A Phase 1 Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX

Study Summary

Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding. This study will focus on the severe, most common type of hemophilia B. This study plans to use a virus called adeno-associated virus (AAV), which in nature causes no disease, and can be engineered to deliver the human FIX gene (AAV8-hFIX19 vector) to liver cells, where FIX is normally made. This study will use the AAV8-hFIX19 vector.

Interventions

Study Locations

Facility	City	State	Country
The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States
University of Pittsburgh	Pittsburgh	Pennsylvania	United States
Royal Prince Alfred Hospital	Camperdown	New South Wales	Australia

Official Trial Information

View on ClinicalTrials.gov