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NCT Number: NCT06120582

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive 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 1 / Phase 2

Primary location

Republican Scientific and Practical Center for Radiation Medicine and Human Ecology, Homyel, Belarus

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About this study

The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.

In Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).

The decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.

Based on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.

In exploratory Cohort 4 patients with anti-AAV5 antibodies and/or hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.

The total duration of participation of one subjects in the study will be 5 years.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Male with hemophilia B.
  • Age ≥18 years.
  • FIX activity at screening ≤2% without FIX inhibitor.
  • ≥150 previous exposure days of treatment with FIX concentrates.

Exclusion criteria

  • Previous gene therapy.
  • Other blood or hematopoietic disorders.
  • Positive Anti-AAV5 antibodies (for Cohorts 1-3).
  • Diagnosed HIV-infection, not controlled with anti-viral therapy.
  • Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
  • Any active systemic infections or recurrent infections requiring systemic therapy at screening.
  • Any other disorders associated with severe immunodeficiency.
  • Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
  • Malignancies with remission <5 years.

Treatment and study plan

ANB-002, dose 1

Genetic

Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.

Other names: arvenacogene sanparvovec

ANB-002, dose 2

Genetic

Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.

Other names: arvenacogene sanparvovec

ANB-002, dose 3

Genetic

Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.

Other names: arvenacogene sanparvovec

Primary outcomes

  1. Change in FIX activity from baseline

    Time frame: 12 months; final assessment - 5 years

  2. Proportion of subjects with adverse reactions

    Time frame: 12 months; final assessment - 5 years

Secondary outcomes

  1. Changes in FIX activity

    Time frame: 5 years

  2. Annualized use of FIX concentrates

    Time frame: 5 years

  3. Annualized bleeding rate

    Time frame: 5 years

  4. Annualized rate of bleedings requiring therapy with FIX concentrates

    Time frame: 5 years

  5. Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline

    Time frame: 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.

  6. Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline

    Time frame: 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.

  7. Changes in SF-36 (Short Form-36) scores from baseline

    Time frame: 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.

  8. Response duration based on FIX activity

    Time frame: 5 years

  9. Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)

    Time frame: 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. ext. 6963

Sponsors and collaborators

Lead sponsor

Biocad

Industry

Registry information

Official study title

An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B

Important dates

Study start
2023
Primary completion
2025
Study completion
2029
First posted
Nov 7, 2023
Registry last updated
Jun 13, 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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