BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
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
Be Biopharma
24 participants
Dec 4, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria5
- 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 Criteria13
- 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
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Interventions
IV Infusion of BE-101 dose from Dose Level 1
IV Infusion of BE-101 dose from Dose Level 2
IV Infusion of BE-101 dose from Dose Level 3
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Locations(4)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06611436