CARTITUDE1

CARTITUDE-1 is a pivotal clinical trial evaluating cilta-cabtagene autoleucel (cilta-cel), a BCMA-directed CAR-T cell therapy, for adults with relapsed or refractory multiple myeloma. The study investigates a patient-specific, autologous CAR-T product designed to target BCMA on malignant plasma cells and recruit immune cells to attack the cancer.

The trial is typically described as a phase 1b/2, open-label, multicenter study enrolling patients who have received multiple prior therapies. Participants undergo collection of their own T cells, genetic modification to express a BCMA-targeting CAR, and a preconditioning lymphodepletion regimen before infusion of the engineered cells. Primary endpoints have focused on overall response rate, with secondary endpoints including duration of response, progression-free survival, overall survival, and depth of response such as minimal residual disease status. Safety monitoring captures adverse events commonly associated with CAR-T therapy, including cytokine release syndrome and neurotoxicity, among others.

Efficacy results from CARTITUDE-1 have reported high rates of objective responses and deep, durable remissions in the studied population, with many patients achieving complete or near-complete responses and MRD negativity in some cases. The safety profile includes known CAR-T–related toxicities, most of which occur within weeks after infusion and are typically managed with standard interventions and supportive care. The findings from CARTITUDE-1 contributed to the regulatory approval and clinical adoption of cilta-cel under various regional programs for relapsed or refractory multiple myeloma.

Ongoing follow-up studies continue to assess long-term durability, safety, and potential use in broader patient groups, as well as combinations with other therapies and earlier-line settings.