BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
- Registration Number
- NCT06611436
- Lead Sponsor
- Be Biopharma
- Brief 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.
- Detailed 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. Additional cohorts for adolescents and redosing for participants in Part 1 of the study will occur following data availability of Part 1.
Up to 24 participants will be enrolled across Part 1 (up to 18) and Part 2a (up to 6). Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing. Following administration, participants will be monitored for safety and clinical activity. The total duration of study participation is approximately 52 weeks post IV administration of BE-101.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 24
- 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.
-
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
-
Within 6 months from BE-101 administration:
- Planned surgical procedure
- Previously dosed with gene therapy or participated in an interventional clinical study
-
Planned participation in clinical trial within one year after BE-101
-
Administration of a vaccine within 28 days of dosing
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part 1 - Dose Escalation, Dose Level 1 BE-101 - Part 1 - Dose Escalation, Dose Level 2 BE-101 - Part 1 - Dose Escalation, Dose Level 3 BE-101 - Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation BE-101 -
- Primary Outcome Measures
Name Time Method Adverse events (AEs) and Serious Adverse Events (SAEs) 1 year post dose Incidence of AEs and SAEs
- Secondary Outcome Measures
Name Time Method FIX Activity Baseline to 1 year post dose Change From Baseline in FIX Activity
Annualized Bleeding Rate (ABR) 1 year post dose ABR (spontaneous and traumatic) for all bleeds. Annualized bleeding rates overall and by severity will be presented.
Bleeding Episodes 1 year post dose Number of bleeding episodes (total, spontaneous, and traumatic). Bleeding episodes will be summarized by subject and overall, and will include number, duration, and severity.
Target Joints 1 year post dose Number of target joints. A target joint is defined as a major joint (e.g. hip, elbow, wrist, shoulder, knee, ankle) into which repeated bleeding occurs (frequency of 3 or more bleeding episodes into the same joint in a consecutive 12 week period) and with symptoms of pre-existing target joint involvement (eg, synovitis, persistent swelling, effusion, limitation of range of motion).
Exogenous FIX Concentrate 1 year post dose Number of infusions of exogenous FIX concentrate
FIX Replacement Therapy 1 year post dose Total consumption of exogenous FIX replacement therapy (IU) after BE-101 administration
FIX Inhibitor 1 year post dose FIX inhibitor development post BE-101 administration
Trial Locations
- Locations (3)
Washington Center for Bleeding Disorders
🇺🇸Seattle, Washington, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States