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NCT Number: NCT06700096

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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Chelyabinsk Regional Clinical Hospital, Chelyabinsk, Russia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ANB-002

Genetic

Adeno-associated viral vector carrying the FIX gene single infusion

Other names: arvenacogene sanparvovec

Primary outcomes

  1. 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

Secondary outcomes

  1. FIX activity

    Time frame: 18 months after drug administration; final assessment - 5 years

  2. Annualized FIX consumption

    Time frame: 18 months after drug administration; final assessment - 5 years

  3. Proportion of subjects not receiving FIX prevention

    Time frame: 18 months after drug administration

  4. Annualized bleeding rate (ABR)

    Time frame: 18 months after drug administration; final assessment - 5 years

  5. Proportion of subjects with spontaneous bleedings

    Time frame: 18 months after drug administration; final assessment - 5 years

  6. Annualized rate of spontaneous bleedings

    Time frame: 18 months after drug administration; final assessment - 5 years

  7. Proportion of subjects with hemarthrosis

    Time frame: 18 months after drug administration; final assessment - 5 years

  8. Annualized hemarthrosis rate

    Time frame: 18 months after drug administration; final assessment - 5 years

  9. Proportion of subjects with bleedings requiring FIX concentrate therapy

    Time frame: 18 months after drug administration; final assessment - 5 years

  10. Annualized rate of bleedings requiring therapy with FIX concentrates

    Time frame: 18 months after drug administration; final assessment - 5 years

  11. 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. Proportion of subjects achieving clinical response

    Time frame: 18 months after drug administration

    Clinical Response is defined as FIX activity 5-150%

  13. Proportion of subjects achieving a normalized response

    Time frame: 18 months after drug administration

    Normalized response is defined as FIX activity 50-150%

Other outcomes

  1. Proportion of subjects with adverse reactions

    Time frame: 18 months after drug administration; final assessment - 5 years

  2. 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.

  3. 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.

  4. 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

  5. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Anna Eremeeva, MD PhD

CONTACT

[email protected]

+7 (812) 380 49 34 ext. 6963

Sponsors and collaborators

Lead sponsor

Biocad

Industry

Registry information

Acronym: MAGNOLIA

Important dates

Study start
2024
Primary completion
2027
Study completion
2032
First posted
Nov 21, 2024
Registry last updated
Nov 18, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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