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A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 130

Last updated 2026-05-19

No results posted yet for this study

Summary

Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the "study drug". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy.

The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it, both in the near term and over time.

The study is looking at several other research questions including:

* How much study drug is in the blood at different times
* Whether the body makes antibodies against parts of the study drug, which could make the drug less effective or could lead to side effects. Antibodies are proteins produced by the body's immune system in response to a foreign substance
* Whether the body makes antibodies against the clotting factor replacement therapy
* How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug)
* Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood

Conditions

  • Hemophilia B

Interventions

DRUG

REGV131

Administered per the protocol before LNP1265

DRUG

LNP1265

Administered per the protocol following REGV131

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-11
Primary Completion
2034-08-14
Completion
2047-08-14
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Italy
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06379789 on ClinicalTrials.gov