Treatment of Hemophilia B with BE-101

BeCoMe-9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

This study is testing a new gene editing treatment called BE-101 to see if it can help adults with Hemophilia B by increasing their Factor IX levels and improving their condition.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: Male
• Sponsor: Be Biopharma (Industry-sponsored)
• Locations: 4 sites (Davis, California and 3 other locations)
• Trial ID: NCT06611436 on ClinicalTrials.gov

What this trial studies

The BeCoMe-9 study evaluates the safety and clinical activity of BE-101, an autologous B Cell Medicine using CRISPR/Cas9 gene editing, in adults with moderately severe or severe Hemophilia B. This Phase 1/2, multi-center, open-label, dose-escalation study aims to identify the optimal dose of BE-101 that can restore active Factor IX levels in the bloodstream. Participants will undergo leukapheresis for cell collection and receive a single intravenous dose of BE-101, followed by monitoring for safety and efficacy over approximately 52 weeks.

Who should consider this trial

Good fit: Ideal candidates are adult males aged 18 and older with moderately severe to severe Hemophilia B who have received sufficient exposure to Factor IX products.

Not a fit: Patients with a history of specific diseases or conditions that could interfere with the treatment, such as B-Cell malignancies or active infections, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of Hemophilia B by restoring normal Factor IX levels in patients.

How similar studies have performed: While gene therapy approaches for Hemophilia have shown promise, this specific application of CRISPR/Cas9 in BE-101 is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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 this trial is running

Davis, California and 3 other locations

• University of California, Davis — Davis, California, United States (Recruiting)
• University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
• University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
• Washington Center for Bleeding Disorders — Seattle, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Be Biopharma

Conditions Hemophilia B; Hemophilia B, Moderately Severe or Severe; Dose Escalation