Table of contents
- Clinical trials overview
- Conditions studied
- Who can take part
- Trial phases and study designs
- Main endpoints and outcome measures
- Notable trials
- Long-term follow-up and safety research
Clinical trials overview
These studies investigate Axicabtagene Ciloleucel in people with blood cancers, mainly lymphoma.[1] The trials ask whether it works better than standard treatment, how well it controls disease, and what results are seen over time.[1]
Several studies are authorised, and some are completed.[1] The research is mostly focused on treatment settings where the cancer has come back, did not respond, or is considered high risk.[1]
Conditions studied
The largest group of trials focuses on large B-cell lymphoma, including diffuse large B-cell lymphoma and high-grade B-cell lymphoma.[1] Other studies include relapsed or refractory follicular lymphoma, primary mediastinal large B-cell lymphoma, marginal zone lymphoma, and indolent non-Hodgkin lymphoma.[1]
Some trials use broader groups such as relapsed or refractory aggressive B-NHL, relapsed or refractory non-Hodgkin lymphoma, or hematology patients.[1] One long-term follow-up study includes solid and hematological malignancies after gene-modified cell therapy.[1]
Who can take part
Many studies include adults with relapsed disease, which means the cancer came back after treatment, or refractory disease, which means the cancer did not respond well to treatment.[1] Some trials also focus on patients with high-risk disease or those who are not eligible for autologous stem cell transplantation.[1]
A few studies look at people in the first-line setting, which means before they have received earlier treatment for their lymphoma.[1] Other studies are designed for second-line treatment, meaning the next treatment after the first one does not work or the disease returns.[1]
Trial phases and study designs
The data show mostly Phase 2 and Phase 3 trials.[1] Phase 2 studies usually check early signs that a treatment may work, while Phase 3 studies compare treatments in larger groups and look for stronger evidence of benefit.[1]
Most of the studies are interventional, which means the researchers give a treatment and then measure the results.[1] Some are open-label, meaning both the patient and the study team know which treatment is given, and some are single-arm, meaning there is no comparison group.[1]
Main endpoints and outcome measures
The trials measure different outcomes to see how well treatment works.[1] Common endpoints include event-free survival, progression-free survival, objective response rate, complete response rate, and complete metabolic response on PET scans.[1]
Some studies use PET-based systems such as the Lugano Classification and Deauville criteria to judge whether the lymphoma is still active.[1] Other studies measure long-term safety, late side effects, and growth or development in younger patients treated with gene-modified cells.[1]
Notable trials
ZUMA-23 (NCT05605899) is a Phase 3 study in adults with high-risk large B-cell lymphoma, including DLBCL and HGBL.[1] It compares Axicabtagene Ciloleucel with standard of care therapy and measures event-free survival by blinded central assessment.[1]
LATE-R (2024-512835-53-00) is a Phase 2, single-arm, open-label, multicenter study in late relapse of diffuse large B-cell lymphoma.[1] It looks at complete metabolic response at month 3 after infusion, using PET/CT, Lugano Classification, and Deauville criteria.[1]
NCT05371093 is a Phase 3 study in relapsed or refractory follicular lymphoma that compares Axicabtagene Ciloleucel with standard of care therapy.[1] Its main endpoint is progression-free survival, measured by blinded central assessment.[1]
NCT03105336 is a completed Phase 2 multicenter study in relapsed or refractory indolent non-Hodgkin lymphoma.[1] It measured objective response rate by central read for up to 15 years.[1]
NCT06912529 studied relapsed or refractory primary mediastinal large B-cell lymphoma and measured complete metabolic response rate at 3 months.[1] This study is completed.[1]
Long-term follow-up and safety research
One important study is the long-term follow-up trial for participants treated with gene-modified cells.[1] It tracks late-onset problems that may include neurologic disorders, autoimmune disorders, hematologic disorders, serious infections, and new malignancies.[1]
This follow-up study also looks at height, weight, and sexual maturation in pediatric and adolescent participants.[1] Another trial, the VESICANS study, examines extracellular vesicles during CAR-T therapy and during immune effector cell-associated neurotoxicity syndrome, also called ICANS.[1]
Some studies do not test Axicabtagene Ciloleucel alone but compare it with other CAR-T products or use it as part of a broader treatment strategy.[1] These trials help researchers understand where Axicabtagene Ciloleucel fits best in treatment plans for lymphoma and related blood cancers.[1]
