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Gene Therapy

Gene Therapy for Hemophilia B (BEYOND-9 Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol

Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or <0.02 IU/mL) or documented genotype known to produce severe hemophilia B

Timeline

Screening 3 weeks

Treatment Varies

Follow Up over 52 weeks; weeks 26 to 78 post-regv131-lnp1265 dosing

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying a gene insertion therapy called REGV131-LNP1265 for people with hemophilia B caused by a genetic mutation in the Factor 9 gene. The therapy aims to help the

Who is the study for?

This trial is for pediatric, adolescent, and adult patients with severe or moderately severe hemophilia B. Participants must have a confirmed diagnosis with Factor IX activity ≤2% or a genotype causing severe hemophilia B. They should be currently on FIX prophylaxis and have prior experience with FIX therapy.

What is being tested?

The study tests REGV131-LNP1265, an experimental gene insertion therapy designed to help the body produce clotting factor long-term in people with hemophilia B. It aims to find a safe dose by monitoring side effects and how it affects quality of life, joint health, bleeding events frequency, and need for replacement therapy.

What are the potential side effects?

Potential side effects include the body's immune response creating antibodies against the study drug or clotting factors which could reduce effectiveness or cause reactions. The exact side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am currently on FIX prophylaxis and have used FIX therapy before.

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I have severe or moderately severe hemophilia B.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ over 52 weeks; weeks 26 to 78 post-regv131-lnp1265 dosing

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~over 52 weeks; weeks 26 to 78 post-regv131-lnp1265 dosing

This trial's timeline: 3 weeks for screening, Varies for treatment, and over 52 weeks; weeks 26 to 78 post-regv131-lnp1265 dosing for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Annualized bleeding rate (ABR) following sustained FIX functional activity among participants receiving the RDE

Change in FIX functional activity in plasma, measured using the chromogenic substrate assay

Coagulation Factor IX (FIX) functional activity measured using the chromogenic substrate assay

+1 more

Secondary study objectives

ABR following sustained FIX functional activity among participants receiving the RDE

Change in FIX functional activity in plasma measured using the chromogenic substrate assay

Detection of vector DNA in blood

+9 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

7Treatment groups

Experimental Treatment

Group I: Part 2: Dose Expansion CExperimental Treatment2 Interventions

Participants ≥2 to \<12 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1

Group II: Part 2: Dose Expansion BExperimental Treatment2 Interventions

Participants ≥12 to \<18 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1

Group III: Part 2: Dose Expansion AExperimental Treatment2 Interventions

Participants ≥18 Years of Age will receive the RDE of REGV131-LNP1265 determined by Part 1

Group IV: Part 1: Cohort 4 Dose Escalation for RDEExperimental Treatment2 Interventions

Dose 4 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE

Group V: Part 1: Cohort 3 Dose Escalation for RDEExperimental Treatment2 Interventions

Dose 3 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE

Group VI: Part 1: Cohort 2 Dose Escalation for RDEExperimental Treatment2 Interventions

Dose 2 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE

Group VII: Part 1: Cohort 1 Dose Escalation for RDEExperimental Treatment2 Interventions

Starting dose to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE

0 / 2Eligibility criteria met