AML - Acute Myeloid LeukemiaPhase 1NCT07566585
Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
A Phase 1 study testing SOC+ BSB-2002 and SOC+BSB-2002 in people with AML - Acute Myeloid Leukemia and AML With Mutated NPM1, run by BlueSphere Bio, Inc at 1 site in United States.
Trial summary
The goal of this clinical trial is to test BSB-2002 which is a new type of cellular therapy to treat blood cancer (AML). It will evaluate the safety of BSB-2002 and also determine whether it works to prevent relapse of your cancer. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML - Acute Myeloid Leukemia, AML With Mutated NPM1, AML, Adult Recurrent
- What is being tested?
- SOC+ BSB-2002 and SOC+BSB-2002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Apr 2026to 8 Sep 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Male or female patients, ages 18 years or older,
- AML diagnosed per ELN criteria1 which has been treated with at least two lines of therapy,
- which is relapsed (after previously complete remission, CR, CRh or CRi), or
- refractory (failed to achieve complete remission) to the last treatment*, *Primary refractory patients should have received at least two cycles of induction treatment
- Patients who are MRD positive by NGS for NPM1 after being MRD negative following the last treatment
- HLA-A*02:01,
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- Positive for NPM1 mutation type A, D, G or H (see Appendix 3)2
- Adequate venous access for apheresis or agree to use of a central line for apheresis collection,
- Willing and able to provide informed consent and adhere to all study requirements.
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.
- Patients with extramedullary only AML.
- Patients that are candidates for hematopoietic stem cell transplant.
- Patients that are eligible to receive an approved targeted therapy.
- Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-2002 (day 1).
- Subject has had hematopoietic stem cell transplant (HSCT) and has any of the following:
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- Is within 3 months of transplant;
- Has clinically significant graft-versus-host disease requiring systemic treatment;
- Has ≥ Grade 2 persistent non-hematological toxicity related to the transplant.
- Other malignancy that requires treatment.
- Uncontrolled bacterial, viral, or fungal infections at time of enrollment.
- Active Hepatitis B or C infection.
- Seropositive for Human Immunodeficiency Virus-1 or -2.
- CNS involvement refractory to intrathecal chemotherapy and/or standard cranial- spinal radiation.
- Subject has congestive heart failure NYHA class 3 or 4, or subject with a history of congestive heart failure NYHA class 3 or 4 in the past, unless an echocardiogram performed within 3 months prior to study entry results in a left ventricular ejection fraction that is ≥ 45%.
- Renal insufficiency, with estimated creatinine clearance of < 40 ml/min/1.73m2 by the Cockcroft-Gault equation with adjustment if the weight is ≥ 125% of ideal body weight OR inadequate renal function defined by serum creatinine > 1.6 mg/dL
- Total bilirubin > 2x upper limit of normal (unless attributed to Gilbert's Syndrome).
- AST or ALT > 3x upper limit of normal.
- Pregnant or lactating women.
- Eastern Cooperative Oncology Group (ECOG) performance status >2.
- Ongoing treatment with chronic immunosuppressants (e.g., cyclosporine or systemic steroids at any dose)
- Women of childbearing potential (WOCBP) and men who are fertile and are unwilling to use an effective birth control method or abstinence for 12 months. Effective forms of birth control are listed in the Contraception section.
- Any condition, in the judgement of the Investigator, that would interfere with study participation, pose a significant risk to the patient, or interfere with study data interpretation.
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University at St Louis
St Louis, Missouri, 63110
About this study
Official title: A Phase 1 Multicenter Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
The goal of this clinical trial is to test BSB-2002 which is a new type of cellular therapy to treat blood cancer (AML). It will evaluate the safety of BSB-2002 and also determine whether it works to prevent relapse of your cancer.
Read the detailed description
This is a Phase I, multicenter, open-label, non-randomized study to characterize the safety and clinical activity of BSB-2002, a genetically modified autologous T cell product incorporating an HLA-A*02:01-restricted mutant NPM1-directed T cell receptor (TCR), administered to patients with relapsed or refractory acute myeloid leukemia (AML). Enrolled patients must be HLA-A*02:01+ and positive for the NPM1 mutation which produces the alternative amino acid sequence CLAVEEVSL (Type A, D, G or H).
The study is an adaptive dose escalation design with up to 3 cohorts to evaluate single doses of BSB-2002, employing the 3+3 design.
- Being tested
- SOC+ BSB-2002, SOC+BSB-2002
- Keywords
- TCR, T-cell therapy
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SOC+ BSB-2002Drug
Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime.
SOC+BSB-2002Drug
Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime.
