ANCA-Vasculitis YTB323 CAR-T Therapy Trial

  • Research type

    Research Study

  • Full title

    A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)

  • IRAS ID

    1010670

  • Contact name

    Léa Dusuel

  • Contact email

    lea-2.dusuel@novartis.com

  • Sponsor organisation

    Novartis Pharma AG

  • Research summary

    This research study to find out if rapcabtagene autoleucel (also referred to as YTB323), an investigational new therapy, is safe and effective (can help) in people who have severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA), which are different types of small vessel vasculitis conditions. YTB323 is a chimeric antigen receptor (CAR) T cell therapy, which is a type of gene therapy/immunotherapy. CAR-T cell therapy involves collecting and using the participant’s own immune cells, specifically their T cells, to treat their disease. T cells are immune cells that help fight bacteria and viruses. After collection, the participant’s T cells are sent to the manufacturer’s (Novartis) laboratory, where they are genetically “modified” to fight and destroy another type of immune cells called B cells, which are believed to contribute to vasculitis. In a part of this study, the effect of YTB323 will be compared against Rituximab, which belongs to a class of drugs known as monoclonal antibodies and destroys B cells in the body.
    About 126 participants in approximately 16 countries will participate in this study. YTB323 is a type of gene therapy/immunotherapy so, patients who have received it during the study, will be asked to join a separate long-term safety follow-up study, which includes onsite follow-up visits and phone follow-up visits for up to 15 years after receiving YTB323.

  • REC name

    London - West London & GTAC Research Ethics Committee

  • REC reference

    25/LO/0141

  • Date of REC Opinion

    20 Mar 2025

  • REC opinion

    Further Information Favourable Opinion