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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 · View on ClinicalTrials.gov ↗
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
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06379789
The ClinicalTrials.gov registry entry for NCT06379789 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 130 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Regeneron Pharmaceuticals, which has 290 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06379789 reports 20 study locations spanning 16 distinct geographic areas — top geographies include California, Colorado, Connecticut. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06379789?
The interventions under investigation include: REGV131 (DRUG), LNP1265 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06379789?
This trial is sponsored by Regeneron Pharmaceuticals, which has 290 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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