A Study to Evaluate the Tolerability, Safety, and Efficacy of an Anti-CD19 CAR-T Product in Patients With B-cell Lymphoproliferative Disorders

This Phase I/II open-label, single-arm clinical trial evaluates the safety, tolerability, and efficacy of anti-CD19 CAR-T cell therapy in adult patients with relapsed or refractory B-cell lymphoproliferative disorders, including B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL). Anti-CD19 CAR-T therapy uses genetically modified T cells engineered to recognize and destroy cancer cells with the CD19 antigen. The investigational therapy utilizes second-generation CAR-T cells. The primary objective of the study is to assess the overall response rate (ORR) at Day 28. In contrast, secondary objectives include evaluating progression-free survival (PFS), duration of response (DoR), and overall survival (OS). The trial will recruit 60 participants between 18 and 70 years old who meet specific eligibility criteria, including CD19 expression on tumor cells and adequate organ function. Participants will first undergo lymphodepletion to prepare their immune systems for CAR-T cell infusion. The CAR-T therapy will be administered in two doses via intravenous infusion. This dosing regimen is designed to optimize safety and maximize therapeutic benefit. The treatment period includes close inpatient monitoring for at least 14 days, followed by additional outpatient assessments. Safety and tolerability are key components of the study, with close observation for potential side effects such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). Other safety measures include evaluating organ function, monitoring for infections, and assessing the presence of antibodies against CAR-T cells. Efficacy will be determined based on tumor responses, measured as complete or partial remission, and by tracking the persistence and expansion of CAR-T cells in the blood. This study is being conducted at a single clinical center in Russia and is sponsored by the National Medical Research Center of Hematology, Ministry of Health of Russia. The expected duration for each participant is approximately 53 days, with long-term follow-up planned to evaluate the durability of treatment responses and overall survival outcomes. The findings from this study will help determine whether anti-CD19 CAR-T therapy is a viable treatment option for patients with difficult-to-treat B-cell blood cancers.