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

Phase 3

Recruiting

• Conditions: Hemophilia B

• Registration Number: NCT06700096
• Lead Sponsor: Biocad

Brief 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.

Study Timeline

• Study Start: 2024-11-07
• Study First Submitted: 2024-11-19
• Study First Submitted QC: 2024-11-19
• Study First Posted: 2024-11-21
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-19
• Last Update Posted: 2025-02-20
• Primary Completion: 2027-04
• Study Completion: 2032-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

• Men diagnosed with hemophilia B aged 18 or older
• FIX activity ≤2%
• Absense of FIX inhibitor
• ≥150 previous exposure days of treatment with FIX concentrates

Exclusion Criteria

• Any diseases of blood and hematopoietic organs other than hemophilia B
• A history of any gene therapy, including ANB-002
• Diagnosed HIV-infection, not controlled with anti-viral therapy
• Active HBV or HCV infection
• Anti-AAV5 antibodies
• Any active systemic infections or recurrent infections requiring systemic therapy
• Any other disorders associated with severe immunodeficiency
• Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder
• Malignancies with less than 5 years of remission

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Annualized bleeding rate (ABR) before and after a single dose of ANB-002	Lead-in period: at least 6 months prior to ANB-002 infusion (Lead-in arm); Month 12 to Month 18 post infusion (ANB-002 arm)	A comparison will be made between the mean ABR values in the lead-in period and within 12 months after stabilization of endogenous FIX activity, but not later than 18 months after the ANB-002 dose

Secondary Outcome Measures

Name	Time	Method
Proportion of subjects with spontaneous bleedings	18 months after drug administration; final assessment - 5 years
Annualized rate of spontaneous bleedings	18 months after drug administration; final assessment - 5 years
Proportion of subjects with hemarthrosis	18 months after drug administration; final assessment - 5 years
Annualized hemarthrosis rate	18 months after drug administration; final assessment - 5 years
Proportion of subjects not receiving FIX prevention	18 months after drug administration
Annualized bleeding rate (ABR)	18 months after drug administration; final assessment - 5 years
Annualized FIX consumption	18 months after drug administration; final assessment - 5 years
FIX activity	18 months after drug administration; final assessment - 5 years
Proportion of subjects with bleedings requiring FIX concentrate therapy	18 months after drug administration; final assessment - 5 years
Annualized rate of bleedings requiring therapy with FIX concentrates	18 months after drug administration; final assessment - 5 years
Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity	12 months after stabilization of endogenous FIX activity
Proportion of subjects achieving clinical response	18 months after drug administration	Clinical Response is defined as FIX activity 5-150%
Proportion of subjects achieving a normalized response	18 months after drug administration	Normalized response is defined as FIX activity 50-150%

Trial Locations

Locations (11)

Chelyabinsk Regional Clinical Hospital; 🇷🇺 Chelyabinsk, Russian Federation

Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"; 🇷🇺 Kirov, Russian Federation

Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Clinical Diagnostics for Hematology and Hemostasis Disorders); 🇷🇺 Moscow, Russian Federation

Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Traumatology and Reconstructive and Restorative Orthopedics for Patients with Hemophilia); 🇷🇺 Moscow, Russian Federation

Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"; 🇷🇺 Moscow, Russian Federation

State Novosibirsk Regional Clinical Hospital; 🇷🇺 Novosibirsk, Russian Federation

State budgetary healthcare institution Leningrad Regional Clinical Hospital; 🇷🇺 Saint Petersburg, Russian Federation

Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency; 🇷🇺 Saint Petersburg, Russian Federation

Almazov National Medical Research Centre; 🇷🇺 Saint Petersburg, Russian Federation

Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation; 🇷🇺 Samara, Russian Federation

Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation; 🇷🇺 Ufa, Russian Federation