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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT06379789
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Hemophilia B

Eligibility Criteria

Sex
MALE
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • REGV131 — DRUG
    Administered per the protocol before LNP1265
  • LNP1265 — DRUG
    Administered per the protocol following REGV131

Study Details

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 Dates

Start date
Sep 11, 2024
Status verified
May 2026
Primary completion
Aug 14, 2034
Completion
Aug 14, 2047

Study Design

Enrollment
130 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: Cohort 1 Dose Escalation for RDE
    Starting dose to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
  • Experimental: Part 1: Cohort 2 Dose Escalation for RDE
    Dose 2 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
  • Experimental: Part 1: Cohort 3 Dose Escalation for RDE
    Dose 3 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
  • Experimental: Part 1: Cohort 4 Dose Escalation for RDE
    Dose 4 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
  • Experimental: Part 2: Dose Expansion A
    Participants ≥18 Years of Age will receive the RDE of REGV131-LNP1265 determined by Part 1
  • Experimental: Part 2: Dose Expansion B
    Participants ≥12 to \<18 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1
  • Experimental: Part 2: Dose Expansion C
    Participants ≥2 to \<12 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1

Primary Outcome Measure

Occurrence of Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to 2 Years ]

Central Contacts

Locations (14)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027-
David Geffen School of Medicine at UCLALos AngelesCalifornia90024-
Orthopaedic Hemophilia Treatment CenterLos AngelesCalifornia90007-
University of California DavisSacramentoCalifornia95817-
University California San FranciscoSan FranciscoCalifornia94143-
University of Colorado Hemophilia and Thrombosis CenterAuroraColorado80045-
Yale HTCNew HavenConnecticut06510-
University of FloridaGainesvilleFlorida32610-
Indiana Hemophilia and Thrombosis CenterIndianapolisIndiana46260-
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting DisordersNew OrleansLouisiana70112-
University of MichiganAnn ArborMichigan48109-
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvania19104-
Vanderbilt University Medical CenterNashvilleTennessee37232-

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles· Los Angeles, CADavid Geffen School of Medicine at UCLA· Los Angeles, CAOrthopaedic Hemophilia Treatment Center· Los Angeles, CAUniversity of California Davis· Sacramento, CAUniversity California San Francisco· San Francisco, CAUniversity of Colorado Hemophilia and Thrombosis Center· Aurora, CO

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