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NCT02576795

A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A

Study Summary

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A.

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Interventions

valoctocogene roxaparvovecBIOLOGICAL
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A

Study Locations

FacilityCityStateCountry
Queen Elizabeth Hospital BirminghamBirminghamUnited Kingdom
Addenbrooke's HospitalCambridgeUnited Kingdom
St. Thomas' HospitalLondonUnited Kingdom
The Royal London HospitalLondonUnited 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