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

NCT06700096 | Recruiting Phase 3 DMC

• 1 other identifier: ANB-002-2
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 14

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Annualized bleeding rate (ABR) before and after a single dose of ANB-002

  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

  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)

Secondary Outcomes (13)

• FIX activity

  18 months after drug administration; final assessment - 5 years

• Annualized FIX consumption

  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

• Proportion of subjects with spontaneous bleedings

  18 months after drug administration; final assessment - 5 years

Other Outcomes (5)

• Proportion of subjects with adverse reactions

  18 months after drug administration; final assessment - 5 years

• Haemo-A-QoL (Hemophilia-Specific Quality of Life) score

  18 months after drug administration; final assessment - 5 years

• EuroQol-5D-3L (European Quality of Life Questionnaire) score

  18 months after drug administration; final assessment - 5 years

Study Arms (2)

Lead-in

NO INTERVENTION

All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption

ANB-002 (arvenacogene sanparvovec)

EXPERIMENTAL

After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion.

Genetic: ANB-002

Interventions

ANB-002 GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion

Also known as: arvenacogene sanparvovec

ANB-002 (arvenacogene sanparvovec)

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biocad: lead

Study Sites (14)

Chelyabinsk Regional Clinical Hospital

Chelyabinsk, Russia

RECRUITING

Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"

Kirov, 610027, Russia

RECRUITING

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, 125167, Russia

RECRUITING

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, 125167, Russia

RECRUITING

Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"

Moscow, 125284, Russia

RECRUITING

State Novosibirsk Regional Clinical Hospital

Novosibirsk, 630087, Russia

RECRUITING

State budgetary healthcare institution Leningrad Regional Clinical Hospital

Saint Petersburg, 188663, Russia

RECRUITING

Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency

Saint Petersburg, 193024, Russia

RECRUITING

Almazov National Medical Research Centre

Saint Petersburg, 197341, Russia

RECRUITING

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

Samara, 443099, Russia

RECRUITING

Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation

Ufa, 450008, Russia

RECRUITING

King Chulalongkorn Memorial Hospital

Bangkok, 10330, Thailand

NOT YET RECRUITING

Ramathibodi Hospital

Bangkok, 10400, Thailand

NOT YET RECRUITING

Siriraj Hospital

Bangkok, 10700, Thailand

NOT YET RECRUITING

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Arina Arina V Zinkina-Orikhan

  Biocad

  STUDY DIRECTOR

Central Study Contacts

Anna Eremeeva, MD PhD

CONTACT

+7 (812) 380 49 34; eremeevaav@biocad.ru

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: November 21, 2024
• Study Start: November 7, 2024
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): March 1, 2032
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

TH (3); RU (11)