Hemophilia BPhase 3MAGNOLIANCT06700096
An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
A Phase 3 study testing ANB-002 in people with Hemophilia B, run by Biocad at 14 sites in 2 countries.
Trial summary
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. 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 7 Nov 2024to Mar 2032
- Phase
- Phase 3
- 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
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
14 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 2 countries
Russia11 sites, 11 recruiting
- Recruiting
Chelyabinsk Regional Clinical Hospital
Chelyabinsk
Alexander Korobkin, +7 (351) 749-37-27, chelokb@mail.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 (800) 775 05 82, 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
State Novosibirsk Regional Clinical Hospital
Novosibirsk, 630087
Tatyana Pospelova, +7 (383) 315 99 99, gnokb@oblmed.nsk.ru
- Recruiting
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Saint Petersburg, 188663
Sergey Voloshin, +7 (812) 670 18 88, lokb@47lokb.ru
- Recruiting
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
Saint Petersburg, 193024
Andrey Garifullin, +7 (812) 309 79 81, bloodscience@mail.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
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, bashsmu@yandex.ru
Thailand3 sites, 3 recruiting
- Not yet recruiting
King Chulalongkorn Memorial Hospital
Bangkok, 10330
Ponlapat Rojnuckarin, 6685-911-521, ponlapat.r@chula.ac.th
- Not yet recruiting
Ramathibodi Hospital
Bangkok, 10400
Pantep Angchaisuksiri, pantep.ang@mahidol.ac.th
- Not yet recruiting
Siriraj Hospital
Bangkok, 10700
Yingyong Chinnathammamit, 6681-426-4252, dryingyong@gmail.com
About this study
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.
Read the detailed description
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.
- Conditions
- Hemophilia B
- Being tested
- ANB-002
- 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-002Genetic
Also called arvenacogene sanparvovec
Adeno-associated viral vector carrying the FIX gene single infusion
| Study group | Role | What they receive |
|---|---|---|
| Lead-in | No intervention | All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption |
| ANB-002 (arvenacogene sanparvovec) | Experimental | After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion. Receives: Genetic: ANB-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized bleeding rate (ABR) before and after a single dose of ANB-002
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
Measured: 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)
Secondary outcomes (13)
FIX activity
Measured: 18 months after drug administration; final assessment - 5 years
Annualized FIX consumption
Measured: 18 months after drug administration; final assessment - 5 years
Proportion of subjects not receiving FIX prevention
Measured: 18 months after drug administration
Annualized bleeding rate (ABR)
Measured: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with spontaneous bleedings
Measured: 18 months after drug administration; final assessment - 5 years
Annualized rate of spontaneous bleedings
Measured: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with hemarthrosis
Measured: 18 months after drug administration; final assessment - 5 years
Annualized hemarthrosis rate
Measured: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with bleedings requiring FIX concentrate therapy
Measured: 18 months after drug administration; final assessment - 5 years
Annualized rate of bleedings requiring therapy with FIX concentrates
Measured: 18 months after drug administration; final assessment - 5 years
Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity
Measured: 12 months after stabilization of endogenous FIX activity
Proportion of subjects achieving clinical response
Clinical Response is defined as FIX activity 5-150%
Measured: 18 months after drug administration
Proportion of subjects achieving a normalized response
Normalized response is defined as FIX activity 50-150%
Measured: 18 months after drug administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2024
- Study start7 Nov 2024
- Primary completionApr 2027 (estimated)
- Study completionMar 2032 (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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biocad
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arina Arina V Zinkina-Orikhan, Study director, Biocad
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06700096
- Sponsor's protocol ID
- ANB-002-2
- Acronym
- MAGNOLIA
- Status last verified
- Nov 2025
- First submitted
- 19 Nov 2024
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
- ClinicalTrials.gov ID
- NCT06700096
- Acronym / sponsor ID
- MAGNOLIA / ANB-002-2
- Conditions
- Hemophilia B
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- BiocadIndustry (pharma, biotech, devices)
- Study start
- 7 Nov 2024 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Mar 2032 (estimated)
- First posted
- 21 Nov 2024 (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 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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lead-in | No intervention | –All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption |
| ANB-002 (arvenacogene sanparvovec) | Experimental | Genetic: ANB-002After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion. |
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)
- 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
Key exclusion criteria (9 listed)
- 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
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-002Also: arvenacogene sanparvovecAdeno-associated viral vector carrying the FIX gene single infusion | Genetic | ANB-002 (arvenacogene sanparvovec) |
Primary outcome
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
13 secondary outcomes
FIX activity
Time frame: 18 months after drug administration; final assessment - 5 years
Annualized FIX consumption
Time frame: 18 months after drug administration; final assessment - 5 years
Proportion of subjects not receiving FIX prevention
Time frame: 18 months after drug administration
Annualized bleeding rate (ABR)
Time frame: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with spontaneous bleedings
Time frame: 18 months after drug administration; final assessment - 5 years
Annualized rate of spontaneous bleedings
Time frame: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with hemarthrosis
Time frame: 18 months after drug administration; final assessment - 5 years
Annualized hemarthrosis rate
Time frame: 18 months after drug administration; final assessment - 5 years
Proportion of subjects with bleedings requiring FIX concentrate therapy
Time frame: 18 months after drug administration; final assessment - 5 years
Annualized rate of bleedings requiring therapy with FIX concentrates
Time frame: 18 months after drug administration; final assessment - 5 years
Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity
Time frame: 12 months after stabilization of endogenous FIX activity
Proportion of subjects achieving clinical response
Time frame: 18 months after drug administration
Proportion of subjects achieving a normalized response
Time frame: 18 months after drug administration
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 18 Nov 2025.
- Individual participant dataSharing planned: No
Related trials for Hemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
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
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
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Hemophilia
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 Arina V Zinkina-Orikhan study director
Biocad
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ANB-002 in people with Hemophilia B, run by Biocad at 14 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 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Russia, Thailand. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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. 6963, eremeevaav@biocad.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). 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 18 Nov 2025.