BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS

NCT06704152 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BSB1001-CL-101
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 6

Brief Summary

The goal of this clinical trial is to test BSB-1001 which is a new type of cellular therapy to treat blood cancers (AML, ALL and MDS). It will evaluate the safety of BSB-1001 and also determine whether it works to prevent relapse of your cancer.

Conditions

AML, Adult Recurrent; ALL, Recurrent, Adult; MDS

Keywords

TCR, T-cell therapy

Outcome Measures

Primary Outcomes (2)

• Number of participants with treatment-emergent adverse events (TEAEs), including SAEs, GVHD and dose-limiting toxicities

  Incidence of TEAEs (per Common Terminology Criteria for Adverse Events \[CTCAE\])

  365 days

• Cellular kinetics of BSB-1001 in peripheral blood

  Quantitation of BSB-1001 (copies per μL of genomic DNA)

  365 days

Secondary Outcomes (10)

• Number of patients with relapse

  Through 365 days post HSCT

• Incidence of Grades II-IV acute GVHD

  Through 100 days post HSCT

• Incidence of Grades III-IV acute GVHD

  Through 100 days post HSCT

• Time to neutrophil engraftment

  Through 365 days post HSCT

• Time to platelet engraftment

  Through 365 days post HSCT

Study Arms (2)

Dose Escalation Cohorts

EXPERIMENTAL

AML, ALL and MDS HLA-A\*02:01 and HA-1-positive (H/H or H/R) patients with an identified HLA-matched, HA-1-negative (R/R) donor will be dosed in dose escalation cohorts

Drug: SOC + BSB-1001 Dose Escalation Cohort

BSB-1001 Expansion Dose

EXPERIMENTAL

Once the maximum tolerated dose (MTD) or promising dose is reached additional AML HLA-A\*02:01 and HA-1-positive (H/H or H/R) patients with an identified HLA-matched, HA-1-negative (R/R) donor will be enrolled in the expansion cohort.

Drug: SOC+BSB-1001 Expansion Dose

Interventions

SOC + BSB-1001 Dose Escalation Cohort DRUG

Patients will receive BSB-1001 a single intravenous (IV) infusion on day 0 following the infusion of the CD34-allo hematopoietic stem cell transplant (HCT).

Dose Escalation Cohorts

SOC+BSB-1001 Expansion Dose DRUG

Patients will receive BSB-1001 a single intravenous (IV) infusion on day 0 following the infusion of the CD34-allo hematopoietic cell transplant (HCT).

BSB-1001 Expansion Dose

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients, ages 18 - 70 years inclusive, undergoing alloHCT.
• Any of the following high-risk hematologic malignancies:
• AML diagnosed which has been treated with at least two lines of therapy\* Refractory or relapsed (CR, CRh or CRi,), including myeloblasts up to 25% OR MRD positive OR persistent disease-defining cytogenetic abnormality OR MRD-negative, but with high-risk disease For patients in remission meeting criteria a, consolidation regimens would be considered another line of therapy of eligibility purposes
• ALL which has been with abnormal lymphoblasts ≥5% and up to 25% in bone marrow OR persistent disease-defining cytogenetic abnormality or MRD positive
• MDS after at least one line of therapy, which includes hypomethylating agent(s) and must be high or very high risk by Revised International Prognostic Scoring System (IPSS-R), monosomy, or complex karyotype or TP53 mutation.
• In the expansion phase AML patients diagnosed which has been treated with at least two lines of therapy, and refractory or relapsed (CR, CRh or CRi,), including myeloblasts up to 25% OR MRD positive OR persistent disease-defining cytogenetic abnormality OR MRD-negative, but with high- risk disease
• HLA-A\*02:01 AND HA-1 positive (either H/H or H/R).
• Suitable for one of the approved conditioning regimens as defined in the protocol.
• Patient must have an identified donor that is HA 1-negative with 10/10 matched related or unrelated donor

You may not qualify if:

• Weight \> 100 kg.
• Prior history of allogeneic stem cell transplantation
• Prior history of autologous stem cell transplantation within 1 year prior to the planned dosing of BSB-1001 (day 0)
• Previous genetically engineered chimeric antigen receptor T Cell therapy (CAR-T), approved or investigational, within 2 years of screening, with the exception of patients with ALL previously treated with an autologous CAR-T product.
• Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-1001 (day 0).
• History of treatment with checkpoint inhibitor therapy within 3 months of transplantation.
• Other malignancy with life expectancy \< 1year.
• Pregnant or lactating women.
• Uncontrolled bacterial, viral, or fungal infections at time of enrollment.
• Past or current viral infections as defined in the protocol.
• CNS involvement refractory to intrathecal chemotherapy and/or standard cranial- spinal radiation. 12 Karnofsky Performance Score \< 60%.
• \. Inadequate organ function as defined in protocol.

Sponsors & Collaborators

• BlueSphere Bio, Inc: lead

Study Sites (6)

City of Hope National Medical Center

Duarte, California, 91010, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Washington University at St Louis

St Louis, Missouri, 63110, United States

RECRUITING

The Ohio State University

Columbus, Ohio, 43210, 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, MD, BlueSphere Bio

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

Study Record Dates

• First Submitted: November 22, 2024
• First Posted: November 25, 2024
• Study Start: February 4, 2025
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): March 1, 2029
• Last Updated: September 18, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)