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
Recruiting now · Phase 1/Phase 2
Conditions studied: Hemophilia B
In brief
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
Key facts
- Study ID
- NCT06379789
- Run by
- Regeneron Pharmaceuticals
- People needed
- 130
- Starts
- 2024-09-11
- Expected to finish
- 2047-08-14
- Last updated by the study team
- 2026-08-05
Who can join
Age: 2 and older. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States (enrolling)
- David Geffen School of Medicine at UCLA — Los Angeles, California, United States (enrolling)
- Children's Hospital Los Angeles — Los Angeles, California, United States (enrolling)
- University of California Davis — Sacramento, California, United States (enrolling)
- University California San Francisco — San Francisco, California, United States (enrolling)
- University of Colorado Hemophilia and Thrombosis Center — Aurora, Colorado, United States (enrolling)
- Yale HTC — New Haven, Connecticut, United States (enrolling)
- University of Florida — Gainesville, Florida, United States (enrolling)
- Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana, United States (enrolling)
- Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders — New Orleans, Louisiana, United States (enrolling)
- University of Michigan — Ann Arbor, Michigan, United States (enrolling)
- Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
- Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States (enrolling)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
- University of Texas Health Science Center at Houston — Houston, Texas, United States (enrolling)
- Royal Prince Alfred Hospital, Haemophilia Treatment Centre — Camperdown, New South Wales, Australia (enrolling)
- Royal Brisbane and Women's Hospital — Herston, Queensland, Australia (enrolling)
- Royal Adelaide Hospital — Adelaide, South Australia, Australia (enrolling)
- Alfred Hospital — Melbourne, Victoria, Australia (enrolling)
- University of Alberta Hospital — Edmonton, Alberta, Canada (enrolling)
- McMaster University Medical Centre - Hamilton Health Sciences — Hamilton, Ontario, Canada (enrolling)
- McGill University Health Center (MUHC) — Montreal, Quebec, Canada (enrolling)
- Hospices Civils de Lyon — Bron, Lyon, France (enrolling)
- Hemostase Clinique, Institut Coeur Poumon — Lille, Nord, France (enrolling)
- Hopital Necker — Paris, Île-de-France Region, France (enrolling)
Full record on ClinicalTrials.gov
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