Hemophilia BPhase 1/2NCT06120582
Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
A Phase 1/2 study testing ANB-002, dose 1, ANB-002, dose 2 and ANB-002, dose 3 in people with Hemophilia B, run by Biocad at 17 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- ANB-002, dose 1, ANB-002, dose 2 and ANB-002, dose 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 17 sitesin 2 countries
- Study dates
- 2 May 2023to Nov 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male with hemophilia B.
- Age ≥18 years.
- FIX activity at screening ≤2% without FIX inhibitor.
- ≥150 previous exposure days of treatment with FIX concentrates.
Who cannot join
- 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.
Show all 9Show fewer
- Any other disorders associated with severe immunodeficiency.
- Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
- Malignancies with remission <5 years.
Trial locations and contacts
17 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 2 countries
Russia15 sites, 15 recruiting
- Recruiting
State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"
Chelyabinsk, 454048
Aleksandr Korobkin, +7 (351) 729 86 60, chelokb@mail.ru
- Recruiting
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Gatchina, 188300
Vladimir Vorobyev, +7 (812) 670 18 88, lokb@47lokb.ru
- Recruiting
Kuzbass Clinical Hospital named after S.V. Belyaev
Kemerovo, 650066
Marina Kosinova, +7 (3842) 39 65 33, 05-guz-kokb@kuzdrav.ru
- Recruiting
Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
Kirov, 610027
Margarita Timofeeva, +7 (8332) 25 59 19, niigpk@fmbamail.ru
- 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)
Moscow, 125167
Nadezhda Zozulya, +7 (800) 775 05 82, director@blood.ru
- 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)
Moscow, 125167
Vladimir Zorenko, +7 (495) 613 24 69, director@blood.ru
- Recruiting
Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
Moscow, 125284
Vadim Ptushkin, +7 (499) 490 03 03, glavbotkin@zdrav.mos.ru
- Recruiting
LLC "Medis"
Nizhny Novgorod, 603137
Svetlana Volkova, +7 (831) 215 20 00, info@medisnn.ru
- Recruiting
State Novosibirsk Regional Clinical Hospital
Novosibirsk, 630087
Tatyana Pospelova, +7 (383) 315 99 99, gnokb@oblmed.nsk.ru
- Recruiting
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
Saint Petersburg, 191024
Sergey Voloshin, +7 (812) 309 79 81, bloodscience@mail.ru
- Recruiting
City Polyclinic №37
Saint Petersburg, 191186
Tatyana Andreeva, +7(812) 312 82 43, p37@zdrav.spb.ru
- Recruiting
Almazov National Medical Research Centre
Saint Petersburg, 197341
Vladimir Ivanov, +7 (812) 702 37 16, pmu@almazovcentre.ru
- Recruiting
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
Samara, 443099
Igor Davydkin, +7 (846) 374 91 00, info@samsmu.ru
- Recruiting
State Institution "Komi Republican Oncological Dispensary"
Syktyvkar, 167904
Andrey Proydakov, +7 (800) 100 40 28, mail@gukrod.ru
- Recruiting
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
Ufa, 450008
Bulat Bakhirov, +7 (347) 272 41 73, rectorat@bashgmu.ru
Belarus2 sites, 2 recruiting
- Recruiting
Republican Scientific and Practical Center for Radiation Medicine and Human Ecology
Homyel, 246040
Dmitrii Novik, +375232336333, novikdk@mail.ru
- Recruiting
Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology
Minsk, 220089
Anatoly Uss, +375172771091, bmtc@mail.ru
About this study
Official title: 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.
Read the 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.
