The Mississippi center for Advanced Medicine is recruiting Hemophilia B males to understand a new factor IX (FIX) gene transfer method.

Patients Must Be:
  • Male 18 years or older with Hemophilia B
  • Factor iX deficiency (≤ 2%)
  • On-demand therapy or prophylaxis with history of bleeding
  • Available for study-related visits for about 12 months Identifier: NCT02484092


Since the factor IX gene was discovered, scientists have been working on a way to transfer the normal factor IX gene into patients with hemophilia to produce a normal factor IX protein.  SPK-9001 (the study drug) is an adeno-associated viral (AAV) vector that is packed with the factor IX gene. Viral vectors are tools commonly used to deliver genetic material into cells. The AAV vector is a modified adeno-associated virus that has been optimized for gene transfer applications. The unmodified adeno-associated virus infects humans, but has not been described to result in illness. In this study, SPK-9001 (the study drug) will be injected into the bloodstream by an intravenous (IV) infusion.  SPK-9001 is attracted to the liver.  In the liver, the normal factor IX gene makes the factor IX protein.  The factor IX protein is then released into the bloodstream.  If the production of factor IX by the liver results in high enough levels in the bloodstream, the number of bleeding events may be reduced.  This research study is to determine if factor IX gene transfer is safe for the treatment of hemophilia B.

Can I participate?

Yes, you may be elgible if you meet the following criteria:

  • Adult male 18 years or older with hemophilia B (FIX level is 2% or less)
  • Have a history of bleeding that requires prophylaxis or on demand therapy
  • No history of inhibitor to factor IX or allergic reaction to factor IX products
  • Don’t have active hepatitis B or C (eligible if treated and clear for at least 6 months)
  • Not on antiviral therapy for Hepatitis B or C
  • HIV positive is allowed if on effective treatment
  • Don’t have severe liver disease
  • Available for study-related visits for about 12 months
What is involved?
  • A screening phase that may last up to several weeks
  • The infusion day may last up to 6 hours.  On the infusion day, the study volunteer will have an intravenous (IV) infusion of his regular factor IX product for 10 minutes then the infusion of the gene transfer study drug for about an hour.
  • After the infusion, he will have follow-up visits* up to 52 weeks.
  • Most visits will include physical exams, questionnaire, blood draws, and other tests (ranging from 15 minutes to 60 minutes per visit).

* There is flexibility regarding the location of follow-up visits after the study drug infusion and some visits can be arranged by mobile phlebotomists for your convenience (please inquire for more details.)

How will this study be different than previous hemophilia studies?
  • AAV vector used in this study was modified to have a higher attraction to the liver.
  • The factor IX gene used in this study produces “super-active” factor IX protein (about 5-8 times more active than normal factor IX protein).
For inquiry about this study, please contact: Spencer Sullivan, MD at or (601) 499-0935.