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Gene Therapy

BE-101 for Hemophilia B

Phase 1 & 2
Recruiting
Research Sponsored by Be Biopharma
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)
* Received ≥50 exposure days to Factor IX products preceding enrollment.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post dose
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment called BE-101 for adults with moderate to severe Hemophilia B. BE-101 is given as a single IV dose and is designed to continuously produce a protein called

Who is the study for?
Adult men (18+) with moderately severe to severe Hemophilia B, currently on prophylaxis treatment, who have had over 50 days of exposure to Factor IX products. Participants must be able to undergo leukapheresis and have good organ function.
What is being tested?
BE-101 is being tested in this study. It's a gene-edited cell therapy designed for one-time infusion into adults with Hemophilia B, aiming to continuously produce the blood clotting factor they lack.
What are the potential side effects?
Potential side effects are not detailed but may include reactions related to CRISPR/Cas9 gene editing, immune responses due to autologous cell therapy, or complications from intravenous administration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a man over 18 with severe Hemophilia B.
Select...
I have used Factor IX products for more than 50 days before joining.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post dose for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse events (AEs) and Serious Adverse Events (SAEs)
Secondary study objectives
Annualized Bleeding Rate (ABR)
Bleeding Episodes
Exogenous FIX Concentrate
+4 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose EscalationExperimental Treatment1 Intervention
Group II: Part 1 - Dose Escalation, Dose Level 3Experimental Treatment1 Intervention
Group III: Part 1 - Dose Escalation, Dose Level 2Experimental Treatment1 Intervention
Group IV: Part 1 - Dose Escalation, Dose Level 1Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

Be BiopharmaLead Sponsor
~16 spots leftby May 2027
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