Actively Recruiting
BeCoMe-9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for Treatment of Adults With Moderately Severe or Severe Hemophilia B
Led by Be Biopharma · Updated on 2025-10-20
24
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating BE-101, a new gene-editing treatment for adults with moderately severe or severe Hemophilia B, a condition caused by Factor IX deficiency. This Phase 1/2 study aims to assess the safety and clinical activity of a single intravenous dose of BE-101, which uses CRISPR/Cas9 gene editing to insert the human FIX gene into a person's own B cells to restore meaningful levels of active Factor IX. The trial includes dose escalation and expansion phases to identify and confirm the optimal dose. The study is divided into two parts. Part 1 uses a stepwise dose escalation to find the dose that achieves desired FIX activity 28 days after infusion. Part 2a expands on this with up to six adult participants receiving the selected dose to further evaluate safety and activity. Up to 24 participants in total will receive an intravenous infusion of BE-101. Before treatment, participants undergo leukapheresis to collect cells for manufacturing BE-101. Additional cohorts may include adolescents and redosing, depending on Part 1 data. Participants will be involved in a screening period, leukapheresis collection, BE-101 infusion, and approximately 52 weeks of follow-up monitoring. Researchers will assess safety by tracking adverse events for one year, alongside measuring Factor IX activity, bleeding rates, joint health, and use of Factor IX replacement therapy. Clinical labs and other evaluations will support ongoing safety and effectiveness monitoring throughout the study period.
CONDITIONS
Brief Title
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult males aged 18 years or older with moderately severe to severe Hemophilia B (Factor IX deficiency)
- Received at least 50 exposure days to Factor IX products before enrollment
- Currently receiving prophylaxis treatment
- Adequate organ function and clinical laboratory results
- Able to tolerate leukapheresis and other study procedures
You will not qualify if you...
- History or presence of B-Cell malignancy or EBV lymphoproliferative disease
- Primary immunodeficiency disease or systemic immunosuppression
- Arterial or venous thromboembolic events within 2 years before dosing
- History of anaphylaxis or nephrotic syndrome
- Active infection with HIV, Hepatitis B or C
- History of inhibitor to Factor IX or inhibitor presence
- History of allergic reaction or anaphylaxis to Factor IX products
- Planned surgery within 6 months after BE-101 administration
- Prior treatment with gene therapy
- Participation in another interventional study or receipt of investigational drug within 30 days or five half-lives before consent
- Planned participation in another clinical trial within one year after BE-101
- Use of investigational agents or vaccines within 28 days before leukapheresis and BE-101 dosing
- Other protocol-defined inclusion/exclusion criteria may apply
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 - Up to 4 weeks
Participants undergo leukapheresis collection to support BE-101 manufacturing prior to receiving treatment.
1 to 2 visits depending on manufacturing schedule
Duration - Single dose with monitoring for up to 52 weeks post-infusion
Participants receive an IV infusion of BE-101 at escalating doses or the selected optimal dose to treat Hemophilia B.
Multiple visits for treatment administration and follow-up within 52 weeks
Duration - Up to 52 weeks post treatment
Participants are monitored for safety, clinical activity, and adverse events after receiving BE-101 treatment.
Regular visits for safety and clinical assessments
Trial Site Locations
Total: 4 locations
1
University of California, Davis
Davis, California, United States, 95616
Actively Recruiting
2
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
3
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
4
Washington Center for Bleeding Disorders
Seattle, Washington, United States, 98101
Actively Recruiting
Research Team
B
Be Biopharma
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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