- Conditions
- Hemophilia B
- Being tested
- ANB-002, dose 1, ANB-002, dose 2, ANB-002, dose 3
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANB-002, dose 1Genetic
Also called arvenacogene sanparvovec
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
ANB-002, dose 2Genetic
Also called arvenacogene sanparvovec
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
ANB-002, dose 3Genetic
Also called arvenacogene sanparvovec
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 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. Receives: Genetic: ANB-002, dose 1 |
| Cohort 2 | Experimental | 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. Receives: Genetic: ANB-002, dose 2 |
| Cohort 3 | Experimental | 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. Receives: Genetic: ANB-002, dose 3 |
| Cohort 4 | Experimental | 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. Receives: Genetic: ANB-002, dose 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in FIX activity from baseline
Measured: 12 months; final assessment - 5 years
Proportion of subjects with adverse reactions
Measured: 12 months; final assessment - 5 years
Secondary outcomes (9)
Changes in FIX activity
Measured: 5 years
Annualized use of FIX concentrates
Measured: 5 years
Annualized bleeding rate
Measured: 5 years
Annualized rate of bleedings requiring therapy with FIX concentrates
Measured: 5 years
Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline
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.
Measured: 5 years
Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline
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.
Measured: 5 years
Changes in SF-36 (Short Form-36) scores from baseline
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.
Measured: 5 years
Response duration based on FIX activity
Measured: 5 years
Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)
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.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start2 May 2023
- Primary completionNov 2025 (estimated)
- Study completionNov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by BiocadThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biocad
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arina V Zinkina-Orikhan, MD, Study director, Director of Clinical Development Department, BIOCAD
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06120582
- Sponsor's protocol ID
- ANB-002-1
- Status last verified
- Jun 2025
- First submitted
- 27 Oct 2023
- Last updated
- 13 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
- ClinicalTrials.gov ID
- NCT06120582
- Acronym / sponsor ID
- ANB-002-1
- Conditions
- Hemophilia B
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- BiocadIndustry (pharma, biotech, devices)
- Study start
- 2 May 2023 (actual)
- Primary completion
- Nov 2025 (estimated)
- Study completion
- Nov 2029 (estimated)
- First posted
- 7 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: ANB-002, dose 1Subjects 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. |
| Cohort 2 | Experimental | Genetic: ANB-002, dose 2Subjects 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. |
| Cohort 3 | Experimental | Genetic: ANB-002, dose 3Subjects 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. |
| Cohort 4 | Experimental | Genetic: ANB-002, dose 3Exploratory 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male with hemophilia B.
- Age ≥18 years.
- FIX activity at screening ≤2% without FIX inhibitor.
- ≥150 previous exposure days of treatment with FIX concentrates.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ANB-002, dose 1Also: arvenacogene sanparvovecAdeno-associated viral vector carrying the FIX gene single infusion at dose 1. | Genetic | Cohort 1 |
| ANB-002, dose 2Also: arvenacogene sanparvovecAdeno-associated viral vector carrying the FIX gene single infusion at dose 2. | Genetic | Cohort 2 |
| ANB-002, dose 3Also: arvenacogene sanparvovecAdeno-associated viral vector carrying the FIX gene single infusion at dose 3. | Genetic | Cohort 3, Cohort 4 |
Primary outcome
Change in FIX activity from baseline
Time frame: 12 months; final assessment - 5 years
Proportion of subjects with adverse reactions
Time frame: 12 months; final assessment - 5 years
9 secondary outcomes
Changes in FIX activity
Time frame: 5 years
Annualized use of FIX concentrates
Time frame: 5 years
Annualized bleeding rate
Time frame: 5 years
Annualized rate of bleedings requiring therapy with FIX concentrates
Time frame: 5 years
Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline
Time frame: 5 years
Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline
Time frame: 5 years
Changes in SF-36 (Short Form-36) scores from baseline
Time frame: 5 years
Response duration based on FIX activity
Time frame: 5 years
Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2025.
- Related InfoLink provided by the sponsor.
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A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
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A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Arina V Zinkina-Orikhan, MD study director
Director of Clinical Development Department, BIOCAD
17 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ANB-002, dose 1, ANB-002, dose 2 and ANB-002, dose 3 in people with Hemophilia B, run by Biocad at 17 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Russia, Belarus. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Anna Eremeeva, MD PhD (+7 (812) 380 49 34 ext. ext. 6963, eremeevaav@biocad.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in FIX activity from baseline (time frame: 12 months; final assessment - 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2025.