Clinical trial NCT06611436: BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Hemophilia B, Severe
Hemophilia B
Keywords: Hemophilia B
Dose Escalation
Lead sponsor: Be Biopharma
Sponsor class: INDUSTRY
First posted: 2024-09-25
Enrollment: 24
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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
* 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

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Adverse events (AEs) and Serious Adverse Events (SAEs)
timeFrame: 1 year post dose
Secondary outcomes:
measure: FIX Activity
timeFrame: Baseline to 1 year post dose
measure: Annualized Bleeding Rate (ABR)
timeFrame: 1 year post dose
measure: Bleeding Episodes
timeFrame: 1 year post dose
measure: Target Joints
timeFrame: 1 year post dose
measure: Exogenous FIX Concentrate
timeFrame: 1 year post dose
measure: FIX Replacement Therapy
timeFrame: 1 year post dose
measure: FIX Inhibitor
timeFrame: 1 year post dose
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Seattle
country: United States
facility: Washington Center for Bleeding Disorders
state: Washington
Study officials:
References: