drugset / Trial / NCT01620801
Hemophilia B Gene Therapy With AAV8 Vector
Phase 1
Terminated
4 enrolled
Spark Therapeutics, Inc.
Children's Hospital of Philadelphia · collabRoyal Prince Alfred Hospital, Sydney, Australia · collabSt. James's Hospital, Ireland · collabUniversity of Pittsburgh · collab
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2012-10
- Primary completion
- 2016-03
- Completion
- 2016-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | fidanacogene elaparvovec | Gene therapy (AAV / viral vector) | — | Intravenous |