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 · Phase 1, Phase 2 · NCT06379789
Conditions: Hemophilia B
At a glance
- Research payments reported, 2025
- $340,867
- U.S. investigators named
- 6
- Sites paid
- 6
- Planned or actual enrollment
- 130
About the trial
- Sponsor
- Regeneron Pharmaceuticals (with Intellia Therapeutics)
- Tested
- REGV131 (drug), LNP1265 (drug)
- Dates
- Started 2024-09-11; completion 2047-08-14
- Results posted
- Not yet
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 que…
From ClinicalTrials.gov, last updated 2026-10-21. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Regeneron Pharmaceuticals, Inc. reported $340,867 in research payments for this study in 2025 (98 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Noah Federman, M.D. Pediatric Hematology & Oncology · Los Angeles, CA
- Adam Giermasz, M.D., PHD Hematology & Oncology · Sacramento, CA
- Guy A. Young, M.D. Pediatric Hematology & Oncology · Los Angeles, CA
- Maissaa Janbain Hematology & Oncology · New Orleans, LA
- Steven W. Pipe, MD Pediatric Hematology & Oncology · Ann Arbor, MI
- Benjamin J. Samelson-jones, MD Pediatric Hematology & Oncology · Philadephia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of Michigan Health System, MI $110,031
- Children's Hospital of Philadelphia, PA $57,518
- Ronald Reagan UCLA Medical Center, CA $53,446
- University of California Davis Medical Center, CA $47,298
- The Administrators of the Tulane Educational Fund $31,730
- Children's Hosp of Los Angeles, CA $27,775
- $13,069
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.