Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA
This study is investigating a new treatment called rapcabtagene autoleucel for people with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA). These are types of a rare disease called ANCA-Associated Vasculitis (AAV), which causes inflammation of blood vessels. Researchers want to compare the new treatment with existing therapies to understand if it's more effective and safe. The study involves different groups, with some receiving the new treatment and others receiving a standard comparison treatment, along with steroids. Participants who receive the new treatment will be followed closely for up to 15 years to monitor their health long-term.
At a glance
What is this study about?
This research study is looking into a new way to treat certain serious conditions called Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA). These are rare diseases where the body's immune system mistakenly attacks its own blood vessels, causing inflammation. This can lead to problems in different parts of the body, like the kidneys, lungs, or nose.
The main goal of this study is to test a new treatment called rapcabtagene autoleucel. Doctors want to see if this new treatment works better and is safer than the current treatments available for people with severe active GPA or MPA. They will compare it to an existing treatment, and participants in both groups will also receive steroid medication like they normally would.
By carefully studying how people respond to the new treatment compared to the standard one, researchers hope to find better ways to manage these challenging conditions and improve the lives of those affected.
Key takeaways
- This study tests a new treatment for severe GPA or MPA.
- It compares a new treatment with existing ones.
- Participants will also receive steroid medication.
- Some participants will be followed for up to 15 years.
- Eligibility is for adults aged 18-75 with severe active disease.
Who may be eligible?
To join this study, you need to be an adult between 18 and 75 years old and have a confirmed diagnosis of either GPA or MPA, showing signs of severe active disease.
You also need to have positive ANCA-autoantibodies, which is a specific type of blood test result common in these conditions.
There are several reasons why someone might not be able to join. For example, if you have other serious health problems not related to GPA/MPA that could make the new treatment unsafe, or if you have severe issues with your organs. Also, if you have allergies to any of the medications used in the study, you wouldn't be able to participate.
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 GPA or MPA?
- Is your GPA or MPA currently severe and active?
- Do you have positive ANCA-autoantibodies?
- Are you free of other serious health problems that might make the study unsafe for you?
What does participation involve?
If you join this study, you'll be placed into one of two groups: one receiving the new treatment (rapcabtagene autoleucel) or one receiving a standard comparison treatment, both alongside steroid medication. The study is "assessor-blinded," meaning the doctors checking your progress won't know which treatment you're getting, to ensure a fair assessment.
After you finish the main part of the study, if you received the new treatment, you will then enter a long-term follow-up period. This means you'll have regular check-ups for up to 15 years to make sure you're doing well and to monitor any long-term effects of the treatment. The research team will explain exactly what these visits involve, including blood tests and other assessments, and how long you might need to stay in the study.
Potential risks and benefits
Locations (35)
- University Of ColoradoVerified postcodeAurora, United States· Recruiting
- Mayo Clinic JacksonvilleVerified postcodeJacksonville, United States· Recruiting
- Northwestern UniversityVerified postcodeChicago, United States· Recruiting
- Massachusetts General HospitalVerified postcodeBoston, United States· Recruiting
- Michigan Med University of MichiganVerified postcodeAnn Arbor, United States· Recruiting
- University of MinnesotaVerified postcodeMinneapolis, United States· Recruiting
- Mayo Clinic RochesterVerified postcodeRochester, United States· Recruiting
- Oregon Health Sciences UniversityVerified postcodePortland, United States· Recruiting
- Baylor University Medical CenterVerified postcodeDallas, United States· Recruiting
- Houston Methodist HospitalVerified postcodeHouston, United States· Recruiting
- LDS HospitalVerified postcodeSalt Lake City, United States· Recruiting
- Novartis Investigative SiteVerified postcodeSalvador, Brazil· Recruiting
Common questions
What is ANCA-Associated Vasculitis (AAV)?
AAV is a group of rare diseases where your body's immune system attacks its own small blood vessels, causing inflammation and damage.
What are GPA and MPA?
GPA (Granulomatosis with Polyangiitis) and MPA (Microscopic Polyangiitis) are the two main types of ANCA-Associated Vasculitis.
What is a 'Phase 2' study?
A Phase 2 study typically involves a larger group of patients than earlier phases and aims to see if a new treatment is effective and safe for a specific condition.
What does 'rapcabtagene autoleucel' mean?
This is the name of the new experimental treatment being tested in the study. It's a type of therapy that aims to retrain your immune cells.
Will I know which treatment I'm getting?
You might not know if you're getting the new treatment or the comparison treatment. This is common in studies to ensure fair results.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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