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

November 17, 2025 updated by: Biocad
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Russia
      • Chelyabinsk, Russia
        • Recruiting
        • Chelyabinsk Regional Clinical Hospital
        • Contact:
          • Alexander Korobkin
          • Phone Number: +7 (351) 749-37-27
          • Email: chelokb@mail.ru
      • Kirov, Russia, 610027
        • Recruiting
        • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
        • Contact:
      • Moscow, Russia, 125167
        • 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)
        • Contact:
      • Moscow, Russia, 125167
        • 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)
        • Contact:
      • Moscow, Russia, 125284
        • Recruiting
        • Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
        • Contact:
      • Novosibirsk, Russia, 630087
        • Recruiting
        • State Novosibirsk regional clinical hospital
        • Contact:
      • Saint Petersburg, Russia, 188663
        • Recruiting
        • State Budgetary Healthcare Institution Leningrad Regional Clinical Hospital
        • Contact:
          • Sergey Voloshin
          • Phone Number: +7 (812) 670 18 88
          • Email: lokb@47lokb.ru
      • Saint Petersburg, Russia, 193024
        • Recruiting
        • Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
        • Contact:
      • Saint Petersburg, Russia, 197341
        • Recruiting
        • Almazov National Medical Research Centre
        • Contact:
      • Samara, Russia, 443099
        • Recruiting
        • Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
        • Contact:
          • Igor Davydkin
          • Phone Number: +7 (846) 374 91 00
          • Email: info@samsmu.ru
      • Ufa, Russia, 450008
        • Recruiting
        • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
        • Contact:
  • Thailand
      • Bangkok, Thailand, 10330
        • Not yet recruiting
        • King Chulalongkorn Memorial Hospital
        • Contact:
      • Bangkok, Thailand, 10700
        • Not yet recruiting
        • Siriraj Hospital
        • Contact:
      • Bangkok, Thailand, 10400

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Lead-in
All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption
Experimental: ANB-002 (arvenacogene sanparvovec)
After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion.
Genetic: ANB-002
Adeno-associated viral vector carrying the FIX gene single infusion
Other Names:
  • arvenacogene sanparvovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized bleeding rate (ABR) before and after a single dose of ANB-002
Time Frame: 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
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 Outcome Measures

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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects with adverse reactions
Time Frame: 18 months after drug administration; final assessment - 5 years
18 months after drug administration; final assessment - 5 years
Haemo-A-QoL (Hemophilia-Specific Quality of Life) score
Time Frame: 18 months after drug administration; final assessment - 5 years
Haemo-A-QoL is a quality of life (QoL) assessment instrument for patients with haemophilia, including the domains of consequences of bleeding, emotional impact, physical functioning, role functioning, treatment concern, and worry. Using scale overall score ranges from 0 to 100.The high score represent low quality of life.
18 months after drug administration; final assessment - 5 years
EuroQol-5D-3L (European Quality of Life Questionnaire) score
Time Frame: 18 months after drug administration; final assessment - 5 years
The EQ-5D-5L descriptive system of health-related QoL states consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). The EQ-5D-5L VAS overall score ranges from 0 to 100. A higher score is considered to be more favorable.
18 months after drug administration; final assessment - 5 years
SF-36 (Short Form-36) score
Time Frame: 18 months after drug administration; final assessment - 5 years
SF-36 is a questionnaire for evaluating health-related QoL (Quality of Life). The 36 questions are meant to reflect 8 domains of health, including physical functioning, physical role, pain, general health, vitality, social function, emotional role, and mental health. SF-3 overall score ranges from 0 to 100. The lower the score the more disability
18 months after drug administration; final assessment - 5 years
Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)
Time Frame: 18 months after drug administration; final assessment - 5 years
Hemophilia Joint Health Score (HJHS) is the assessment system of joints in patients with haemophilia. Each joint (knee, elbow and ankle) receives a numeric score, which can be compared to itself over time to determine whether a joint is showing degeneration. The maximum score for an individual index joint is 20. Gait is scored 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
18 months after drug administration; final assessment - 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Biocad

Investigators

  • Study Director: Arina Arina V Zinkina-Orikhan, Biocad

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 7, 2024

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

March 1, 2032

Study Registration Dates

First Submitted

November 19, 2024

First Submitted That Met QC Criteria

November 19, 2024

First Posted (Actual)

November 21, 2024

Study Record Updates

Last Update Posted (Actual)

November 18, 2025

Last Update Submitted That Met QC Criteria

November 17, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ANB-002-2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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