Table of Contents
- Trial overview
- Conditions and patient groups
- Trial phases and study designs
- What the trials measure
- Notable studies in the data
- Patient-focused takeaways
Trial overview
The trial data show several studies that investigate Lisocabtagene Maraleucel in blood cancers, with a strong focus on lymphoma.[1] These studies are designed to learn how well the treatment works and how safe it is in different patient groups.[1][2]
Most of the listed studies are interventional trials, which means researchers give a treatment and then measure the results.[1] The trials include both standalone studies and studies where Lisocabtagene Maraleucel is part of a larger treatment plan or long-term follow-up program.[1][3]
Conditions and patient groups
One study looks at adults with relapsed or refractory indolent B-cell Non-Hodgkin Lymphoma, which means a slow-growing lymphoma that either came back after treatment or did not respond well to treatment.[1] This study includes follicular lymphoma and marginal zone lymphoma, and it aims to test treatment effect in these specific subtypes.[1]
Another study focuses on adults with Primary Central Nervous System Lymphoma, and it is designed for people who are unable to undergo autologous stem cell transplant.[2] The trial asks whether Lisocabtagene Maraleucel can help stop the cancer from getting worse within one year after treatment.[2]
Other studies include people with relapsed or refractory aggressive large B-cell lymphoma or relapsed/refractory Large B-Cell Lymphoma, including patients at high risk of relapse.[3][4] These trials are aimed at harder-to-treat disease, where researchers want to improve response after CAR-T therapy or use it in a broader treatment strategy.[3][4]
One long-term follow-up study includes adult and pediatric participants who previously received at least one genetically modified T-cell infusion in an earlier Celgene-sponsored study, including Lisocabtagene Maraleucel.[5] This study is not limited to one cancer type and instead follows people over time for safety and long-term outcomes.[5]
Trial phases and study designs
The listed studies include Phase 2, Phase 3, and Phase 4 trials.[1][5] Phase 2 studies usually look closely at whether a treatment works in a specific disease group and continue to monitor safety.[1][2]
The Phase 3 study in the data is the VESICANS study, which examines changes in extracellular vesicles during CAR-T therapy and during immune effector cell-associated neurotoxicity syndrome, also called ICANS.[6] This study uses several CAR-T products and is focused on hematology, or blood-related diseases.[6]
The Phase 4 study is a long-term follow-up protocol for people treated with gene-modified T cells.[5] It tracks participants for up to 15 years after the last infusion, showing that some research is meant to understand delayed effects and long-term recovery rather than short-term response only.[5]
What the trials measure
Several trials measure overall response rate, which means the percentage of patients whose cancer shrinks or disappears after treatment.[1] In the indolent lymphoma study, this is assessed by PET-CT for follicular lymphoma or by CT for marginal zone lymphoma using the Lugano Classification.[1]
The primary central nervous system lymphoma study measures the proportion of participants whose cancer gets worse or who die within 12 months after treatment.[2] This is a practical way to see whether the treatment can help control disease during the first year after therapy.[2]
In the aggressive large B-cell lymphoma studies, one endpoint is complete metabolic response at 3 months after anti-CD19 CAR-T infusion, and another is complete response rate at the end of treatment.[3][4] A complete response means no clear signs of active cancer are found on study testing.[4]
The long-term follow-up study measures delayed adverse events, persistence of gene-modified T-cell products, vector integration sites, replication competent lentivirus, overall survival, and whether participants progress on study.[5] For younger participants, it also tracks physical growth and sexual maturity until Tanner stage 5 is reached.[5]
The VESICANS study measures endothelial extracellular vesicles, which are tiny particles released by cells, and studies how they change before and after CAR-T treatment according to ICANS grade.[6] This is a more laboratory-based study that helps researchers understand treatment-related neurotoxicity, or nerve system side effects.[6]
Notable studies in the data
NCT04245839 is a Phase 2 study in 232 adults with relapsed or refractory indolent B-cell Non-Hodgkin Lymphoma, and its main goal is to evaluate efficacy and safety.[1] The primary endpoint is overall response rate measured by imaging and the Lugano Classification.[1]
NCT07015242 is a Phase 2 study in 55 adults with newly diagnosed primary central nervous system lymphoma who cannot have autologous stem cell transplant.[2] It looks at whether the cancer worsens or the patient dies within 12 months after receiving Lisocabtagene Maraleucel.[2]
NCT03435796 is a long-term Phase 4 follow-up study with 1,410 participants who previously received gene-modified T-cell therapy, including Lisocabtagene Maraleucel.[5] It is designed to watch for delayed safety issues and long-term outcomes for many years after treatment.[5]
2023-506705-20-00 and 2025-523806-34-00 are Phase 2 studies in aggressive or relapsed/refractory large B-cell lymphoma, with endpoints focused on response after CAR-T-based treatment strategies.[3][4] These trials help show how Lisocabtagene Maraleucel is being studied in difficult lymphoma settings where relapse risk is high.[3][4]
2024-515328-35-00 is a Phase 3 hematology study focused on extracellular vesicles and ICANS during CAR-T therapy, with a total enrollment of 60.[6] While not centered only on Lisocabtagene Maraleucel, it is part of the broader CAR-T research landscape in the source data.[6]
Patient-focused takeaways
These trials show that Lisocabtagene Maraleucel is being studied mainly in lymphoma, especially cases that are hard to treat or have returned after earlier therapy.[1][2][3]
The research is not only about short-term tumor response, but also about long-term safety, survival, and recovery after treatment.[5] Some studies also look at special groups such as people who cannot have transplant and younger participants who need growth follow-up.[2][5]
Because the trials use different phases and different endpoints, they answer different questions: does the treatment work, how long does it work, and what happens over time after treatment.[1][2][5]
