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

NCT07566585 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BSB2002-CL-102
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

AML - Acute Myeloid Leukemia; AML With Mutated NPM1; AML, Adult Recurrent

Keywords

TCR; T-cell therapy

Outcome Measures

Primary Outcomes (1)

• 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)

  365 days

Secondary Outcomes (3)

• Number of Patients with Relapse

  365 days

• Cellular kinetics of BSB-2002 in peripheral blood

  365 days

• Overall survival

  Through 365 days

Other Outcomes (2)

• Malignant Cell presence detected by Molecular MRD Methods

  365 days

• Cellular kinetics of serum cytokines and biomarkers

  365 days

Study Arms (2)

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

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.

Drug: SOC+ BSB-2002

Interventions

SOC+ BSB-2002 DRUG

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

Expansion Cohort

SOC+BSB-2002 DRUG

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

Dose Escalation Cohorts

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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%.

Sponsors & Collaborators

• BlueSphere Bio, Inc: lead

Study Sites (1)

Washington University at St Louis

St Louis, Missouri, 63110, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Medical Director: Nawazish Khan, BlueSphere Bio, MD

CONTACT

252-347-4938; nkhan@bluespherebio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: April 21, 2026
• First Posted: May 5, 2026
• Study Start: April 21, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)