Study of Single-Dose ANB-002 Gene Therapy in Adult Men with Hemophilia B with Low Factor IX Levels

What this Trial Is About

Researchers are evaluating the safety, effectiveness, and pharmacodynamics of a single dose of ANB-002, a gene therapy, in adult men with hemophilia B who have low factor IX FIX activity and no FIX inhibitor. The study aims to show that ANB-002 is not less effective than standard preventive treatment using FIX concentrates. This is an open-label, phase 3 trial sponsored by Biocad. Participants first undergo a non-interventional lead-in period lasting at least six months during which they receive standard FIX preventive treatment. After this, they enter the main interventional period where they receive a single infusion of ANB-002. This main period lasts 18 months, followed by a follow-up period of up to five years after the ANB-002 infusion. During the study, researchers will monitor participants bleeding rates before and after treatment, FIX activity levels, and FIX concentrate use. They will also assess the proportion of subjects not needing FIX prevention, and track various bleeding events including spontaneous bleedings and hemarthrosis. Safety and treatment effects will be evaluated over the long-term follow-up, with study participation lasting up to five years after treatment.

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B