Actively Recruiting
BeCoMe-9: A Clinical Study of BE-101 for the 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
134 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.
CONDITIONS
Official 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 (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)
- Received ≥50 exposure days to Factor IX products preceding enrollment.
- Currently receiving prophylaxis treatment
- Adequate organ function and clinical labs
- Able to tolerate study procedures including leukapheresis.
You will not qualify if you...
-
Pre-existing or history of specific diseases
- B-Cell malignancy, EBV lymphoproliferative disease
- Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression
- Arterial and/or venous thromboembolic events within 2 years prior to dosing
- History of anaphylaxis or nephrotic syndrome
- Active infection (HIV, Hep B or C)
-
History of inhibitor to FIX or inhibitor
-
History of an allergic reaction or anaphylaxis to FIX products
-
Planned surgical procedure within 6 months from BE-101 administration
-
Previously dosed with gene therapy
-
Participated in an interventional study and/or received an interventional study drug within 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 and for the duration of the study
-
Planned participation in clinical trial within one year after BE-101
-
Administration of an investigational agent or vaccine within 28 days of leukapheresis and dosing of BE-101
Other protocol-defined inclusion/exclusion criteria may apply.
AI-Screening
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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
CONTACT
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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