A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B

Status: Recruiting
Location: See all (29) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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. 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 quality of life is affected by hemophilia B and if it changes after taking study drug * How joint health is affected by hemophilia B and if it changes after taking study drug * How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug * 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

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
United States
California
Children's Hospital Los Angeles
RECRUITING
Los Angeles
David Geffen School of Medicine at UCLA
RECRUITING
Los Angeles
Orthopaedic Hemophilia Treatment Center
RECRUITING
Los Angeles
University of California Davis
RECRUITING
Sacramento
University California San Francisco
RECRUITING
San Francisco
Connecticut
Yale HTC
RECRUITING
New Haven
Louisiana
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
RECRUITING
New Orleans
Michigan
University of Michigan
RECRUITING
Ann Arbor
Pennsylvania
Children's Hospital of Philadelphia (CHOP)
RECRUITING
Philadelphia
Other Locations
Australia
Royal Prince Alfred Hospital, Haemophilia Treatment Centre
RECRUITING
Camperdown
Canada
McMaster University Medical Centre - Hamilton Health Sciences
RECRUITING
Hamilton
France
Hospices Civils de Lyon
RECRUITING
Bron
Hopital Necker
RECRUITING
Paris
Germany
University Hospital Frankfurt
RECRUITING
Frankfurt Am Main
University Hospital Hamburg Eppendorf
RECRUITING
Hamburg
Spain
Complejo Hospitalario Universitario de A Coruna
RECRUITING
A Coruña
Hospital Clinico Universitario Virgen De La Arrixaca
RECRUITING
El Palmar
Hospital Universitario La Paz
RECRUITING
Madrid
Hospital Universitario Central de Asturias
RECRUITING
Oviedo
Hospital Universitario Virgen del Rocio
RECRUITING
Seville
Haemostasis and Thrombosis Unit, Hospital La Fe
RECRUITING
Valencia
Hospital Universitario Miguel Servet
RECRUITING
Zaragoza
United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING
Birmingham
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
RECRUITING
Cambridge
Glasgow Royal Infirmary - Clinical Research Facility
RECRUITING
Glasgow
Hammersmith Hospital Comprehensive Care Centre
RECRUITING
London
Pathology and Pharmacy Building, The Royal London Hospital
RECRUITING
London
Royal Free London NHS Foundation Trust
RECRUITING
London
St. Thomas' Hospital
RECRUITING
London
Contact Information
Primary
Clinical Trials Administrator
clinicaltrials@regeneron.com
844-734-6643
Time Frame
Start Date: 2024-09-11
Estimated Completion Date: 2032-12-17
Participants
Target number of participants: 130
Treatments
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
Related Therapeutic Areas
Sponsors
Collaborators: Intellia Therapeutics
Leads: Regeneron Pharmaceuticals

This content was sourced from clinicaltrials.gov