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

Be Biopharma

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Hemophilia B; Hemophilia B, Moderately Severe or Severe

Treatments: Drug: BE-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT06611436

BE-101-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 24 estimated patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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24 participants in 4 patient groups

Part 1 - Dose Escalation, Dose Level 1

Experimental group

Treatment:

Drug: BE-101

Drug: BE-101

Drug: BE-101

Drug: BE-101

Part 1 - Dose Escalation, Dose Level 2

Experimental group

Treatment:

Drug: BE-101

Drug: BE-101

Drug: BE-101

Drug: BE-101

Part 1 - Dose Escalation, Dose Level 3

Experimental group

Treatment:

Drug: BE-101

Drug: BE-101

Drug: BE-101

Drug: BE-101

Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation

Experimental group

Treatment:

Drug: BE-101

Drug: BE-101

Drug: BE-101

Drug: BE-101