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NCT06379789 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study of Hemophilia B, sponsored by Regeneron Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 130
- Enrollment target
- 20
- Study locations
NCT06379789: Recruiting Phase 1 study of Hemophilia B, sponsored by Regeneron Pharmaceuticals.
NCT06379789 is a Phase 1 study of Hemophilia B that is actively recruiting participants, run by Regeneron Pharmaceuticals. The registered enrollment target is 130 participants, above the 90-participant average among 20 other Hemophilia B trials with a reported enrollment target (44% higher). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06379789, a Phase 1 study of Hemophilia B, is actively recruiting participants, sponsored by Regeneron Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 130 participants
- Enrollment target
- 20
- Study locations
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. 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 quality of life is affected by hemophilia B and if it changes after taking study drug * How joint health is affected by hemophilia B and if it changes after taking study drug * How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug * 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
Primary Outcome
Part 1, 2B, and 2C
Conditions Studied
Interventions
- DRUG REGV131
- DRUG LNP1265
Study Locations (20)
California
- Orthopaedic Hemophilia Treatment Center - Los Angeles
- David Geffen School of Medicine at UCLA - Los Angeles
- Children's Hospital Los Angeles - Los Angeles
- University of California Davis - Sacramento
- University California San Francisco - San Francisco
Colorado
- University of Colorado Hemophilia and Thrombosis Center - Aurora
Connecticut
- Yale HTC - New Haven
Florida
- University of Florida - Gainesville
Indiana
- Indiana Hemophilia and Thrombosis Center - Indianapolis
Louisiana
- Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders - New Orleans
Michigan
- University of Michigan - Ann Arbor
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 130 participants |
| Start Date | 2024-09-11 |
| Est. Completion | 2032-12-17 |
| Phase | Phase 1 |
What NCT06379789 shows while recruiting
NCT06379789 is an interventional study that assigns participants to a tested intervention. The registered 130 participants enrollment target is mid-sized for trials with a published cap, above the 90-participant average among 20 other Hemophilia B trials with a reported enrollment target (44% higher).
The record links to 1 condition, with Hemophilia B appearing as the primary indexed condition, and to 2 interventions - of which REGV131 is the first listed.
NCT06379789 names 20 study sites across 16 states, led by California, Colorado, Connecticut.
Frequently Asked Questions
What is clinical trial NCT06379789 about?
NCT06379789 is a clinical study titled "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". 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 ...
What is the current status of trial NCT06379789?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 130 participants. The study started on 2024-09-11. Estimated completion is 2032-12-17.
What conditions does trial NCT06379789 study?
This clinical trial studies the following conditions: Hemophilia B.
What interventions are being tested in trial NCT06379789?
The interventions under investigation include: REGV131 (DRUG), LNP1265 (DRUG).
Who is sponsoring clinical trial NCT06379789?
This trial is sponsored by Regeneron Pharmaceuticals, which has 280 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06379789 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, Florida, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06379789's enrollment target sits among peer trials
130 6th of 20 higher than 15 of 20 other Hemophilia B trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia B trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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