RecruitingPHASE2INTERVENTIONAL

Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Diffuse Cutaneous Systemic Sclerosis

This research study is looking at a new treatment called rapcabtagene autoleucel for a severe form of scleroderma, specifically diffuse cutaneous systemic sclerosis (dcSSc). Scleroderma is a rare condition that causes hardening of the skin and sometimes internal organs. The study aims to compare the new treatment with rituximab, a current treatment option, to see which is more effective and safe. Participants will receive either the new treatment or rituximab, and researchers will carefully monitor their health over five years to understand the benefits and any potential side effects. The goal is to find better ways to manage this challenging condition.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

29 Oct 2024

Estimated completion

30 Aug 2032

Scleroderma, Diffuse

What is this study about?

Imagine your body's immune system, which normally fights off infections, starts to mistakenly attack your own healthy tissues. That's a bit like what happens in scleroderma, a rare condition that causes the skin and sometimes internal organs to harden and tighten. This study is specifically for a severe type called diffuse cutaneous systemic sclerosis (dcSSc), where these changes can affect larger areas of skin and potentially vital organs like the lungs or heart. We are looking for new and better ways to help people living with this condition.

This study, called a Phase 2 trial, is testing a new treatment called rapcabtagene autoleucel. It's an advanced therapy, and we want to see how well it works compared to a treatment already in use, rituximab. The main goal is to find out if rapcabtagene autoleucel can make a meaningful difference in the lives of people with severe scleroderma, by reducing symptoms and improving their overall health. We'll be carefully watching for both its helpful effects and any potential side effects it might have.

This important research will involve two groups of participants. Some will start with rapcabtagene autoleucel, and others will be randomly chosen to receive either rapcabtagene autoleucel or rituximab. If those on rituximab aren't improving enough, they might get the chance to switch to rapcabtagene autoleucel if it's right for them. Everyone involved will be looked after closely by a medical team over five years, to ensure their safety and track how their condition responds to the treatment.

Key takeaways

It's exploring a new treatment for severe diffuse scleroderma.
It compares a new treatment with an existing one.
Participants will be closely monitored for five years.
It aims to find better ways to manage scleroderma.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you would generally need to be between 18 and 70 years old and have been diagnosed with severe diffuse scleroderma (dcSSc). Your scleroderma symptoms should have started within the last seven years, and it should be affecting you significantly, for example, through progressive lung disease, severe skin problems, or heart involvement.

There are also some reasons why you might not be able to join. For example, if you have other serious medical conditions that aren't related to your scleroderma, or if you've had a bad reaction to similar medicines in the past. Having certain lung (pulmonary hypertension), kidney problems, or uncontrolled high blood pressure would also prevent you from taking part. The study team will check to make sure you are suitable for the specific treatments being tested.

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 70 years old?
Have you been diagnosed with severe diffuse scleroderma (dcSSc)?
Did your scleroderma symptoms start within the last 7 years?
Are your doctors happy that this study is suitable for you?
Do you have any other serious medical conditions not related to scleroderma?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be part of a pioneering effort over five years. Initially, you might be in a group that first tries rapcabtagene autoleucel or you could be randomly assigned to receive either rapcabtagene autoleucel or rituximab. Both treatments involve infusions (medicines given directly into your bloodstream). The rapcabtagene autoleucel treatment is given once, following a short preparation period. You'll have regular visits to the clinic for medical check-ups, tests, and to discuss how you're feeling. These visits help the study team monitor your health, how the treatment is working, and if you're experiencing any side effects. After the main five-year study, if you received rapcabtagene autoleucel, you'll enter a separate, longer-term follow-up period to continue monitoring your health, which will be explained in detail at that time.

Potential risks and benefits

Participating in this study could offer you access to a new, experimental treatment (rapcabtagene autoleucel) for your severe scleroderma, which might improve your symptoms or slow the progression of your disease, potentially offering benefits beyond standard treatments. However, like all medications, both rapcabtagene autoleucel and rituximab can have side effects, some of which may be serious. The study team will carefully monitor you for these and will explain all known risks. Your safety and well-being are paramount, and you have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (87)

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

UCLA
Verified postcode
Los Angeles, United States· Recruiting
UCSF
Verified postcode
San Francisco, United States· Recruiting
UCSF
Verified postcode
San Francisco, United States· Recruiting
Sutter Health Network
Verified postcode
San Pablo, United States· Recruiting
FL Medical Clinic Orlando Health
Verified postcode
Zephyrhills, United States· Recruiting
Northwestern University
Verified postcode
Chicago, United States· Recruiting
University Of Iowa
Verified postcode
Iowa City, United States· Recruiting
Boston Medical Center
Verified postcode
Boston, United States· Recruiting
Michigan Med University of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
University of Minnesota
Verified postcode
Minneapolis, United States· Recruiting
James Cancer Hospital
Verified postcode
Columbus, United States· Recruiting
Oregon Health Sciences University
Verified postcode
Portland, United States· Recruiting

Common questions

What is diffuse scleroderma?

Diffuse scleroderma is a type of scleroderma where skin hardening affects large areas of the body, including the trunk and upper arms and legs, and can also impact internal organs like the lungs or digestive system.

What is rapcabtagene autoleucel?

Rapcabtagene autoleucel is a new type of treatment being tested in this study. It's a special kind of cell therapy that aims to retrain your immune system to help manage scleroderma.

What is rituximab?

Rituximab is a medication already used for certain autoimmune conditions. It works by targeting specific immune cells that contribute to these diseases.

How long will the study last if I take part?

The main part of the study will last for five years. If you receive rapcabtagene autoleucel, you will then enter a separate long-term follow-up period.

Will I know which treatment I'm getting?

Yes, this is an 'open-label' study, meaning you and your doctor will know whether you are receiving rapcabtagene autoleucel or rituximab.

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.