Actively Recruiting
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
Led by Regeneron Pharmaceuticals · Updated on 2026-04-15
130
Participants Needed
41
Research Sites
1196 weeks
Total Duration
On this page
Sponsors
R
Regeneron Pharmaceuticals
Lead Sponsor
I
Intellia Therapeutics
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official 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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of severe or moderately severe hemophilia B with Factor IX functional activity ≤2% or documented genotype for severe hemophilia B
- Currently receiving Factor IX prophylaxis treatment with prior experience
- Participation in a lead-in period of this study or a separate lead-in study for at least 6 months for bleeding rate data while on Factor IX prophylaxis
You will not qualify if you...
- History of Factor IX inhibitor on two or more occasions
- Bethesda inhibitor titer above the normal upper limit at screening
- Detectable pre-existing antibodies to the AAV8 capsid at prescreening
- Significant underlying liver disease such as cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, or hepatic encephalopathy
- Evidence of advanced liver fibrosis or significant fatty liver
- Evidence of cirrhosis or portal hypertension by abdominal ultrasound within 6 months prior to screening
- History of arterial or venous thrombo-embolic events
- History of hypersensitivity to corticosteroids or chronic corticosteroid use due to medical condition
- Previous or planned treatment with any AAV-based gene therapy other than REGV131-LNP1265 during the study period
- Other protocol-defined inclusion and exclusion criteria apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 41 locations
1
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States, 90007
Actively Recruiting
2
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90024
Actively Recruiting
3
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
4
University of California Davis
Sacramento, California, United States, 95817
Actively Recruiting
5
University California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
6
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, United States, 80045
Actively Recruiting
7
Yale HTC
New Haven, Connecticut, United States, 06510
Actively Recruiting
8
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
9
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
Actively Recruiting
10
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, United States, 70112
Actively Recruiting
11
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
12
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
13
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
14
Royal Prince Alfred Hospital, Haemophilia Treatment Centre
Camperdown, New South Wales, Australia, 2050
Actively Recruiting
15
University of Alberta Hospital
Edmonton, Alberta, Canada, T6G 2B7
Actively Recruiting
16
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada, L8N 3Z5
Actively Recruiting
17
McGill University Health Center (MUHC)
Montreal, Quebec, Canada, H4J 3A1
Actively Recruiting
18
Hospices Civils de Lyon
Bron, Lyon, France, 69677
Actively Recruiting
19
Hemostase Clinique, Institut Coeur Poumon
Lille, Nord, France, 59037
Actively Recruiting
20
Hopital Necker
Paris, Île-de-France Region, France, 75015
Actively Recruiting
21
University Hospital Frankfurt
Frankfurt am Main, Hesse, Germany, 60590
Actively Recruiting
22
University Hospital Hamburg Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
23
Careggi University Hospital
Florence, Firenze, Italy, 50134
Actively Recruiting
24
Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy, 20122
Actively Recruiting
25
Irccs Humanitas Research Hospital
Rozzano, Lombardy, Italy, 20089
Actively Recruiting
26
Ospedale san Bortolo
Vicenza, Italy, 36100
Actively Recruiting
27
Hospital Universitario Virgen del Rocio
Seville, Andalusia, Spain, 41013
Actively Recruiting
28
Complejo Hospitalario Universitario de A Coruña (Edificio Teresa Herrera-Materno Infantil)
A Coruña, Galicia, Spain, 15006
Actively Recruiting
29
Hospital Clinico Universitario Virgen De La Arrixaca
El Palmar, Murcia, Spain, 30120
Actively Recruiting
30
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, Spain, 33011
Actively Recruiting
31
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 08035
Actively Recruiting
32
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
33
Haemostasis and Thrombosis Unit, Hospital La Fe
Valencia, Spain, 46026
Actively Recruiting
34
Hospital Universitario Miguel Servet
Zaragoza, Spain, 50009
Actively Recruiting
35
Glasgow Royal Infirmary - Clinical Research Facility
Glasgow, Scotland, United Kingdom, G31 2ER
Actively Recruiting
36
Queen Elizabeth Hospital Birmingham
Birmingham, West Midlands, United Kingdom, B15 2TH
Actively Recruiting
37
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
38
Pathology and Pharmacy Building, The Royal London Hospital
London, United Kingdom, E1 2ES
Actively Recruiting
39
Royal Free London NHS Foundation Trust
London, United Kingdom, NW3 2QG
Actively Recruiting
40
St. Thomas' Hospital
London, United Kingdom, SE1 7EH
Actively Recruiting
41
Hammersmith Hospital Comprehensive Care Centre
London, United Kingdom, W12 0HS
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
7
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