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
← catalyst calendarNCT06379789 · readout in 2,917 d
Sponsored by Regeneron Pharmaceuticals (industry) · REGN — their whole pipeline →. With Intellia Therapeutics.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
130estimated
Sites
46
Countries
Australia, Canada, France +5
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-09-11 | actual | When the study began enrolling |
| Primary completion | 2034-08-14 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2047-08-14 | estimated | The whole study's end, after follow-up |
| First posted | 2024-04-23 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-08-05 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Hemophilia B
Interventions
- Drug: REGV131
- Drug: LNP1265
Primary outcomes
what the primary-completion date above is the date OFOccurrence of Treatment-Emergent Adverse Events (TEAEs)
measured Up to 2 Years
Severity of TEAEs
measured Up to 2 Years
Coagulation Factor IX (FIX) functional activity measured using the chromogenic substrate assay
measured Up to 2 Years
Change in FIX functional activity in plasma, measured using the chromogenic substrate assay
measured Up to 2 Years
Annualized Bleeding Rate (ABR) following sustained FIX functional activity among participants receiving the RDE
measured Up to 2 Years
Occurrence of Serious Adverse Events (SAEs)
measured Through Long Term Follow Up (LTFU), Up to 15 Years
Severity of SAEs
measured Through LTFU, Up to 15 Years
Occurrence of Adverse Event of Special Interests (AESIs)
measured Through LTFU, Up to 15 Years
Severity of AESIs
measured Through LTFU, Up to 15 Years
Occurrence of clinically meaningful Adverse Events (AEs)
measured Through LTFU, Up to 15 Years
Severity of clinically meaningful AEs
measured Through LTFU, Up to 15 Years
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