NCT02618915

Phase I/II Open-Label Safety and Dose Finding Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B

Study Summary

A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.

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Interventions

DTX101GENETIC
solution for IV infusion

Study Locations

FacilityCityStateCountry
Arkansas Children's HospitalLittle RockArkansasUnited States
Orthopaedic Institute for ChildrenLos AngelesCaliforniaUnited States
University of FloridaGainesvilleFloridaUnited States
Boston Children's HospitalBostonMassachusettsUnited States
University of Michigan Hospital and Health Systems, Michigan Clinical Research UnitAnn ArborMichiganUnited States
Vanderbilt Hemostasis-Thrombosis ClinicNashvilleTennesseeUnited States
Specialized Hospital for Active Treatment for Hematological DiseaseSofiaBulgaria
Basingstoke and North Hampshire Hospital, Haemophilia, Haemostasis and Thrombosis CentreBasingstokeHampshireUnited Kingdom
The Christie NHS Foundation TrustManchesterUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

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