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

Recruiting now · Phase 1

Conditions studied: AML - Acute Myeloid Leukemia, AML With Mutated NPM1, AML, Adult Recurrent

In brief

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.

Key facts

Study ID: NCT07566585
Run by: BlueSphere Bio, Inc
People needed: 19
Starts: 2026-04-21
Expected to finish: 2027-09-01
Last updated by the study team: 2026-05-05

Who can join

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

You may qualify if…

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,
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.

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.
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:
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.

Where it is running

Washington University at St Louis — St Louis, Missouri, United States (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.