This protocol provides long-term follow-up for people who received the AAV2-hFIX16 vector into the liver for severe hemophilia B between 2001 and 2009. Following US FDA guidelines for long-term follow-up (LTFU) of investigational gene therapy products, it records clinical outcomes and any adverse events. Participants undergo annual history/physical examination, blood tests and periodic liver ultrasound, with data collected for up to 15 years after gene transfer.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria -Adult subjects who participated in prior intrahepatic AAV2-hFIX16 gene transfer studies Exclusion Criteria * Subjects who will not consent for study * Subjects who the investigators believe are not capable of performing endpoints of the study