Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
NCT ID: NCT06120582
Last Updated: 2025-06-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
28 participants
INTERVENTIONAL
2023-05-02
2029-11-30
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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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.
ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
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.
ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
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.
ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
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.
ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Interventions
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ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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.
18 Years
MALE
No
Sponsors
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Biocad
INDUSTRY
Responsible Party
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Principal Investigators
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Arina V Zinkina-Orikhan, MD
Role: STUDY_DIRECTOR
Director of Clinical Development Department, BIOCAD
Locations
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Republican Scientific and Practical Center for Radiation Medicine and Human Ecology
Homyel, , Belarus
Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology
Minsk, , Belarus
State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"
Chelyabinsk, , Russia
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Gatchina, , Russia
Kuzbass Clinical Hospital named after S.V. Belyaev
Kemerovo, , Russia
Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
Kirov, , Russia
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, , Russia
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, , Russia
Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
Moscow, , Russia
LLC "Medis"
Nizhny Novgorod, , Russia
State Novosibirsk Regional Clinical Hospital
Novosibirsk, , Russia
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
Saint Petersburg, , Russia
City Polyclinic â„–37
Saint Petersburg, , Russia
Almazov National Medical Research Centre
Saint Petersburg, , Russia
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
Samara, , Russia
State Institution "Komi Republican Oncological Dispensary"
Syktyvkar, , Russia
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
Ufa, , Russia
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Other Identifiers
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ANB-002-1
Identifier Type: -
Identifier Source: org_study_id
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