Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as rapcabtagene autoleucel (YTB323), Rapcabtagene autoleucel single agent, YTB323
Rapcabtagene autoleucel · 11 trials · 16 indications
Event-free survival (EFS) defined as the time from Randomization to the first occurrence of as per protocol defined events.
The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to moderate-to-major improvement.
To demonstrate the superiority of rapcabtagene autoleucel as a single infusion compared to rituximab, with respect to the proportion of participants achieving a Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) response at Week 52. This response is assessed across 5 assessment domains: (1) modified Rodnan Skin Score (mRSS), (2) Health Assessment Questionnaire Disability Index (HAQ-DI), (3) patient global assessment (PGA), (4) physician global assessment (PhGA) and (5) percent-predicted forced vital capacity (FVC%).
Defined as: Meeting the criteria of the Definition Of Remission In Systemic Lupus Erythematosus (DORIS) or Achieving complete renal response (CRR)
Proportion of participants who achieve conversion from MRD-positive status at baseline to MRD-negative status at Day 90 (± 2 weeks) following infusion of rapcabtagene autoleucel (YTB323).
Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation
Incidence of AE's, including Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANs), changes in Vital Signs, Laboratory parameters, ECG, and neurological status qualifying and reported as AEs.
Incidence of dose limiting toxicities (DLTs), AEs and SAEs, including changes in vital signs, electrocardiograms (ECGs), laboratory parameters, neurological status and magnetic resonance (MRI) of the brain and spinal cord qualifying and reported as AEs.
Occurrence, severity, and frequency of dose limiting toxicities (DLTs), AEs and SAEs, including changes in vital signs, electrocardiograms (ECGs), laboratory parameters, neurological status and magnetic resonance (MRI) of the brain and spinal cord qualifying and reported as AEs.
Long term safety follow up
CRR defined as best overall response (BOR) of CR after rapcabtagene autoleucel infusion as per Lugano criteria for 3L+ Diffuse Large B-Cell Lymphoma (DLBCL) and 1L High Risk Large B-Cell (HR LBCL)
| Arm | Type | Description |
|---|---|---|
| Rapcabtagene autoleucel | EXPERIMENTAL | Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol |
| Active comparator | ACTIVE_COMPARATOR | Comparator and concomitant glucocorticoids as per protocol |
| Comparator | ACTIVE_COMPARATOR | Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab) |
| rapcabtagene autoleucel arm | EXPERIMENTAL | rapcabtagene autoleucel |
| rituximab arm | ACTIVE_COMPARATOR | rituximab |
| Observational Cohort (MRD-Negative) | NO_INTERVENTION | Participants who are minimal residual disease (MRD) negative following frontline therapy will not receive study intervention and will be followed for subsequent treatments, response, disease status, and survival. |
| Rapcabtagene Autoleucel (YTB323) Treatment Cohort | EXPERIMENTAL | Participants who are MRD-positive following frontline therapy and meet eligibility criteria will undergo leukapheresis, lymphodepleting chemotherapy, and infusion of rapcabtagene autoleucel (YTB323). Participants will be followed for MRD conversion, safety, disease status, survival, and long-term gene therapy follow-up. |
| Rapcabtagene autoleucel-rheumatoid arthritis | EXPERIMENTAL | Single infusion of Rapcabtagene autoleucel in participants with rheumatoid arthritis |
| Rapcabtagene autoleucel- Sjögren's Disease | EXPERIMENTAL | Single infusion of Rapcabtagene autoleucel in participants with Sjögren's Disease |
| YTB323 | EXPERIMENTAL | YTB323 single intravenous (i.v.) infusion |
| YTB323 Cohort 1 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 2 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 3 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| YTB323 Cohort 4 | EXPERIMENTAL | Participants will receive one dose of YTB323 |
| CLL/SLL | EXPERIMENTAL | Dose escalation of rapcabtagene autoleucel in combination with ibrutinib |
| 3L+ DLBCL | EXPERIMENTAL | Dose escalation and expansion of rapcabtagene autoleucel single agent in 3L+ DLBCL |
| Adult ALL | EXPERIMENTAL | Dose escalation of rapcabtagene autoleucel single agent in adult ALL |
| 1L HR LBCL | EXPERIMENTAL | Rapcabtagene autoleucel single agent in 1L HR LBCL |
| Name | Type | Description |
|---|---|---|
| Rapcabtagene autoleucel | BIOLOGICAL | Single infusion of rapcabtagene autoleucel |
| Active Comparator | OTHER | Active comparator option as per protocol |
| Glucocorticoids | DRUG | Concomitant glucocorticoids as per protocol |
| Active Comparator Option | OTHER | Investigator choice of treatment as per protocol |
| rituximab | BIOLOGICAL | rituximab intravenous infusion (i.v.) as per protocol |
| Rapcabtagene autoleucel (YTB323) | BIOLOGICAL | Autologous CD19-directed chimeric antigen receptor (CAR) T-cell therapy manufactured from participant-derived T cells. Participants undergo leukapheresis for cell collection, receive lymphodepleting chemotherapy, followed by a single intravenous infusion of rapcabtagene autoleucel (YTB323). |
| YTB323 | GENETIC | CAR-T cell suspension for intravenous infusion |
| Rapcabtagene autoleucel single agent | BIOLOGICAL | Single infusion of rapcabtagene autoleucel |
| Ibrutinib | DRUG | Tablets or capsules for oral daily use |
Key inclusion criteria: 1. Men and women, aged ≥18 and ≤ 75 years with a diagnosis of GPA or MPA according to the American College of Rheumatology/ European League Against Rheumatism 2022 (ACR/EULAR 2022) classification criteria 2. Positive test for ANCA-autoantibodies 3. GPA and MPA participants w...
Rapcabtagene autoleucel is an investigational CAR-T cell therapy developed by Novartis AG (ticker NVS). It is being studied in immunology and oncology indications, including ANCA associated vasculitis, generalized myasthenia gravis, idiopathic inflammatory myopathies, progressive multiple sclerosis, chronic lymphocytic leukemia, and large-cell lymphoma. It remains in clinical development and is not approved.
Rapcabtagene autoleucel is being evaluated in a Phase 2 study for severe active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), both forms of ANCA associated vasculitis. The trial, NCT06868290, has enrolled 126 participants across the United States, Brazil, Israel, Japan, Saudi Arabia, Singapore, Switzerland, and the United Kingdom. It is investigational and not approved for this use.
Rapcabtagene autoleucel is developed by Novartis AG, which trades under the ticker NVS. Novartis is the sponsor of the clinical trials evaluating the therapy across its immunology and oncology indications.
Rapcabtagene autoleucel is in Phase 2 development. Its most advanced trial is a Phase 2 study in severe active GPA or MPA, while other studies in large-cell lymphoma, rheumatoid arthritis, Sjogren's disease, and generalized myasthenia gravis are Phase 1. It is investigational and has not been approved by the FDA.
Rapcabtagene autoleucel is being studied in four registered trials: NCT06868290 (Phase 2, ANCA associated vasculitis), NCT07443137 (Phase 1, large-cell lymphoma), NCT07048197 (Phase 1, rheumatoid arthritis and Sjogren's disease), and NCT06704269 (Phase 1, generalized myasthenia gravis). Total enrollment across these studies is 217 participants.
Yes, Rapcabtagene autoleucel is also known as YTB323. The two names refer to the same investigational CAR-T cell therapy developed by Novartis AG. Clinical trial records use both names interchangeably, such as NCT06704269, which lists YTB323 in its title.