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
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.
Description
The study includes 2 distinct parts: Part 1 and Part 2. In Part 1, an ascending-dose design will be utilized to enable evaluation of increasing doses in a stepwise manner. The objective for this dose escalation is to identify the dose of BE-101 required to achieve desired FIX activity 28 days after infusion. Upon identification of a safe and efficacious dose in Part 1, an expansion phase (Part 2) will initiate. The initial cohort in the Part 2 expansion (Part 2a) phase will include up to 6 adult participants to further characterize the safety and activity of BE-101 at the selected dose. Additi…
Eligibility
- Age range
- 18+ years
- Sex
- Male
- Healthy volunteers
- No
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 year…
Interventions
- DrugBE-101
IV Infusion of BE-101 dose from Dose Level 1
- DrugBE-101
IV Infusion of BE-101 dose from Dose Level 2
- DrugBE-101
IV Infusion of BE-101 dose from Dose Level 3
- DrugBE-101
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Locations (4)
- University of California, DavisDavis, California
- University of MichiganAnn Arbor, Michigan
- University of MinnesotaMinneapolis, Minnesota
- Washington Center for Bleeding DisordersSeattle, Washington