NCT06003387

Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies

Study Summary

The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

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Interventions

CSL222 (AAV5-hFIXco-Padua)GENETIC
Administered as a single IV infusion.

Study Locations

FacilityCityStateCountry
University of California, San Diego (UCSD)San DiegoCaliforniaUnited States
University of MichiganAnn ArborMichiganUnited States
Royal Prince Alfred HospitalCamperdownNew South WalesAustralia
Royal Brisbane HospitalHerstonQueenslandAustralia
Royal Adelaide HospitalAdelaideSouth AustraliaAustralia
The Alfred HospitalMelbourneVictoriaAustralia
McMaster University - HamiltonHamiltonOntarioCanada
Queen Mary HospitalHong KongHong Kong
Prince of Wales Hospital Chinese University of Hong KongShatinHong Kong
Sheba Medical CenterTel LitwinskyIsrael
Centro de Investigacion Clinica GRAMEL S.C.Mexico CityMexico CityMexico
King Faisal Specialist Hospital and Research CenterRiyadhSaudi Arabia
National University HospitalSingaporeSingapore
Singapore General HospitalSingaporeSingapore
Haemophilia Comprehensive Care CentreJohannesburgSouth Africa
Kyungpook National University HospitalDaeguSouth Korea
Kyung Hee University Hospital at GangdongSeoulSouth Korea
Severance Hospital, Yonsei University Health SystemSeoulSouth Korea
Tri-Service General HospitalTaipeiNeihu DistrictTaiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)Kaohsiung CitySanmin DistrictTaiwan
Changhua Christian Hospital (CCH)Chang-huaTaiwan
Taichung Veterans General Hospital -TaichungTaiwan
National Taiwan University HospitalTaipeiTaiwan
Ege University Medical FacultyBornovaTurkey (Türkiye)
Gaziantep University Sahinbey Research and Practice HospitalGaziantepTurkey (Türkiye)
Özel Acibadem Adana HastanesiSeyhanTurkey (Türkiye)

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026