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REGV131 · BEYOND-9

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

Ensayo de REGENERON PHARMACEUTICALS, INC. en Hemophilia B (del registro, en inglés).

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

130

previstos

Centros

49

Fin del objetivo primario

ago 2034

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en sep 2024.

Qué mide

Occurrence of Treatment-Emergent Adverse Events (TEAEs) (del registro, en inglés) (hasta 2 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroParticipants 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

Más ensayos de REGN

Todos los de REGN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06379789), actualizada en sep 2026.