NCT06700096

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Biocad · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.

Detailed description

After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject. After completiong the lead-in period subjects will enter the main (interventional) period. At the first visit of the main period subjects will recieve investigational product ANB-002. The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	ANB-002	Adeno-associated viral vector carrying the FIX gene single infusion

Timeline

• Start date: 2024-11-07
• Primary completion: 2027-04-01
• Completion: 2032-03-01
• First posted: 2024-11-21
• Last updated: 2025-11-18

Locations

14 sites across 2 countries: Russia, Thailand