A Phase 3, Randomized, Open-label, Multicenter Study to Compare the Efficacy and Safety of BMS-986353, CD19-targeted NEX-T CAR T Cells, Versus Standard of Care in Participants with Active Systemic Sclerosis (Breakfree-SSc)
Systemic sclerosis, also known as scleroderma, is an autoimmune condition where the body attacks its own tissues, causing skin and internal organs like the lungs to thicken and scar. This Phase 3 trial compares a modern cell therapy called BMS-986353 (a CAR T-cell treatment) with standard medicines already used for this condition, such as nintedanib, tocilizumab, or rituximab. In CAR T-cell therapy, a patient's own immune cells are collected, modified in a specialist laboratory to target the cells causing inflammation, and then infused back into the body. The main goal of this study is to discover whether this approach is better at preserving lung capacity—measured by how much air a person can blow out—and improving skin flexibility, while checking its overall safety compared to standard treatments.
At a glance
What is this study about?
Systemic sclerosis, sometimes called scleroderma, is a rare condition that causes the immune system to mistakenly attack healthy tissue. This leads to the overproduction of collagen, causing the skin to become tight and hard. Crucially, it can also cause scarring in the lungs, making it harder to breathe and transfer oxygen into the blood. Currently, treatments can slow down the illness, but doctors are looking for more effective ways to halt or reverse this damage.
This study, called Breakfree-SSc, is testing a type of treatment called CAR T-cell therapy. Normally used in certain cancers, CAR T therapy involves collecting some of your own white blood cells, adapting them in a laboratory so they can find and destroy the specific immune cells driving the inflammation, and returning them to your bloodstream. This trial compares this one-off cell treatment against the usual standard medicines doctors currently prescribe, such as nintedanib, tocilizumab, or rituximab.
The main question researchers want to answer is whether the CAR T-cell treatment helps protect the lungs better than standard options. They will measure this by tracking forced vital capacity (the amount of air you can forcefully blow out of your lungs) and checking how well oxygen moves into your blood. They will also look closely at whether the treatment softens thickened skin and delays the condition from worsening.
Because this is a Phase 3 trial, researchers are gathering the final evidence needed to confirm whether this treatment is safe and more effective than standard care. The results could help reshape how severe systemic sclerosis is managed in the future.
Key takeaways
- Tests a new cell therapy (CAR T) against standard medicines for systemic sclerosis.
- Aims to find out if the new treatment better preserves lung function and skin softness.
- Open to adults aged 18 and older with active systemic sclerosis.
- Participants are randomly assigned to either the experimental cell therapy or standard care.
- Taking part is completely voluntary and requires close health and breathing monitoring.
Who may be eligible?
This study is open to adults aged 18 and over who have been diagnosed with active systemic sclerosis that is affecting their internal organs, particularly their lungs, or causing progressive skin thickening. Both men and women can join.
To take part, your doctor will check that you are well enough to undergo the treatment procedures. Because CAR T-cell therapy requires preparation, participants must have adequate heart, kidney, and general organ function to safely receive the medications.
You may not be able to take part if you have certain other active infections, serious heart conditions, or severe medical issues that make taking part unsafe. The study team will review your full medical history and current medications to confirm if the trial is suitable for you.
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.
- I am aged 18 or older.
- I have been diagnosed with active systemic sclerosis (scleroderma).
- My condition is currently active and affecting my skin or lungs.
- I am well enough to attend regular hospital appointments and complete breathing tests.
- I am open to receiving either an advanced cell therapy or standard medicines.
What does participation involve?
If you take part, you will first undergo screening tests such as blood work, heart tracings, and breathing tests to ensure it is safe for you to join. Because this is a randomised study, a computer will assign you by chance to either receive the new CAR T-cell therapy or one of the standard medicines chosen by your doctor.
If you are selected to receive the CAR T therapy, your white blood cells will be collected through a simple blood-filtering procedure. While these cells are modified in a laboratory, you may receive standard care. Before your new cells are given back via an intravenous drip, you will receive a short course of chemotherapy medicines (fludarabine and cyclophosphamide) over a few days to prepare your body. You will then stay in hospital or visit the clinic frequently so the medical team can monitor you closely for any reactions.
If you are assigned to the standard treatment group, you will take approved medications, such as daily tablets (like nintedanib) or regular injections (like tocilizumab or rituximab). All participants, regardless of group, will have regular clinic visits over several months to test lung capacity, check skin thickness, and monitor health.
Potential risks and benefits
Locations (5)
- —UnverifiedSpain
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedItaly
- —UnverifiedBelgium
Common questions
What is CAR T-cell therapy?
It is an innovative treatment where your own immune cells are collected, trained in a laboratory to target the cells causing your disease, and given back to you through a drip.
Can I choose which treatment I receive?
No. A computer will randomly assign you to receive either the CAR T-cell therapy or a standard medicine to make sure the trial results are completely fair and unbiased.
How is my lung function measured in the trial?
You will do simple breathing tests where you blow as hard and completely as you can into a tube connected to a machine. This measures how much air your lungs can hold.
Will I have to stay in hospital?
If you receive the CAR T-cell therapy, you will likely need a short stay in hospital so the medical team can watch you closely for any initial reactions.
What happens if I decide to stop taking part?
You are free to leave the trial at any point. Your decision will never impact the care and treatment your regular doctors provide.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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