RECRUITING

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

Description

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.

Conditions

Hemophilia B, SevereHemophilia B
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Related Conditions:

Hemophilia B, SevereHemophilia B

Study Overview

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Study Details

Study overview

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.

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

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

Condition
Hemophilia B, Severe
Intervention / Treatment

-

Contacts and Locations

Ann Arbor

University of Michighan, Ann Arbor, Michigan, United States, 48109

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55455

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Be Biopharma,

Study Record Dates

2027-05