BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Stopped early · Phase 1/Phase 2

Conditions studied: Hemophilia B, Hemophilia B, Moderately Severe or Severe

In brief

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.

Key facts

Study ID

NCT06611436

Run by

Be Biopharma

People needed

5

Starts

2024-12-04

Expected to finish

2026-05-29

Last updated by the study team

2026-06-15

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify if…

• 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.

Where it is running

• University of California, Davis — Davis, California, United States
• Georgetown University — Washington D.C., District of Columbia, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington Center for Bleeding Disorders — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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