Open-Label Study of Single-Dose ANB-002 in Men with Hemophilia B to Assess Safety and Effectiveness

What this Trial Is About

Researchers are evaluating the safety, immune response, and effectiveness of a gene therapy called ANB-002 in adult males with Hemophilia B. This multicenter, open-label study uses a dose-escalation approach combining phase I and II elements to find the best dose of ANB-002, which carries the gene for clotting factor IX FIX. Participants are grouped into four cohorts receiving single intravenous infusions of ANB-002 at increasing doses. Cohorts 1 to 3 involve escalating doses with monitoring for dose-limiting toxicities over 28 days after each infusion. Cohort 4 includes patients with certain antibodies or past hepatitis B infection receiving the highest dose. Decisions to proceed with dosing or enrollment are made by an independent committee. The total participation lasts up to five years. Throughout the study, participants undergo regular assessments including measures of FIX activity, bleeding rates, quality of life questionnaires, and joint health evaluations. Safety monitoring includes tracking adverse reactions during and after treatment. The primary outcomes focus on changes in clotting factor activity and side effects over a five-year period, with secondary outcomes assessing bleeding frequency, treatment use, and well-being.

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)