NCT01687608

A Phase 1/2 Open-Label, Single Ascending Dose Trial of a Self-Complementing Optimized Adeno-associated Virus Serotype 8 Factor IX Gene Therapy (AskBio009) in Adults With Hemophilia B

Study Summary

The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.

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Interventions

AskBio009BIOLOGICAL
Single dose IV injection

Study Locations

FacilityCityStateCountry
Orthopaedic Hemophilia Treatment CenterLos AngelesCaliforniaUnited States
Children's Hospital Los AngelesLos AngelesCaliforniaUnited States
University of California Davis Medical CenterSacramentoCaliforniaUnited States
University of California at San Diego Medical CenterSan DiegoCaliforniaUnited States
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment CenterAuroraColoradoUnited States
Emory UniversityAtlantaGeorgiaUnited States
Rush University Medical CenterChicagoIllinoisUnited States
Children's Hospital of BostonBostonMassachusettsUnited States
University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science InstituteMinneapolisMinnesotaUnited States
Mount Sinai Medical CenterNew YorkNew YorkUnited States
The Hemophilia Center, Oregon Health and Science UniversityPortlandOregonUnited States
Medical University of South CarolinaCharlestonSouth CarolinaUnited States
Gulf States Hemophilia and Thrombosis CenterHoustonTexasUnited States
Bloodworks NorthwestSeattleWashingtonUnited States
BloodCenter of WisconsinMilwaukeeWisconsinUnited States

Official Trial Information

View on ClinicalTrials.gov

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