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AML, Adult RecurrentPhase 1/2NCT06704152

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

A Phase 1/2 study testing SOC + BSB-1001 Dose Escalation Cohort and SOC+BSB-1001 Expansion Dose in people with AML, Adult Recurrent and ALL, Recurrent, Adult, run by BlueSphere Bio, Inc at 6 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
AML, Adult Recurrent, ALL, Recurrent, Adult, MDS
What is being tested?
SOC + BSB-1001 Dose Escalation Cohort and SOC+BSB-1001 Expansion Dose
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
2 participants
Where
6 sitesUnited States
Study dates
4 Feb 2025to 8 Sep 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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.
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  • 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%.
  • 13. Inadequate organ function as defined in protocol.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • The Ohio State University

    Columbus, Ohio, 43210

  • City of Hope National Medical Center

    Duarte, California, 91010

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • Washington University at St Louis

    St Louis, Missouri, 63110

  • Moffitt Cancer Center

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase 1/2a Multicenter Ascending Dose Study to Evaluate the Safety of HA-1 Minor Histocompatibility Antigen-Reactive TCR-Modified T Cells (BSB-1001) in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS

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.

Read the detailed description

This is a first-in-human, multicenter, open-label, dose-finding study for the evaluation of an HA-1 minor histocompatibility antigen (miHA)-reactive TCR-modified T cell product (BSB-1001) derived from an HLA-matched allogenic donor, in patients with AML, ALL or MDS undergoing an HLA-matched alloHSCT who are at a high risk for relapse post-HSCT. BSB-1001 targets the HLA-A*02:01-restricted HA-1 miHA.

Enrolled patients must be HLA-A*02:01 and HA-1-positive (H/H or H/R), with an identified HLA-matched, HA-1-negative (R/R) donor. Patients will undergo one of the following myeloablative conditioning regimens, according to standard institutional procedures, which include either fludarabine+thiotepa+total body irradiation, or busulfan+ melphalan+ fludarabine. After conditioning is completed, patients will receive the CD34-selected alloHSCT followed by BSB-1001 on day 0, without any prophylactic immunosuppression.

The study is an adaptive dose escalation design with 1 to 3 cohorts to evaluate single doses of BSB-1001. Three to six patients will be enrolled in each cohort and enrolled patients will be followed until completion of the study.

If the maximum tolerated dose (MTD) is reached or if a dose is deemed promising, the Sponsor may determine to either cease enrollment or open an expansion cohort at the desired dose level. The optional expansion part of the study is planned to include approximately 20 additional AML patients at the recommended dose.

Being tested
SOC + BSB-1001 Dose Escalation Cohort, SOC+BSB-1001 Expansion Dose
Keywords
TCR, T-cell therapy
Medical subject headings
Leukemia, Myeloid, Acute, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SOC + BSB-1001 Dose Escalation CohortDrug

    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).

  • SOC+BSB-1001 Expansion DoseDrug

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

Study groups in NCT06704152
Study groupRoleWhat they receive
Dose Escalation CohortsExperimentalAML, 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
Receives: Drug: SOC + BSB-1001 Dose Escalation Cohort
BSB-1001 Expansion DoseExperimentalOnce 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.
Receives: Drug: SOC+BSB-1001 Expansion Dose

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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])

    Measured: 365 days

  2. Cellular kinetics of BSB-1001 in peripheral blood

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

    Measured: 365 days

Secondary outcomes (10)

  1. Number of patients with relapse

    Presence of malignant cells in marrow (>5%), peripheral blood (>1%), or extramedullary sites by histopathology after achievement of CR, CRh or CRi at any time after HSCT

    Measured: Through 365 days post HSCT

  2. Incidence of Grades II-IV acute GVHD

    Acute GVHD will be graded and assessed for response based on the MAGIC consortium scoring system

    Measured: Through 100 days post HSCT

  3. Incidence of Grades III-IV acute GVHD

    Acute GVHD will be graded and assessed for response based on the MAGIC consortium scoring system

    Measured: Through 100 days post HSCT

  4. Time to neutrophil engraftment

    Time to the first of 3 consecutive days of absolute neutrophil counts ≥ 500/µL

    Measured: Through 365 days post HSCT

  5. Time to platelet engraftment

    Time to ≥ 50,000/µL platelets for the first of 3 consecutive days without transfusion

    Measured: Through 365 days post HSCT

  6. Incidence of moderate to severe chronic GVHD

    Moderate-to-severe chronic GVHD graded according to NIH scale

    Measured: Through 365 days post HSCT

  7. Overall survival

    Defined as the time from treatment to death due to any cause

    Measured: Through 365 days

  8. GVHD-free, relapse-free survival (GFRS)

    Defined as time to any of the following events: 1) Grade III-IV aGVHD; 2) chronic GVHD requiring systemic immunosuppression; 3) primary malignancy relapse; or 4) death

    Measured: Through 365 days post HSCT

  9. GVHD-free survival (GFS)

    Defined as time to any of the following events: 1) GVHD event or 2) death from any cause

    Measured: Through 365 days post HSCT

  10. Incidence of systemic infections

    Defined as infections with a severity of Grades 3 to 5 (as graded by CTCAE v 5.0 or higher) which require treatment with systemic anti-infectives

    Measured: Through 365 days post HSCT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Nov 2024
  2. Study start4 Feb 2025
  3. Primary completion8 Sep 2026
  4. Study completion8 Sep 2026

Why it stopped: Company Decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
2 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06704152
Sponsor's protocol ID
BSB1001-CL-101
Status last verified
Sep 2026
First submitted
22 Nov 2024
Last updated
10 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing SOC + BSB-1001 Dose Escalation Cohort and SOC+BSB-1001 Expansion Dose in people with AML, Adult Recurrent and ALL, Recurrent, Adult, run by BlueSphere Bio, Inc at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Company Decision). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with treatment-emergent adverse events (TEAEs), including SAEs, GVHD and dose-limiting toxicities (time frame: 365 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06704152.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.

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