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
Public ClinicalTrials.gov record NCT06379789. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT06379789
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 130 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 10, 2024
- Primary completion
- Aug 13, 2034
- Completion
- Aug 13, 2047
- Last update posted
- Aug 4, 2026
2024 – 2047
United States locations
- U.S. sites
- 15
- U.S. states
- 11
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Orthopaedic Hemophilia Treatment Center | Los Angeles | California | 90007 | Recruiting |
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90024 | Recruiting |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | Recruiting |
| University of California Davis | Sacramento | California | 95817 | Recruiting |
| University California San Francisco | San Francisco | California | 94143 | Recruiting |
| University of Colorado Hemophilia and Thrombosis Center | Aurora | Colorado | 80045 | Recruiting |
| Yale HTC | New Haven | Connecticut | 06510 | Recruiting |
| University of Florida | Gainesville | Florida | 32610 | Recruiting |
| Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana | 46260 | Recruiting |
| Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders | New Orleans | Louisiana | 70112 | Recruiting |
| University of Michigan | Ann Arbor | Michigan | 48109 | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Recruiting |
| Children's Hospital of Philadelphia (CHOP) | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | Recruiting |
| University of Texas Health Science Center at Houston | Houston | Texas | 77030 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 31 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06379789, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 4, 2026 · Synced Sep 7, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06379789 live on ClinicalTrials.gov.