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation Cohorts | Experimental | AML HLA-A*02:01 and Positive for NPM1 mutation type A, D, G or H patients with an identified will be dosed in dose escalation cohorts Receives: Drug: SOC+BSB-2002 |
| Expansion Cohort | Experimental | Once the maximum tolerated dose (MTD) or promising dose is reached additional AML HLA-A*02:01 and Positive for NPM1 mutation type A, D, G or H patients will be enrolled in the expansion cohort. Receives: Drug: SOC+ BSB-2002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with dose-limiting toxicity, adverse events (AEs) and serious AEs (SAEs)
Incidence of dose-limiting toxicity, frequency and severity of adverse events (AEs) and serious AEs (SAEs)
Measured: 365 days
Secondary outcomes (3)
Number of Patients with Relapse
Presence of malignant cells in marrow (>5%), peripheral blood (>1%), or extramedullary sites by histopathology after achievement of CR, CRh or CRi any time after study treatment.
Measured: 365 days
Cellular kinetics of BSB-2002 in peripheral blood
Quantitation of BSB-2002 (copies per μL of genomic DNA)
Measured: 365 days
Overall survival
Defined as the time from treatment to death due to any cause
Measured: Through 365 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2026
- Study start21 Apr 2026
- Primary completion8 Sep 2026
- Study completion8 Sep 2026
Why it stopped: Company Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML - Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Non-randomised
- Intervention model
- Sequential
- Model description
- Adaptive dose escalation design with 1 to 3 cohorts to evaluate single doses of BSB-2002. Three to six patients will be enrolle in each cohort.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- BlueSphere Bio, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07566585
- Sponsor's protocol ID
- BSB2002-CL-102
- Status last verified
- Sep 2026
- First submitted
- 21 Apr 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Multicenter Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
- ClinicalTrials.gov ID
- NCT07566585
- Acronym / sponsor ID
- BSB2002-CL-102
- Keywords
- TCR, T-cell therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Company Decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- BlueSphere Bio, IncIndustry (pharma, biotech, devices)
- Study start
- 21 Apr 2026 (actual)
- Primary completion
- 8 Sep 2026 (actual)
- Study completion
- 8 Sep 2026 (actual)
- First posted
- 5 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to test BSB-2002 which is a new type of cellular therapy to treat blood cancer (AML). It will evaluate the safety of BSB-2002 and also determine whether it works to prevent relapse of your cancer.
Detailed description
This is a Phase I, multicenter, open-label, non-randomized study to characterize the safety and clinical activity of BSB-2002, a genetically modified autologous T cell product incorporating an HLA-A*02:01-restricted mutant NPM1-directed T cell receptor (TCR), administered to patients with relapsed or refractory acute myeloid leukemia (AML). Enrolled patients must be HLA-A*02:01+ and positive for the NPM1 mutation which produces the alternative amino acid sequence CLAVEEVSL (Type A, D, G or H).
The study is an adaptive dose escalation design with up to 3 cohorts to evaluate single doses of BSB-2002, employing the 3+3 design.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Adaptive dose escalation design with 1 to 3 cohorts to evaluate single doses of BSB-2002. Three to six patients will be enrolle in each cohort.
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation Cohorts | Experimental | Drug: SOC+BSB-2002AML HLA-A*02:01 and Positive for NPM1 mutation type A, D, G or H patients with an identified will be dosed in dose escalation cohorts |
| Expansion Cohort | Experimental | Drug: SOC+ BSB-2002Once the maximum tolerated dose (MTD) or promising dose is reached additional AML HLA-A*02:01 and Positive for NPM1 mutation type A, D, G or H patients will be enrolled in the expansion cohort. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female patients, ages 18 years or older,
- AML diagnosed per ELN criteria1 which has been treated with at least two lines of therapy,
- Patients who are MRD positive by NGS for NPM1 after being MRD negative following the last treatment
- HLA-A*02:01,
- Positive for NPM1 mutation type A, D, G or H (see Appendix 3)2
Key exclusion criteria (23 listed)
- Leukemic blast count of >20,000/μl. If the blast count can be maintained below the threshold with hydroxyurea, the patient would be eligible.
- Patients with extramedullary only AML.
- Patients that are candidates for hematopoietic stem cell transplant.
- Patients that are eligible to receive an approved targeted therapy.
- Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-2002 (day 1).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SOC+ BSB-2002Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime. | Drug | Expansion Cohort |
| SOC+BSB-2002Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime. | Drug | Dose Escalation Cohorts |
Primary outcome
Number of participants with dose-limiting toxicity, adverse events (AEs) and serious AEs (SAEs)
Time frame: 365 days
Incidence of dose-limiting toxicity, frequency and severity of adverse events (AEs) and serious AEs (SAEs)
3 secondary outcomes
Number of Patients with Relapse
Time frame: 365 days
Cellular kinetics of BSB-2002 in peripheral blood
Time frame: 365 days
Overall survival
Time frame: Through 365 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SOC+ BSB-2002 and SOC+BSB-2002 in people with AML - Acute Myeloid Leukemia and AML With Mutated NPM1, run by BlueSphere Bio, Inc 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 9 inclusion and 23 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: withdrawn, as of the sponsor's last update on 10 Sep 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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with dose-limiting toxicity, adverse events (AEs) and serious AEs (SAEs) (time frame: 365 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Sep 2026.