatory muscle disease (myositis).
It tests a new cell therapy called rapcabtagene autoleucel.
The study aims to check if the new therapy is safe and helps improve muscle weakness.
Participants are randomly assigned to either the new therapy or a standard treatment.
Long-term follow-up is required for those who receive the new cell therapy.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you need to be an adult between 18 and 75 years old and have a clear diagnosis of inflammatory myositis that is considered active and severe. This means your muscles are still inflamed and causing weakness, even after trying other treatments. Doctors will look for signs of active disease, such as abnormal muscle tests or specific changes seen on scans like an MRI.

You wouldn't be able to join if you have certain other serious health issues, very severe muscle damage already, or if you can't tolerate the medicines used in the study. Your overall health and how well your organs are working will be checked. There are also specific rules about your body weight (BMI). The medical team needs to ensure you are well enough to undergo this specific type of cell therapy.

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 between 18 and 75 years old?
Do you have a diagnosis of inflammatory myositis that is severe and active?
Have other treatments for your myositis not worked well enough?
Are you generally well enough to undergo a new type of cell therapy?
Do you not have other serious health problems that would prevent you from taking part?

Answer every question to see your result.

What does participation involve?

If you join this study, it will involve several stages. Initially, there's a 'lead-in' group who will receive the new treatment. After this, most participants will be put into one of two groups by chance (randomised): one group gets the new cell therapy, and the other gets a standard treatment chosen by your doctor. If you are in the standard treatment group and your myositis isn't getting better, you might be offered the new cell therapy later on, if you meet certain criteria.

You will have regular hospital visits for check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. This will help the study team understand the effects of the treatment. For those who receive the new cell therapy, there will be a separate, very important long-term follow-up study to track your health for many years after the initial treatment. The total duration of your participation in the main study will depend on which group you are in and will be fully explained by the study team.

Potential risks and benefits

Participating in this study might offer a potential benefit if the new cell therapy proves to be effective for your severe myositis, especially if other treatments haven't worked. However, like all medical treatments, there are potential risks and side effects, as this is a new therapy. These will be fully explained to you by the study team, and your safety will be closely monitored throughout the study. Remember, you can choose to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (68)

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

University Of Alabama
Verified postcode
Birmingham, United States· Recruiting
FL Medical Clinic Orlando Health
Verified postcode
Zephyrhills, United States· Recruiting
Northwestern University
Verified postcode
Chicago, United States· Recruiting
Northwestern University
Verified postcode
Chicago, United States· Recruiting
University Of Iowa
Verified postcode
Iowa City, United States· Recruiting
Duke Clinical Research Institute
Verified postcode
Durham, United States· Recruiting
University of Pittsburgh Medical Center
Verified postcode
Pittsburgh, United States· Recruiting
Baylor University Medical Center
Verified postcode
Dallas, United States· Recruiting
Houston Methodist Hospital
Verified postcode
Houston, United States· Recruiting
LDS Hospital
Verified postcode
Salt Lake City, United States· Recruiting
Novartis Investigative Site
Verified postcode
Melbourne, Australia· Recruiting
Novartis Investigative Site
Verified postcode
Barretos, Brazil· Recruiting

Common questions

What is inflammatory myositis?

It's a condition where your immune system mistakenly attacks and inflames your muscles, causing weakness and other symptoms.

What is rapcabtagene autoleucel?

It's a new type of cell therapy that uses your own specially modified immune cells to fight the disease.

Will I definitely get the new treatment if I join?

Not necessarily. Most participants will be randomly assigned to either receive the new treatment or a standard care option, though some may get the new treatment later if their initial treatment isn't working.

How long will I be involved in the study?

The main study's length will vary, but if you receive the new cell therapy, you will also join a long-term follow-up study which lasts much longer.

Can I leave the study if I change my mind?

Yes, you are free to stop participating at any time without it affecting your regular 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.