BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
Purpose
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, Severe
- Hemophilia B
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- Male
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
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Experimental Part 1 - Dose Escalation, Dose Level 1 |
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Experimental Part 1 - Dose Escalation, Dose Level 2 |
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Experimental Part 1 - Dose Escalation, Dose Level 3 |
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Experimental Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation |
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Recruiting Locations
Ann Arbor, Michigan 48109
Minneapolis, Minnesota 55455
Seattle, Washington 98101
More Details
- NCT ID
- NCT06611436
- Status
- Recruiting
- Sponsor
- Be Biopharma
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.