This phase I trial studies the side effects and best dose of BSB-2002 when given after cyclophosphamide and fludarabine and tests how well it works in treating NPM1-mutated acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). BSB-2002 is a type of personalized autologous T cell receptor-modified T cell therapy. T cells are infection fighting blood cells that can kill cancer cells. The T cells given in this study come from the patient and have a new gene put in them that makes them able to recognize mutated NPM1, a protein on the surface of cancer cells. These NPM1 mutated-specific T cells may help the body's immune system identify and kill NPM1-mutated AML cells. Giving chemotherapy, such as cyclophosphamide and fludarabine, before BSB-2002 helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. Giving BSB-2002 after cyclophosphamide and fludarabine may be safe, tolerable, and/or effective in treating relapsed or refractory AML in patients with NPM1 mutation.
Documented informed consent of the participant
Note: For research participants who do not speak English, a short form consent may be used with a City of Hope (COH) certified interpreter/translator to proceed with screening, while the request for a translated full consent is processed
Age: ≥ 18 years
HLA-A*02:01
Leukemic blast count of > 20,000/µl. If the blast count can be maintained below the threshold with hydroxyurea, the patient would be eligible
Extramedullary only AML
Central nervous system (CNS) involvement refractory to intrathecal chemotherapy and/or standard cranial- spinal radiation
Candidates for hematopoietic cell transplant
Single group
Biospecimen Collection
Procedure/SurgeryUndergo blood sample collection
Bone Marrow Aspiration
Procedure/SurgeryUndergo bone marrow aspiration
Bone Marrow Biopsy
Procedure/SurgeryUndergo bone marrow biopsy
Cyclophosphamide
DrugGiven IV
Echocardiography Test
Procedure/SurgeryUndergo echocardiography
Fludarabine
DrugGiven IV
Leukapheresis
Procedure/SurgeryUndergo leukapheresis
Single Agent Therapy
DrugGiven BSB-2002 IV
Incidence of dose-limiting toxicity
Dose-limiting toxicity (DLT) will be assessed using the National Cancer Institute (NCI)'s Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Adverse events will be coded according to the Medical Dictionary for Regulatory Activities. Each adverse event will be counted once per patient. Separate tables and listings will present treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESI), and dose-limiting toxicities (DLT). Tables will include system organ class, high-level term, and severity grade. Listings will also include treatment interruption or discontinuation, dose level, relevant severity scales, relationship to study intervention, expectedness, onset day, duration day, action taken, outcome, sex, age, and race.
Frequency and severity of adverse events (AEs)
AEs will be graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) scale version (v) 5.0, cytokine release syndrome (CRS) and neurotoxicity (immune effector cell associated neurotoxicity syndrome \[ICANS\]) events will be graded by the American Society for Transplantation and Cellular Therapy (ASTCT) Consensus criteria scales for CRS and ICANS, respectively. Tables and listings will present AEs. Tables will include system organ class, high-level term, and severity grade. Listings will also include treatment interruption or discontinuation, dose level, relevant severity scales, relationship to study intervention, expectedness, onset day, duration day, action taken, outcome, sex, age, and race.
Frequency and severity of serious AEs
AEs will be graded by NCI CTCAE scale v 5.0, CRS and neurotoxicity (ICANS-Immune Effector Cell-Associated Neurotoxicity) events will be graded by the ASTCT Consensus criteria scales for CRS and ICANS, respectively. Tables and listings will present serious AEs. Tables will include system organ class, high-level term, and severity grade. Listings will also include treatment interruption or discontinuation, dose level, relevant severity scales, relationship to study intervention, expectedness, onset day, duration day, action taken, outcome, sex, age, and race.
Complete remission (CR)
Will assess the percentage of patients achieving CR (including CR with measurable residual disease negative, CR, CR with incomplete hematologic recovery \[CRi\], CR with partial hematologic recovery \[CRh\]) or partial remission as determined by the investigator according to the European LeukemiaNet criteria for acute myeloid leukemia. Will provide listings of patients who experience complete remission, including CR, CRi, and CRh, and partial remission.
Duration of response
Duration of response (time from first tumor assessment at which the overall response was recorded as CR/CRi / CRi, CRh (AML) until documented relapse, or death from any cause, whichever occurred first.
Overall survival
The length of time from either the date of diagnosis or the start of treatment for a disease, such as cancer, that patients diagnosed with the disease are still alive. In a clinical trial, measuring the overall survival is one way to see how well a new treatment works. Also called OS.
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City of Hope Medical Center
Lead sponsor
National Cancer Institute (NCI)
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
