KEY TAKEAWAYS
- CARTIFAN-1 is a pivotal phase II, open-label study conducted across eight sites in China that evaluated the efficacy and safety of ciltacabtagene autoleucel (cilta-cel).
- The study’s primary endpoint was the overall response rate in patients who received a single infusion of cilta-cel.
- The study enrolled adult patients with RRMM who had received ≥ 3 lines of prior therapy, including a proteasome inhibitor and immunomodulatory drug.
- At an 18-month median follow-up, the study showed a favorable risk-benefit profile for cilta-cel, with an overall response rate of 89.6%.
CARTIFAN-1 had the primary objective of assessing the safety and efficacy of cilta-cel. The chimeric antigen receptor T-cell therapy targets B-cell maturation antigen in Chinese patients diagnosed with relapsed or refractory multiple myeloma (R/R MM).
In an open-label phase II study conducted at eight sites in China, adult patients diagnosed with relapsed and refractory multiple myeloma (R/R MM) were enrolled. These patients had previously undergone at least three lines of therapy, including treatment with a proteasome inhibitor and immunomodulatory drug. The intervention consisted of a single infusion of cilta-cel, with a target dose of 0.75 × 106 chimeric antigen receptor-positive viable T cells per kilogram. The study aimed to evaluate the overall response rate as the primary endpoint. Secondary endpoints included assessing progression-free survival (PFS), overall survival (OS), and monitoring the incidence and severity of adverse events (AEs).
As of the clinical evaluation conducted on July 19, 2021, a total of 48 patients were administered cilta-cel infusion. At a median follow-up of 18 months, the study reported an overall response rate of 89.6% (95% CI, 77.3 to 96.5), with a median time to first response of approximately 1 month. Furthermore, 77.1% of patients (95% CI, 62.7 to 88.0) achieved a complete response or better. The medians for the duration of response, progression-free survival (PFS), and overall survival (OS) was not reached. The 18-month PFS rate was 66.8% (95% CI, 49.4 to 79.4), and the 18-month OS rate was 78.7% (95% CI, 64.0 to 88.0). Common hematologic adverse events (AEs) observed in the study population included anemia (100%), neutropenia (97.9%), lymphopenia (95.8%), and thrombocytopenia (87.5%). Cytokine release syndrome occurred in 97.9% of patients, with 35.4% experiencing grade 3 or 4 severity. The median time to onset of cytokine release syndrome was 7 days, and the median duration was 5 days. Infections were reported in 85.4% of patients, with 37.5% classified as grade 3 or 4. A total of ten deaths were recorded following cilta-cel infusion, with eight of them attributed to treatment-related AEs.
The data presented indicated a favorable balance between risks and benefits associated with a single infusion of cilta-cel, leading to early, profound, and long-lasting responses in extensively treated patients with R/R MM in China.
Source:https://pubmed.ncbi.nlm.nih.gov/36269898/
Clinical Trial: https://clinicaltrials.gov/ct2/show/NCT03758417
Mi JQ, Zhao W, Jing H, Fu W, Hu J, Chen L, Zhang Y, Yao D, Chen D, Schecter JM, Yang F, Tian X, Sun H, Zhuang SH, Ren J, Fan X, Jin J, Niu T, Chen SJ. Phase II, Open-Label Study of Ciltacabtagene Autoleucel, an Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor-T-Cell Therapy, in Chinese Patients With Relapsed/Refractory Multiple Myeloma (CARTIFAN-1). J Clin Oncol. 2023 Feb 20;41(6):1275-1284. doi: 10.1200/JCO.22.00690. Epub 2022 Oct 21. PMID: 36269898.
