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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
Sponsor: Regeneron Pharmaceuticals
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
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
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
130
Start Date
2024-09-11
Completion Date
2032-12-17
Last Updated
2026-03-20
Healthy Volunteers
No
Conditions
Interventions
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Locations (40)
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis
Sacramento, California, United States
University California San Francisco
San Francisco, California, United States
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, United States
Yale HTC
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Royal Prince Alfred Hospital, Haemophilia Treatment Centre
Camperdown, New South Wales, Australia
University of Alberta Hospital
Edmonton, Alberta, Canada
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada
McGill University Health Center (MUHC)
Montreal, Quebec, Canada
Hospices Civils de Lyon
Bron, Lyon, France
Hemostase Clinique, Institut Coeur Poumon
Lille, Nord, France
Hopital Necker
Paris, Île-de-France Region, France
University Hospital Frankfurt
Frankfurt am Main, Hesse, Germany
University Hospital Hamburg Eppendorf
Hamburg, Germany
Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy
Irccs Humanitas Research Hospital
Rozzano, Lombardy, Italy
Ospedale san Bortolo
Vicenza, Italy
Hospital Universitario Virgen del Rocio
Seville, Andalusia, Spain
Complejo Hospitalario Universitario de A Coruña (Edificio Teresa Herrera-Materno Infantil)
A Coruña, Galicia, Spain
Hospital Clinico Universitario Virgen De La Arrixaca
El Palmar, Murcia, Spain
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, Spain
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Hospital Universitario La Paz
Madrid, Spain
Haemostasis and Thrombosis Unit, Hospital La Fe
Valencia, Spain
Hospital Universitario Miguel Servet
Zaragoza, Spain
Glasgow Royal Infirmary - Clinical Research Facility
Glasgow, Scotland, United Kingdom
Queen Elizabeth Hospital Birmingham
Birmingham, West Midlands, United Kingdom
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
Cambridge, United Kingdom
Pathology and Pharmacy Building, The Royal London Hospital
London, United Kingdom
Royal Free London NHS Foundation Trust
London, United Kingdom
St. Thomas' Hospital
London, United Kingdom
Hammersmith Hospital Comprehensive Care Centre
London, United Kingdom