BSB-2002 After Cyclophosphamide and Fludarabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation

Starting soon · Phase 1

Conditions studied: Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia

In brief

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.

Key facts

Study ID

NCT07583303

Run by

City of Hope Medical Center

People needed

19

Starts

2026-12-14

Expected to finish

2027-04-16

Last updated by the study team

2026-05-13

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Eastern Cooperative Oncology Group (ECOG) ≤ 2
• Adequate venous access for apheresis or agree to use of a central line for apheresis collection
• AML diagnosed per ELN criteria which has been treated with at least two lines of therapy, and meet one of these 3 criteria:
• Which is relapsed (after previously complete remission, CR, CRh or CRi), OR
• Are refractory (failed to achieve complete remission) to the last treatment
• Primary refractory patients should have received at least two cycles of induction treatment, OR
• MRD positive (at least 1% leukemic blasts in blood or bone marrow) after being MRD negative following the last treatment
• Positive for NPM1 mutation type A, D, G or H. The confirmation for NPM1 mutation must be performed by NGS within 3 months from enrollment
• If participant has had prior hematopoietic cell transplant (HCT), all 3 of the following must be met:
• More than 3 months from transplant at the time of enrollment
• No clinically significant graft-versus (vs)-host disease requiring systemic treatment
• Any non-hematological toxicity related to transplant has resolved to < grade 2 per Common Terminology Criteria for Adverse Events (CTCAE)
• Total bilirubin ≤ 2 X upper limit of normal (ULN) (unless has Gilbert's disease or related to leukemia involving the liver) (performed within 6 weeks prior to leukapheresis unless otherwise stated)
• Aspartate aminotransferase (AST) ≤ 3.0 x ULN (unless related to leukemia involving the liver) (performed within 6 weeks prior to leukapheresis unless otherwise stated)
• Alanine aminotransferase (ALT) ≤ 3.0 x ULN (unless related to leukemia involving the liver) (performed within 6 weeks prior to leukapheresis unless otherwise stated)
• Creatinine clearance of ≥ 40 mL/min per 24 hour urine test or the Cockcroft-Gault formula, or serum creatinine ≤ 1.6mg/dL and the participant is not on hemodialysis (performed within 6 weeks prior to leukapheresis unless otherwise stated)
• No history of congestive heart failure class III or IV New York Heart Association (NYHA) OR left ventricular ejection fraction (LVEF) ≥ 45% up to 90 days before enrollment
• If able to perform pulmonary function tests: Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and diffusion capacity of the lung for carbon monoxide (DLCO) (diffusion capacity) ≥ 50% of predicted (corrected for hemoglobin)
• Note To be performed up to 90 days before enrollment
• If unable to perform pulmonary function tests: Oxygen (O2) saturation > 92% on room air
• Note To be performed up to 90 days before enrollment

You may not qualify if…

• 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
• Eligible to receive an approved targeted therapy
• Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-2002 (day 0)
• Ongoing treatment with chronic immunosuppressants (e.g., cyclosporine or systemic steroids at any dose)
• Unstable cardiac disease as defined by one of the following:
• Cardiac events such as myocardial infarction (MI) within the past 6 months
• NYHA (New York Heart Association) heart failure class III-IV
• Uncontrolled atrial fibrillation or hypertension
• Uncontrolled bacterial, viral, or fungal infections at time of enrollment
• Other active malignancy that requires treatment. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
• Females only: Pregnant or breastfeeding
• Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures or interference with study participation or data interpretation
• Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)

Where it is running

• City of Hope Medical Center — Duarte, California, United States

Full record on ClinicalTrials.gov

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