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

June 10, 2025 updated by: Biocad

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

28

Phase

  • Phase 2
  • Phase 1

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

  • Name: Anna Eremeeva, MD PhD
  • Phone Number: ext. 6963 +7 (812) 380 49 34
  • Email: eremeevaav@biocad.ru

Study Locations

  • Belarus
      • Gomel, Belarus, 246040
        • Recruiting
        • Republican Scientific and Practical Center for Radiation Medicine and Human Ecology
        • Contact:
      • Minsk, Belarus, 220089
        • Recruiting
        • Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology
        • Contact:
          • Anatoly Uss
          • Phone Number: +375172771091
          • Email: bmtc@mail.ru
  • Russian Federation
      • Chelyabinsk, Russian Federation, 454048
        • Recruiting
        • State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"
        • Contact:
          • Aleksandr Korobkin
          • Phone Number: +7 (351) 729 86 60
          • Email: chelokb@mail.ru
      • Gatchina, Russian Federation, 188300
        • Recruiting
        • State Budgetary Healthcare Institution Leningrad Regional Clinical Hospital
        • Contact:
          • Vladimir Vorobyev
          • Phone Number: +7 (812) 670 18 88
          • Email: lokb@47lokb.ru
      • Kemerovo, Russian Federation, 650066
        • Recruiting
        • Kuzbass Clinical Hospital named after S.V. Belyaev
        • Contact:
      • Kirov, Russian Federation, 610027
        • Recruiting
        • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
        • Contact:
      • Moscow, Russian Federation, 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, Russian Federation, 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, Russian Federation, 125284
        • Recruiting
        • Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
        • Contact:
      • Nizhny Novgorod, Russian Federation, 603137
        • Recruiting
        • LLC "Medis"
        • Contact:
      • Novosibirsk, Russian Federation, 630087
        • Recruiting
        • State Novosibirsk regional clinical hospital
        • Contact:
      • Saint Petersburg, Russian Federation, 197341
        • Recruiting
        • Almazov National Medical Research Centre
        • Contact:
      • Saint Petersburg, Russian Federation, 191024
        • Recruiting
        • Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
        • Contact:
      • Saint Petersburg, Russian Federation, 191186
        • Recruiting
        • City Polyclinic №37
        • Contact:
      • Samara, Russian Federation, 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
      • Syktyvkar, Russian Federation, 167904
        • Recruiting
        • State Institution "Komi Republican Oncological Dispensary"
        • Contact:
          • Andrey Proydakov
          • Phone Number: +7 (800) 100 40 28
          • Email: mail@gukrod.ru
      • Ufa, Russian Federation, 450008
        • Recruiting
        • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
        • Contact:

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:

  1. Male with hemophilia B.
  2. Age ≥18 years.
  3. FIX activity at screening ≤2% without FIX inhibitor.
  4. ≥150 previous exposure days of treatment with FIX concentrates.

Exclusion Criteria:

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

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: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Subjects in Cohort 1 will receive ANB-002 (arvenacogene sanparvovec) at a dose 1. 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.
Genetic: ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
Other Names:
  • arvenacogene sanparvovec
Experimental: Cohort 2
Subjects in Cohort 2 will receive ANB-002 (arvenacogene sanparvovec) at a dose 2. The decision to continue enrolling in Cohort 1 or dosing in Cohort 2 will be made at the IDMC meeting. After the IDMC makes a decision regarding the dosing in Cohort 2 subjects, the next subject will be included in Cohort 2. If no DLT events are observed in the subject of Cohort 2, the following subjects will be included in Cohort 2.
Genetic: ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
Other Names:
  • arvenacogene sanparvovec
Experimental: Cohort 3
Subjects in Cohort 3 will receive ANB-002 (arvenacogene sanparvovec) at a dose 3. The decision to continue enrolling in Cohort 2 or dosing in Cohort 3 will be made at the IDMC meeting.
Genetic: ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Other Names:
  • arvenacogene sanparvovec
Experimental: Cohort 4
Exploratory cohort. Subjects in Cohort 4 (with anti-AAV5 antibodies and/or hepatitis B in anamnesis) will receive ANB-002 (arvenacogene sanparvovec) at the dose 3.
Genetic: ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Other Names:
  • arvenacogene sanparvovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion of subjects with adverse reactions
Time Frame: 12 months; final assessment - 5 years
12 months; final assessment - 5 years
Change in FIX activity from baseline
Time Frame: 12 months; final assessment - 5 years
12 months; final assessment - 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in FIX activity
Time Frame: 5 years
5 years
Annualized use of FIX concentrates
Time Frame: 5 years
5 years
Annualized bleeding rate
Time Frame: 5 years
5 years
Annualized rate of bleedings requiring therapy with FIX concentrates
Time Frame: 5 years
5 years
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.
5 years
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.
5 years
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.
5 years
Response duration based on FIX activity
Time Frame: 5 years
5 years
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.
5 years

Collaborators and Investigators

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

Sponsor

Biocad

Investigators

  • Study Director: Arina V Zinkina-Orikhan, MD, Director of Clinical Development Department, BIOCAD

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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)

May 2, 2023

Primary Completion (Estimated)

November 1, 2025

Study Completion (Estimated)

November 1, 2029

Study Registration Dates

First Submitted

October 27, 2023

First Submitted That Met QC Criteria

November 4, 2023

First Posted (Actual)

November 7, 2023

Study Record Updates

Last Update Posted (Actual)

June 13, 2025

Last Update Submitted That Met QC Criteria

June 10, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ANB-002-1

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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.

Clinical Trials on Hemophilia B

Clinical Trials on ANB-002, dose 1

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Slovakia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe