NCT00979238

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

An Open Label Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector (scAAV 2/8-LP1-hFIXco) For Gene Transfer in Hemophilia B

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor. Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and who have been treated/exposed to Factor IX concentrates for at least ten years and have had an average of 3 bleeding episodes per year requiring FIX administration will be enrolled. Patients will be recruited within the United States for treatment at St. Jude Children's Research Hospital, and patients will be recruited in England and other countries for treatment in London by our British collaborators.

Detailed description

Hemophilia B is caused by an absence or abnormality in the gene that produces the factor IX protein. Affected individuals cannot make a blood clot effectively and suffer from severe bleeding episodes. Repeated bleeding episodes, specifically into joints, can cause chronic joint disease and lead to disability. This research study will test the safety of giving an affected individual a normal factor IX gene which can produce factor IX protein in his body. We will give the normal gene for factor IX by using an inactivated (not able to function) virus called "the vector." The vector used in this study was developed from an adeno-associated virus that has been changed so that it is unable to cause a viral infection in humans. This inactivated virus was further altered to carry the factor IX gene and to locate within liver cells where factor IX protein is normally made. The protocol long-term follow-up (LTFU) period was changed from 15 years to 5 years in accordance with the FDA guidance for participants who received human gene therapy products. The FDA updated the guidance stating their current recommendations for the duration of an LTFU protocol, based on product, to be up to five years for AAV vectors. All study participants have completed 5 years of follow-up and are being transitioned off study.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	Gene Transfer	Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant
DRUG	scAAV2/8-LP1-hFIXco	Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant.

Timeline

• Start date: 2010-02-22
• Primary completion: 2026-03-26
• Completion: 2026-03-26
• First posted: 2009-09-17
• Last updated: 2026-04-02

Locations

4 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study