Actively Recruiting
A Phase 1/2a Study Evaluating Safety of BSB-1001 TCR-Modified T Cells in Patients Undergoing HLA-Matched Allogenic Stem Cell Transplant for AML, ALL, or MDS
Led by BlueSphere Bio, Inc · Updated on 2025-09-18
38
Participants Needed
6
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating BSB-1001, a new type of cellular therapy, in patients with blood cancers such as AML, ALL, and MDS who are undergoing an HLA-matched allogenic hematopoietic stem cell transplant (alloHSCT). This first-in-human study aims to assess the safety of BSB-1001 and determine if it can help prevent cancer relapse. The trial focuses on patients at high risk of relapse after transplant and involves a dose-finding design with multiple cohorts. Participants will receive one of several doses of BSB-1001, which is a TCR-modified T cell product targeting the HA-1 minor histocompatibility antigen. The treatment is given as a single intravenous infusion on the day of stem cell transplant (day 0), following a myeloablative conditioning regimen and CD34-selected alloHSCT. The study includes dose escalation cohorts to find the maximum tolerated dose, and an optional expansion phase enrolling additional AML patients at the recommended dose. During the study, participants will be closely monitored for treatment-emergent adverse events, graft-versus-host disease (GVHD), and dose-limiting toxicities for up to 365 days. Researchers will also measure cell kinetics of BSB-1001 in the blood and track outcomes such as relapse, survival, engraftment times, infections, and GVHD incidence. Follow-up assessments will include clinical evaluations and laboratory tests to ensure safety and effectiveness over the study period.
CONDITIONS
Brief Title
BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients aged 18 to 70 years undergoing allogeneic hematopoietic stem cell transplant (alloHCT).
- Diagnosed with high-risk AML treated with at least two lines of therapy, refractory or relapsed, or meeting specific remission or disease criteria.
- Diagnosed with ALL with abnormal lymphoblasts 5% to 25% in bone marrow or persistent disease markers.
- Diagnosed with high or very high-risk MDS after at least one line of therapy including hypomethylating agents.
- Positive for HLA-A*02:01 and HA-1 (H/H or H/R).
- Suitable for approved conditioning regimens as defined in the protocol.
- Have an identified HLA-matched, HA-1-negative (R/R) donor with 10/10 match related or unrelated donor.
You will not qualify if you...
- Weight over 100 kg.
- Prior history of allogeneic stem cell transplantation.
- Autologous stem cell transplantation within 1 year prior to planned BSB-1001 dosing.
- Previous genetically engineered CAR-T therapy within 2 years of screening except for certain ALL patients.
- Treatment with other investigational agents within 5 half-lives of BSB-1001 dosing.
- History of checkpoint inhibitor therapy within 3 months of transplant.
- Other malignancy with life expectancy less than 1 year.
- Pregnant or lactating women.
- Uncontrolled bacterial, viral, or fungal infections at enrollment.
- Past or current viral infections as defined in the protocol.
- CNS involvement refractory to intrathecal chemotherapy or cranial-spinal radiation.
- Karnofsky Performance Score below 60%.
- Inadequate organ function as defined in protocol.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Varies per institutional protocol
Participants undergo a myeloablative conditioning regimen according to standard institutional procedures before transplantation.
Multiple visits as per routine care
Duration - 1 day
Participants receive a CD34-selected allogenic hematopoietic stem cell transplant followed by a single intravenous infusion of BSB-1001 on day 0 without prophylactic immunosuppression.
1 treatment day (in-person)
Duration - Up to 12 months
Participants are monitored for safety, adverse events, and effectiveness outcomes including graft-versus-host disease, relapse, and survival for up to 365 days post-transplant.
Regular visits throughout 12 months of follow-up
Trial Site Locations
Total: 6 locations
1
City of Hope National Medical Center
Duarte, California, United States, 91010
Actively Recruiting
2
Moffitt Cancer Center
Tampa, Florida, United States, 33612
Actively Recruiting
3
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
4
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
5
Washington University at St Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
6
The Ohio State University
Columbus, Ohio, United States, 43210
Actively Recruiting
Research Team
M
Medical Director: Nawazish Khan, MD, BlueSphere Bio
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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