RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-10
Study Completion:2027-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06611436

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)
  2. * Received ≥50 exposure days to Factor IX products preceding enrollment.
  3. * Currently receiving prophylaxis treatment
  4. * Adequate organ function and clinical labs
  5. * Able to tolerate study procedures including leukapheresis.
  1. * Pre-existing or history of specific diseases
  2. * B-Cell malignancy, EBV lymphoproliferative disease
  3. * Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression
  4. * Arterial and/or venous thromboembolic events within 2 years prior to dosing
  5. * History of anaphylaxis or nephrotic syndrome
  6. * Active infection (HIV, Hep B or C)
  7. * History of inhibitor to FIX or inhibitor
  8. * History of an allergic reaction or anaphylaxis to FIX products
  9. * Within 6 months from BE-101 administration:
  10. * Planned surgical procedure
  11. * Previously dosed with gene therapy or participated in an interventional clinical study
  12. * Planned participation in clinical trial within one year after BE-101
  13. * Administration of a vaccine within 28 days of dosing

Contacts and Locations

Study Contact

Be Biopharma
CONTACT
askhemophiliab@be.bio

Study Locations (Sites)

University of Michighan
Ann Arbor, Michigan, 48109
United States
University of Minnesota
Minneapolis, Minnesota, 55455
United States

Collaborators and Investigators

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 Registration Dates

Study Start Date2024-10
Study Completion Date2027-05

Study Record Updates

Study Start Date2024-10
Study Completion Date2027-05

Terms related to this study

Keywords Provided by Researchers

  • Hemophilia B
  • Dose Escalation

Additional Relevant MeSH Terms

  • Hemophilia B, Severe
  • Hemophilia B