Tests a gene insertion therapy for hemophilia B in children and adults
Tests a gene insertion therapy's safety in people with hemophilia B.
Plain-language title generated from the registry record
Registry title: 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
Key facts
- Where
- Ann Arbor, Aurora, Columbus, Dallas, Gainesville, Houston, Indianapolis, Los Angeles, Nashville, New Haven, New Orleans, Philadelphia, Phoenix, Sacramento, and San Francisco, United StatesSee the 17 sites
- Phase
- Phase 1/2
Where in United States
- University of MichiganAnn Arbor, United StatesTaking people
- University of Colorado Hemophilia and Thrombosis CenterAurora, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- Children's Medical Center DallasDallas, United StatesTaking people
- University of FloridaGainesville, United StatesTaking people
- University of Texas Health Science Center at HoustonHouston, United StatesTaking people
- Indiana Hemophilia and Thrombosis CenterIndianapolis, United StatesTaking people
- Children's Hospital Los AngelesLos Angeles, United StatesTaking people
- David Geffen School of Medicine at UCLALos Angeles, United StatesTaking people
- Orthopaedic Hemophilia Treatment CenterLos Angeles, United StatesTaking people
- Vanderbilt University Medical CenterNashville, United StatesTaking people
- Yale HTCNew Haven, United StatesTaking people
- Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting DisordersNew Orleans, United StatesTaking people
- Children's Hospital of Philadelphia (CHOP)Philadelphia, United StatesTaking people
- Phoenix Children s HospitalPhoenix, United StatesTaking people
- University of California - DavisSacramento, United StatesTaking people
- University California San FranciscoSan Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Regeneron Pharmaceuticals
- Trial code
- NCT06379789EU 2023-507260-40-00
- Record updated
- 21 October 2026
- Original description
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.Read full original descriptionShow less
A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in 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 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
Inclusion: 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; Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol; Participation in the lead-in period of this interventional study OR a separate lead-in study (R0000-HEMB-2187 [NCT05568459]) for at least 6 months for ABR data while taking FIX prophylaxis, as defined in the protocol.
Exclusion: Detectable pre-existing antibodies to the AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable); Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit; Previously received any AAV gene-based therapy or intends to receive approved or investigational AAV-based gene therapy other than REGV131-LNP1265 during the study period; NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply; Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy; History of arterial or venous thrombo-embolic events, as defined in the protocol; History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids; History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions; Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening; Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol.