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 Multicenter Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation

Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation (NCT07566585) is a Phase 1 interventional studying AML - Acute Myeloid Leukemia and AML With Mutated NPM1, sponsored by BlueSphere Bio, Inc. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For AML - Acute Myeloid Leukemia, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 19 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Male or female patients, ages 18 years or older, 2. AML diagnosed per ELN criteria1 which has been treated with at least two lines of therapy, 1. which is relapsed (after previously complete remission, CR, CRh or CRi), or 2. refractory (failed to achieve complete remission) to the last treatment\*, \*Primary refractory patients should have received at least two cycles of induction treatment 3. Patients who are MRD positive by NGS for NPM1 after being MRD negative following the last treatment 4. HLA-A\*02:01, 5. Positive for NPM1 mutation type A, D, G or H (see Appendix 3)2 6. Adequate venous access for apheresis or agree to use of a central line for apheresis collection, 7. Willing and able to provide willing to sign a consent form and adhere to all study requirements. Who Should NOT Join This Trial: 1. Leukemic blast count of \>20,000/μl. If the blast count can be maintained below the threshold with hydroxyurea, the patient would be eligible. 2. Patients with extramedullary only AML. 3. Patients that are candidates for hematopoietic stem cell transplant. 4. Patients that are eligible to receive an approved targeted therapy. 5. Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-2002 (day 1). 6. Subject has had hematopoietic stem cell transplant (HSCT) and has any of the following: 1. Is within 3 months of transplant; 2. Has clinically significant graft-versus-host disease requiring systemic treatment; 3. Has ≥ Grade 2 persistent non-hematological toxicity related to the transplant. 7. Other malignancy that requires treatment. 8. Uncontrolled bacterial, viral, or fungal infections at time of enrollment. 9. Active Hepatitis B or C infection. 10. Seropositive for Human weakened immune system Virus-1 or -2. 11. CNS involvement refractory to intrathecal chemotherapy and/or standard cranial- spinal radiation. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: 1. Male or female patients, ages 18 years or older, 2. AML diagnosed per ELN criteria1 which has been treated with at least two lines of therapy, 1. which is relapsed (after previously complete remission, CR, CRh or CRi), or 2. refractory (failed to achieve complete remission) to the last treatment\*, \*Primary refractory patients should have received at least two cycles of induction treatment 3. Patients who are MRD positive by NGS for NPM1 after being MRD negative following the last treatment 4. HLA-A\*02:01, 5. Positive for NPM1 mutation type A, D, G or H (see Appendix 3)2 6. Adequate venous access for apheresis or agree to use of a central line for apheresis collection, 7. Willing and able to provide informed consent and adhere to all study requirements. Exclusion Criteria: 1. Leukemic blast count of \>20,000/μl. If the blast count can be maintained below the threshold with hydroxyurea, the patient would be eligible. 2. Patients with extramedullary only AML. 3. Patients that are candidates for hematopoietic stem cell transplant. 4. Patients that are eligible to receive an approved targeted therapy. 5. Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-2002 (day 1). 6. Subject has had hematopoietic stem cell transplant (HSCT) and has any of the following: 1. Is within 3 months of transplant; 2. Has clinically significant graft-versus-host disease requiring systemic treatment; 3. Has ≥ Grade 2 persistent non-hematological toxicity related to the transplant. 7. Other malignancy that requires treatment. 8. Uncontrolled bacterial, viral, or fungal infections at time of enrollment. 9. Active Hepatitis B or C infection. 10. Seropositive for Human Immunodeficiency Virus-1 or -2. 11. CNS involvement refractory to intrathecal chemotherapy and/or standard cranial- spinal radiation. 12. 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%. 13. 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 14. Total bilirubin \> 2x upper limit of normal (unless attributed to Gilbert's Syndrome). 15. AST or ALT \> 3x upper limit of normal. 16. Pregnant or lactating women. 17. Eastern Cooperative Oncology Group (ECOG) performance status \>2. 18. Ongoing treatment with chronic immunosuppressants (e.g., cyclosporine or systemic steroids at any dose) 19. 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. 20. 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.

Treatments Being Tested

DRUG

SOC+ BSB-2002

Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime.

DRUG

SOC+BSB-2002

Patients will receive BSB-2002 as a single IV infusion at day 1 following the lymphodepletion regime.

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Washington University at St Louis

St Louis, Missouri, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07566585), the sponsor (BlueSphere Bio, Inc), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07566585 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07566585?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07566585?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07566585. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07566585. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.