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

Phase

1/2

Condition

Hemophilia

Treatment

BE-101

Clinical Study ID

NCT06611436

BE-101-01

Ages > 18
Male

Study 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.

Eligibility Criteria

Inclusion

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

Exclusion Criteria:

Pre-existing or history of specific diseases
B-Cell malignancy, EBV lymphoproliferative disease
Primary immunodeficiency disease or disorder (PIDD) or systemicimmuno-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 interventionalclinical 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

BE-101

1/2

December 04, 2024

July 31, 2027

Study 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.

Connect with a study center

University of Michigan
Ann Arbor, Michigan 48109
United States
Active - Recruiting
University of Michighan
Ann Arbor, Michigan 48109
United States
Active - Recruiting
University of Minnesota
Minneapolis, Minnesota 55455
United States
Active - Recruiting
Washington Center for Bleeding Disorders
Seattle, Washington 98101
United States
Active - Recruiting

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