A Phase 1 Single Arm, Open Label Study to Evaluate the Safety of UF-KURE-BCMA CAR-T Cells in Patients With Relapsed or Refractory Multiple Myeloma
David Wald
Summary
The purpose of this study is to determine if UF-KURE-BCMA (B-Cell Maturation Antigen) chimeric antigen receptor T cells (CAR-T cells) can be used to treat relapsed or treatment refractory multiple myeloma (RRMM). This treatment uses T cells already present within the body that have been modified outside of the body by a virus and then returned by an infusion to fight cancer. The investigators are evaluating UF-KURE-BCMA because it uses a manufacturing process that is shorter than other Food and Drug Administration (FDA) approved CAR-T cells and only requires a simple blood draw. The standard treatments require weeks to manufacture the cells as well a special procedure to get an individual's cells. While the shorter manufacture time can be an advantage, the safety of this approach has not been demonstrated. The use of UF-KURE-BMCA is investigational and is not approved by the FDA outside of clinical trials. This is the first study of UF-KURE-BCMA in patients. Participants will give a pint of blood, which is the amount one would provide if they were to donate blood. The blood will be used to make the UF-KURE-BCMA cells. Participants will then receive chemotherapy followed by a one-time infusion of the experimental modified CAR-T cells. After this infusion, participants will be watched for side effects and follow up will continue for up to 15 years.
Description
UF-KURE-BCMA is an autologous CAR-T cell therapy consisting of autologous cluster of differentiation 4 (CD4) positive and cluster of differentiation 8 (CD8) positive human T cells that are genetically engineered using a novel ultrafast lentiviral manufacturing system to express a humanized BCMA CAR-T that targets the BCMA receptor to eliminate multiple myeloma cells using simple peripheral blood draws instead of invasive leukapheresis. This ultrafast platform optimizes CAR-T potency and allows for quicker and cheaper manufacturing of these agents. The goal of this phase 1 study is to find reco…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Male or female participants aged 18 years or older. * Subjects must have Multiple Myeloma that is relapsed (defined as non-responsive or progressive disease while on therapy or within 60 days of last treatment in participants who had achieved a minimal response (MR) or better on prior therapy) after three or more lines of therapy including at least one proteasome inhibitor, one imide, and one anti-CD38 agent. * Participants with multiple myeloma with comorbid amyloid and/or extramedullary disease will be eligible. * To be eligible for response assessment, participants mu…
Interventions
- BiologicalUF-KURE-BCMA CAR T-cells
Experimental anti-BCMA CAR T-cells manufactured with a proprietary ultrafast process. Patients will receive a single dose of CAR T-cells at one of three potential doses: * Dose -1: 3x10\^6 cells * Dose 1: 10x10\^6 cells * Dose 2: 15x10\^6 cells
- DrugCyclophosphamide
Patients will receive 3 days of intravenous (IV) cyclophosphamide 500 mg/m\^2 days -5 to -3 or -4 to -2 where day 0 is the day of CAR T-cell infusion.
- DrugFludarabine
Patients will receive 3 days of IV fludarabine 30 mg/m2 days -5 to -3 or -4 to -2 where day 0 is the day of CAR T-cell infusion.
Location
- University Hospitals Cleveland Medical Center Seidman Cancer CenterCleveland, Ohio