BSB-2002 for Relapsed or Refractory AML with NPM1 Mutation
This study is testing a new treatment called BSB-2002 for people with acute myeloid leukemia (AML) that has come back or isn't responding to other treatments, specifically if your AML has an NPM1 mutation. BSB-2002 is a personalized cell therapy where your own infection-fighting T cells are modified to recognize and kill AML cells with the NPM1 mutation. You would receive BSB-2002 after a chemotherapy regimen of cyclophosphamide and fludarabine. The main goals are to see how safe BSB-2002 is, what side effects it might cause, and how well it works. This study is for adults aged 18 and older. The study plans to enroll 19 participants.
- Study design
- This is a dose-escalation study, meaning different doses of BSB-2002 will be tested. It is a single-arm study, involving about 19 participants.
- What's involved
- You would undergo leukapheresis (a procedure to collect your blood cells), receive cyclophosphamide and fludarabine intravenously, and then BSB-2002 intravenously. You will also have blood samples, bone marrow aspirations, bone marrow biopsies, and echocardiography tests.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your safety will be monitored for up to 365 days after treatment. The study will also track the BSB-2002 cells in your blood for up to 365 days.
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NCT07583303
BSB-2002 After Cyclophosphamide and Fludarabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation
Not Yet Recruiting
PHASE1Ages 18+InterventionalTreatmentCity of Hope Medical CenterInvestigator-initiated
~19 participants
Updated 2026-05-13 on ClinicalTrials.gov
What's tested:Biospecimen CollectionBone Marrow AspirationBone Marrow BiopsyCyclophosphamideEchocardiography TestFludarabine
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose-limiting toxicity
Measured over From the start of the infusion on day 0 through day 28
+2 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Ryotaro Nakamura · PRINCIPAL_INVESTIGATOR · City of Hope Medical Center
Who to contact
Ryotaro Nakamura
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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
Seronegative for HIV antigen (Ag)/antibody (Ab) combo and no active hepatitis C virus (HCV) and hepatitis B virus (HBV) (surface antigen negative) (performed within 6 weeks prior to leukapheresis unless otherwise stated)
If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable
Meets other institutional and federal requirements for infectious disease titer requirements
Note Infectious disease testing to be performed within 28 days prior to leukapheresis
Women of childbearing potential (WOCBP): Negative urine or serum pregnancy test (performed within 6 weeks prior to leukapheresis unless otherwise stated)
If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 12 months from the date of BSB-2002 infusion
Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only)
CRITERIA TO PROCEED WITH LEUKAPHERESIS: Research participant has signed the informed consent
CRITERIA TO PROCEED WITH LEUKAPHERESIS: Research participant must have appropriate venous access, have a central line or be willing to undergo central or temporary line placement
CRITERIA TO PROCEED WITH LEUKAPHERESIS: Biochemistry laboratory results have been reviewed for clinical significance and appropriate measures taken. Hematology assessments are expected to be out of the normal range and do not need to be assessed for clinical significance
CRITERIA TO PROCEED WITH LEUKAPHERESIS: The last dose of systemic chemotherapy must be at least 2 weeks or 5 half-lives, whichever is shorter, before the leukapheresis procedure with the following exceptions:
Steroids and vincristine are allowed up to 7 days prior to leukapheresis
Intrathecal chemotherapy is allowed up to 3 days prior to leukapheresis
Hydroxyurea is allowed up to 48 hours prior to leukapheresis
The research participant cannot be on prednisone or equivalent doses of other corticosteroids at the time of leukapheresis. Note: Topical and inhaled corticosteroids in standard doses and physiologic replacement for subjects with adrenal insufficiency are allowed
CRITERIA TO PROCEED WITH LEUKAPHERESIS: The last dose of prior targeted agents, immunotherapy or radiation must be at least 2 weeks or 5 half-lives, whichever is shorter, before the leukapheresis procedure
CRITERIA TO PROCEED WITH LEUKAPHERESIS: If the research participant has undergone prior HCT, at least 3 months must have elapsed since receiving transplant to undergo peripheral blood mononuclear cell (PBMC) collection for BSB-2002 manufacturing
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Research participant's BSB-2002 product is received by COH
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Biochemistry laboratory results have been reviewed for clinical significance and appropriate measures taken. Hematology assessments are expected to be out of the normal range and do not need to be assessed for clinical significance
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Research participant with no active CNS leukemia
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Research participant must meet the following washout criteria:
Regimen: Hydroxyurea; Required washout period: Stopped prior to start of lymphodepletion
CRITERIA TO PROCEED WITH LYMPHODEPLETION: ECOG ≤ 2
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Women of childbearing potential (WOCBP): negative urine or serum pregnancy test within 30 days prior to the start of lymphodepletion. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Research participants of reproductive potential must agree to use and utilize an adequate method of contraception throughout treatment and for at least 12 months after BSB-2002 T cell infusion
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Not requiring supplemental oxygen or mechanical ventilation, oxygen saturation 92% or higher on room air
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Not requiring pressor support, no symptomatic cardiac arrhythmias, no acute coronary syndrome, or uncontrolled hypertension
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Calculated creatinine clearance (absolute value) of ≥ 40 mL/minute
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Total bilirubin ≤ 2 times the institutional upper limit of normal (ULN)
Note: In the event a participant has elevated levels of liver enzymes possibly related to underlying disease/disease progression, the participant will still be considered eligible
CRITERIA TO PROCEED WITH LYMPHODEPLETION: ALT ≤ 3 times the institutional ULN
Note: In the event a participant has elevated levels of liver enzymes possibly related to underlying disease/disease progression, the participant will still be considered eligible
CRITERIA TO PROCEED WITH LYMPHODEPLETION: AST ≤ 3 times the institutional ULN
Note: In the event a participant has elevated levels of liver enzymes possibly related to underlying disease/disease progression, the participant will still be considered eligible
CRITERIA TO PROCEED WITH LYMPHODEPLETION: No new neurological deficits
CRITERIA TO PROCEED WITH LYMPHODEPLETION: No clinical evidence of uncontrolled active infectious process
CRITERIA TO PROCEED WITH LYMPHODEPLETION: Participants must be cytomegalovirus (CMV) negative (by polymerase chain reaction \[PCR\])
CRITERIA TO PROCEED WITH BSB-2002 INFUSION: Biochemistry laboratory results have been reviewed for clinical significance and appropriate measures taken. Hematology assessments are expected to be out of the normal range and do not need to be assessed for clinical significance
CRITERIA TO PROCEED WITH BSB-2002 INFUSION: Prohibited medications have not been administered
CRITERIA TO PROCEED WITH BSB-2002 INFUSION: ECOG ≤ 2
CRITERIA TO PROCEED WITH BSB-2002 INFUSION: Absolute lymphocyte count (ALC) \< 500/µL (0.5 × 10\^⁹/L)
Discuss with sponsor if ALC target is not achieved by day -1
CRITERIA TO PROCEED WITH BSB-2002 INFUSION: Patient must not have any medical condition that render the patient unstable, including but not limited to untreated infection, altered mental status, unstable vital signs, worsening cardiovascular status, requiring discussion with the sponsor
Exclusion
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)
What this trial measures
- Incidence of dose-limiting toxicityFrom the start of the infusion on day 0 through day 28
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)Up to day +365
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 AEsUp to day +365
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.