Skip to content
Bored Studies
RecruitingPhase 1/Phase 2

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

Regeneron Pharmaceuticals · with Intellia Therapeutics · NCT06379789

View Study & Apply (opens in a new tab)

Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 2+ · Men only
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
130
US sites recruiting
17
Study start
September 11, 2024
Record last updated
October 21, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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

Being studied: REGV131; LNP1265

Conditions: Hemophilia B

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Key Inclusion Criteria

  • Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B
  • Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol
  • Participation in the lead-in period of this interventional study OR a separate lead-in study (R0000-HEMB-2187 [NCT05568459]) for at least 6 months for ABR data while taking FIX prophylaxis, as defined in the protocol

Key Exclusion Criteria

  • History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions
  • Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening
  • Detectable pre-existing antibodies to the AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable)
  • Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy
  • Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol
  • Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit
  • History of arterial or venous thrombo-embolic events, as defined in the protocol
  • History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids
  • Previously received any AAV gene-based therapy or intends to receive approved or investigational AAV-based gene therapy other than REGV131-LNP1265 during the study period

NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply

Recruiting US locations

Study contacts (from the official record)

  • Clinical Trials Administrator · 844-734-6643 · clinicaltrials@regeneron.com
All phase 1 studies in Arizona →
A Study to Investigate the Safety and Effectiveness of a… (NCT06379789) | Bored Studies