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

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

130

Participants

Timeline

Start Date

September 11, 2024

Primary Completion Date

December 17, 2032

Study Completion Date

December 17, 2032

Conditions
Hemophilia B
Interventions
DRUG

REGV131

Single dose administered via intravenous (IV) infusion before LNP1265

DRUG

LNP1265

Single dose administered via IV infusion following REGV131

Trial Locations (30)

2050

RECRUITING

Royal Prince Alfred Hospital, Haemophilia Treatment Centre, Camperdown

15006

RECRUITING

Complejo Hospitalario Universitario de A Coruna, A Coruña

19104

RECRUITING

Children's Hospital of Philadelphia (CHOP), Philadelphia

20246

RECRUITING

University Hospital Hamburg Eppendorf, Hamburg

28046

RECRUITING

Hospital Universitario La Paz, Madrid

30120

RECRUITING

Hospital Clinico Universitario Virgen De La Arrixaca, El Palmar

33011

RECRUITING

Hospital Universitario Central de Asturias, Oviedo

41013

RECRUITING

Hospital Universitario Virgen del Rocio, Seville

46026

RECRUITING

Haemostasis and Thrombosis Unit, Hospital La Fe, Valencia

48109

RECRUITING

University of Michigan, Ann Arbor

50009

RECRUITING

Hospital Universitario Miguel Servet, Zaragoza

60590

RECRUITING

University Hospital Frankfurt, Frankfurt am Main

69677

RECRUITING

Hospices Civils de Lyon, Bron

70112

RECRUITING

Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders, New Orleans

75015

RECRUITING

Hopital Necker, Paris

90007

RECRUITING

Orthopaedic Hemophilia Treatment Center, Los Angeles

90024

RECRUITING

David Geffen School of Medicine at UCLA, Los Angeles

90027

RECRUITING

Children's Hospital Los Angeles, Los Angeles

94143

RECRUITING

University California San Francisco, San Francisco

95817

RECRUITING

University of California Davis, Sacramento

06510

RECRUITING

Yale HTC, New Haven

L8N 3Z5

RECRUITING

McMaster University Medical Centre - Hamilton Health Sciences, Hamilton

08035

RECRUITING

Hospital Universitario Vall d'Hebron, Barcelona

G31 2ER

RECRUITING

Glasgow Royal Infirmary - Clinical Research Facility, Glasgow

B15 2TH

RECRUITING

Queen Elizabeth Hospital Birmingham, Birmingham

CB2 0QQ

RECRUITING

Addenbrooke's Hospital, Cambridge University Hospitals NHS FT, Cambridge

E1 2ES

RECRUITING

Pathology and Pharmacy Building, The Royal London Hospital, London

NW3 2QG

RECRUITING

Royal Free London NHS Foundation Trust, London

SE1 7EH

RECRUITING

St. Thomas' Hospital, London

W12 0HS

RECRUITING

Hammersmith Hospital Comprehensive Care Centre, London

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Intellia Therapeutics

INDUSTRY

lead

Regeneron Pharmaceuticals

INDUSTRY

NCT06379789 - 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 | Biotech Hunter | Biotech Hunter