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Recurrent Acute Myeloid LeukemiaPhase 1NCT07583303

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

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Recurrent Acute Myeloid Leukemia and Refractory Acute Myeloid Leukemia, run by City of Hope Medical Center at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
1 siteUnited States
Study dates
14 Dec 2026to 16 Apr 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 69Show fewer
  • 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

Who cannot join

  • 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)
Show all 16Show fewer
  • 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)

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site

  • City of Hope Medical Center

    Duarte, California, 91010

    Ryotaro Nakamura, RNakamura@coh.org

    Ryotaro Nakamura

Other trials in United States

About this study

Official title: A Phase 1 Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. Evaluate the safety of BSB-2002.

SECONDARY OBJECTIVES:

  1. Cellular kinetics of BSB-2002 in peripheral blood through day 365. II. Evaluate the efficacy of BSB-2002 based on IIa. Percentage of patients with complete remission (CR; including CR with measurable residual disease negative [CRMRD-], CR, CR with incomplete hematologic recovery [CRi], CR with partial hematologic recovery [CRh]) or partial remission (PR) as determined by the investigator according to the European LeukemiaNet (ELN) criteria for AML; IIb. Overall survival; IIc. 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.

EXPLORATORY OBJECTIVES:

  1. Assess molecular minimal residual disease (MRD) as measured by mutation specific next generation sequencing (NGS).

II. Evaluate inflammatory cytokines and other potential biomarkers.

OUTLINE: This is a dose-escalation study of BSB-2002 in combination with cyclophosphamide and fludarabine.

Patients undergo leukapheresis between days -45 and -21 for manufacturing of BSB-2002. Patients then receive cyclophosphamide intravenously (IV) and fludarabine IV on days -5 to -3 followed by BSB-2002 IV over 30 minutes on day 0 in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo blood sample collection as well as bone marrow aspiration and possible biopsy throughout the study. Patients also undergo echocardiography during screening.

After completion of study treatment, patients are followed up at days 4, 7, 10, 14, 21, 28, 56, 90, 180, 270, and 365 and then yearly for up to 15 years.

Being tested
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Cyclophosphamide, Echocardiography Test
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518

    Given IV

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo echocardiography

  • FludarabineDrug

    Also called Fluradosa

    Given IV

  • LeukapheresisProcedure or surgery

    Also called Leukocyte Adsorptive Apheresis, Leukocytopheresis, Therapeutic Leukopheresis, White Blood Cell Reduction Apheresis

    Undergo leukapheresis

  • Single Agent TherapyDrug

    Also called Drug Monotherapy, Monotherapy, Single Agent Treatment, Single Drug Therapy

    Given BSB-2002 IV

Study groups in NCT07583303
Study groupRoleWhat they receive
Treatment (cyclophosphamide, fludarabine, BSB-2002)ExperimentalPatients undergo leukapheresis between days -45 and -21 for manufacturing of BSB-2002. Patients then receive cyclophosphamide IV and fludarabine IV on days -5 to -3 followed by BSB-2002 IV over 30 minutes on day 0 in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo blood sample collection as well as bone marrow aspiration and possible biopsy throughout the study. Patients also undergo echocardiography during screening.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Cyclophosphamide; Procedure: Echocardiography Test; Drug: Fludarabine; Procedure: Leukapheresis; Drug: Single Agent Therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: From the start of the infusion on day 0 through day 28

  2. 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.

    Measured: Up to day +365

  3. 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.

    Measured: Up to day +365

Secondary outcomes (3)

  1. 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.

    Measured: Up to day +365

  2. 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.

    Measured: From first tumor assessment at which the overall response was recorded as CR/CRi / CRi, CRh until documented relapse, or death from any cause, whichever occurred first, assessed up to 15 years

  3. 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.

    Measured: Up to 15 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 May 2026
  2. Study start14 Dec 2026 (estimated)
  3. Primary completion16 Apr 2027 (estimated)
  4. Study completion16 Apr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
19 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07583303
Sponsor's protocol ID
25704
Other IDs
NCI-2026-02922, 25704, P30CA033572
Status last verified
May 2026
First submitted
29 Apr 2026
Last updated
13 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Recurrent Acute Myeloid Leukemia and Refractory Acute Myeloid Leukemia, run by City of Hope Medical Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 69 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of dose-limiting toxicity (time frame: From the start of the infusion on day 0 through day 28). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07583303.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.

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