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

June 12, 2026 updated by: Be Biopharma

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Davis, California, United States, 95616
        • University of California, Davis
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20057
        • Georgetown University
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Washington
      • Seattle, Washington, United States, 98101
        • Washington Center for Bleeding Disorders

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part 1 - Dose Escalation, Dose Level 1
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 1
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 2
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 3
Drug: BE-101
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Experimental: Part 1 - Dose Escalation, Dose Level 2
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 1
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 2
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 3
Drug: BE-101
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Experimental: Part 1 - Dose Escalation, Dose Level 3
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 1
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 2
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 3
Drug: BE-101
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Experimental: Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 1
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 2
Drug: BE-101
IV Infusion of BE-101 dose from Dose Level 3
Drug: BE-101
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse events (AEs) and Serious Adverse Events (SAEs)
Time Frame: 1 year post dose
Incidence of AEs and SAEs
1 year post dose

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
FIX Activity
Time Frame: Baseline to 1 year post dose
Change From Baseline in FIX Activity
Baseline to 1 year post dose
Bleeding Episodes
Time Frame: 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.
1 year post dose
Target Joints
Time Frame: 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).
1 year post dose
Exogenous FIX Concentrate
Time Frame: 1 year post dose
Number of infusions of exogenous FIX concentrate
1 year post dose
FIX Replacement Therapy
Time Frame: 1 year post dose
Total consumption of exogenous FIX replacement therapy (IU) after BE-101 administration
1 year post dose
FIX Inhibitor
Time Frame: 1 year post dose
FIX inhibitor development post BE-101 administration
1 year post dose
Annualized Bleed Rate (ABR)
Time Frame: 1 year post dose
ABR (spontaneous and traumatic) for all bleeds. Annualized bleed rates overall and by severity will be presented.
1 year post dose
FIX Antigen Concentration
Time Frame: Baseline to 1 year post dose
Change From Baseline in FIX Antigen concentration
Baseline to 1 year post dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Be Biopharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 4, 2024

Primary Completion (Actual)

May 29, 2026

Study Completion (Actual)

May 29, 2026

Study Registration Dates

First Submitted

September 18, 2024

First Submitted That Met QC Criteria

September 20, 2024

First Posted (Actual)

September 25, 2024

Study Record Updates

Last Update Posted (Actual)

June 15, 2026

Last Update Submitted That Met QC Criteria

June 12, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BE-101-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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