Actively Recruiting
A Phase 1 Multicenter Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation
Led by BlueSphere Bio, Inc · Updated on 2026-05-05
19
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying a new cellular therapy called BSB-2002 to treat adults with relapsed or refractory acute myeloid leukemia (AML) who have a specific mutation called NPM1 and are HLA-A*02:01 positive. The trial aims to assess the safety of this genetically modified T cell therapy and to see if it can prevent the cancer from coming back. This Phase 1, open-label, non-randomized study uses an adaptive dose escalation design to find the best dose for treatment. Participants will receive a single intravenous infusion of BSB-2002 following a lymphodepletion treatment. The trial includes up to three dose escalation groups to evaluate different doses, followed by an expansion group to further study the chosen dose. All patients must have relapsed or refractory AML with the NPM1 mutation types A, D, G, or H, and meet specific genetic and clinical criteria to join. During the study, participants will be closely monitored for side effects, including serious and dose-limiting toxicities, for up to 365 days after treatment. Researchers will also track cancer relapse, survival rates, and the presence of BSB-2002 cells in the blood. Patients will provide informed consent and undergo regular assessments to ensure safety and evaluate treatment effects throughout the trial period, which lasts about one year after infusion.
CONDITIONS
Brief Title
Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients aged 18 years or older
- Diagnosis of AML per ELN criteria treated with at least two prior lines of therapy
- AML that is relapsed after previous remission or refractory to last treatment
- MRD positive by NGS for NPM1 after previously being MRD negative following last treatment
- Positive for HLA-A*02:01
- Positive for NPM1 mutation type A, D, G, or H
- Adequate venous access for apheresis or willingness to use a central line
- Willing and able to provide informed consent and follow study requirements
You will not qualify if you...
- Leukemic blast count greater than 20,000/µl unless controlled with hydroxyurea
- AML only outside the bone marrow (extramedullary only)
- Candidates for hematopoietic stem cell transplant
- Eligible to receive approved targeted therapy
- Treatment with other investigational agents within 5 half-lives before dosing
- Recent hematopoietic stem cell transplant within 3 months or with significant complications
- Other malignancy requiring treatment
- Uncontrolled bacterial, viral, or fungal infections at enrollment
- Active Hepatitis B or C infection
- HIV-1 or HIV-2 positive
- CNS involvement refractory to treatment
- Congestive heart failure NYHA class 3 or 4 or history of same without adequate heart function
- Renal insufficiency with creatinine clearance under 40 ml/min/1.73m2 or high serum creatinine
- Elevated total bilirubin, AST, or ALT beyond specified limits
- Pregnant or nursing women
- ECOG performance status greater than 2
- Ongoing treatment with chronic immunosuppressants
- Unwillingness to use effective birth control or abstinence for 12 months if fertile
- Any condition judged by investigators to interfere with participation or safety
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day for infusion; overall treatment period varies based on dose escalation cohorts
Participants receive a lymphodepletion regimen followed by a single intravenous infusion of BSB-2002 to evaluate safety and clinical activity.
1 infusion visit (in-person) following lymphodepletion regimen
Duration - Up to 365 days
Participants are monitored for safety, adverse events, relapse, cellular kinetics of BSB-2002, and overall survival for up to 365 days after treatment.
Regular follow-up visits throughout 365 days post-infusion
Trial Site Locations
Total: 1 location
1
Washington University at St Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
M
Medical Director: Nawazish Khan, BlueSphere Bio, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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