Rapcabtagene Autoleucel for Vasculitis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called rapcabtagene autoleucel, a type of cell therapy, for individuals with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA), both forms of vasculitis (inflammation of blood vessels). The trial aims to determine the safety and effectiveness of this treatment compared to another. Participants will receive either the new treatment or a comparator, both alongside standard medications. This trial may suit those diagnosed with GPA or MPA who experience severe symptoms affecting daily life. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant medical advancements.
Will I have to stop taking my current medications?
The trial mentions a 'washout period' (time without taking certain medications), so you may need to stop some of your current medications before participating. However, the specific medications you need to stop are not detailed in the information provided.
Is there any evidence suggesting that rapcabtagene autoleucel is likely to be safe for humans?
Research has shown that rapcabtagene autoleucel, a type of CAR T-cell therapy, has a safety profile similar to other treatments of its kind. Other studies found that patients generally tolerated this therapy well. Common side effects of CAR T-cell therapies include fever, tiredness, and low blood cell counts. However, these side effects are usually manageable and often resolve over time. The treatment is currently in a Phase 2 trial, indicating that earlier studies deemed it safe enough for further testing, though more research is needed to confirm its safety for a larger population.12345
Why do researchers think this study treatment might be promising for vasculitis?
Unlike the standard treatments for vasculitis, which often include corticosteroids and immunosuppressants, rapcabtagene autoleucel offers a novel approach by utilizing CAR-T cell therapy. This treatment harnesses the patient’s own immune cells, genetically modifying them to better target and combat the disease. Researchers are excited about this therapy because it represents a precision medicine strategy, potentially offering more targeted and effective relief with fewer side effects compared to traditional therapies.
What evidence suggests that rapcabtagene autoleucel might be an effective treatment for vasculitis?
Research has shown that rapcabtagene autoleucel, which participants in this trial may receive, may help treat conditions like Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA). In earlier studies, this treatment quickly and completely reduced certain immune cells called CD19-positive B cells, which are often involved in these diseases. This reduction lasted, with B cells starting to return after about 90 days. These results suggest that rapcabtagene autoleucel could effectively reset the immune system, potentially helping the disease go into remission. While more research is needed specifically for GPA and MPA, these early findings are promising.24678
Who Is on the Research Team?
Novartis Pharmaceuticals
Principal Investigator
Novartis Pharmaceuticals
Are You a Good Fit for This Trial?
Adults aged 18-75 with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA), diagnosed per ACR/EULAR 2022 criteria and positive for ANCA-autoantibodies. Specific details on who cannot participate are not provided.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single infusion of rapcabtagene autoleucel or comparator with concomitant glucocorticoids
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up period
What Are the Treatments Tested in This Trial?
Interventions
- Rapcabtagene autoleucel
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD