NCT05768386

A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial

Study Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.

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Interventions

No interventions listed.

Study Locations

FacilityCityStateCountry
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment CenterLos AngelesCaliforniaUnited States
UC Davis Hemophilia Treatment CenterSacramentoCaliforniaUnited States
University of California San Diego, Hemophilia & Thrombosis Treatment CenterSan DiegoCaliforniaUnited States
University of California San Francisco - Moffit Hospital, Outpatient Hematology ClinicSan FranciscoCaliforniaUnited States
UCN Hemophilia and Thrombosis CenterShandonCaliforniaUnited States
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical CampusAuroraColoradoUnited States
St Joseph's Children's Hospital, Center for Bleeding and Clotting DisordersTampaFloridaUnited States
University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer CenterLouisvilleKentuckyUnited States
University of Michigan, Pediatric Hematology and OncologyAnn ArborMichiganUnited States
Children's Hospital of Michigan, Hemophilia and Thrombosis CenterDetroitMichiganUnited States
Washington University School of Medicine, Department of Pediatrics, Division of Hematology/OncologySt LouisMissouriUnited States
Nationwide Children's HospitalColumbusOhioUnited States
Hemophilia Center of Western PennsylvaniaPittsburghPennsylvaniaUnited States
Royal Prince Alfred Hospital, Institute of HaematologySydneyNew South WalesAustralia
Royal Brisbane and Women's HospitalHerstonQueenslandAustralia
Royal Adelaide HospitalAdelaideSouth AustraliaAustralia
The Alfred HospitalMelbourneVictoriaAustralia
Fiona Stanley HospitalMurdochWestern AustraliaAustralia
University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular DiseaseLeuvenBelgium
Holy Spirit Hematology and Hemotherapy CenterVitóriaEspírito SantoBrazil
Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating HemocenterRio de JaneiroRio de JaneiroBrazil
Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)CampinasSão PauloBrazil
Sao Paulo University Clinical HospitalSão PauloSão PauloBrazil
CHRU Lille Hospital Center Heart-Lung InstituteLilleFrance
CHU La Timone - La Timone Children's HospitalMarseilleFrance
University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care CenterBonnNorth Rhine-WestphaliaGermany
Vivantes Hospital in FriedrichshainBerlinState of BerlinGermany
Chaim Sheba Medical Center, National Institute of Hemophilia and CoagulationRamat GanIsrael
Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"MilanItaly
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care CenterJohannesburgSouth Africa
Kyung Hee University Hospital at GangdongSeoulSouth Korea
University Hospital Virgen del Rocio (HUVR), Hematology PediatricsSevilleAndalusiaSpain
Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and ThrombosisA CoruñaGaliciaSpain
Addenbrooke's Hospital, Hemophilia and Thrombophilia CenterCambridgeUnited Kingdom
Glasgow Royal Infirmary, Department of HematologyGlasgowUnited Kingdom
Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental MedicineLondonUnited Kingdom
Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia CentreLondonUnited Kingdom
Royal London Hospital, Barts and the London Hemophilia CenterLondonUnited Kingdom
St Thomas' Hospital, Centre for Hemophilia, Hemostasis and ThrombosisLondonUnited Kingdom
Nuffield Orthopaedic Centre, Hemophilia & Thrombosis CenterOxfordUnited Kingdom
University Hospital Southampton NHS Foundation TrustSouthamptonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

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