Hemophilia APhase 1/2EDELWEISSNCT06185335
A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
A Phase 1/2 study testing ANB-010, dose 1, ANB-010, dose 2 and ANB-010, dose 3 in people with Hemophilia A, run by Biocad at 17 sites in Russia.
Trial summary
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A. 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 A
- What is being tested?
- ANB-010, dose 1, ANB-010, dose 2 and ANB-010, dose 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 17 sitesRussia
- Study dates
- 26 Jul 2023to Jun 2033
- 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
- 1. Male subjects aged ≥18 years at the time of signing the informed consent form.
- 3. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
- 4. Therapy with FVIII concentrates for at least 150 exposure days.
Who cannot join
- History of use of any gene therapy product.
- Use of emicizumab within less than 6 months before the date of signing the ICF.
- The presence of other blood or hematopoietic disorders other than hemophilia A.
- Presence of AAV6 antibodies detected by ELISA.
- BMI <16 kg/m² or ≥35 kg/m².
- Diagnosis of HIV infection.
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- HBV infection.
- HCV infection.
- Any active systemic infections or recurrent infections requiring systemic therapy at screening.
- Any other disorders associated with severe immunodeficiency.
- Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.
- Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
Russia17 sites
State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"
Chelyabinsk, 454048
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Gatchina, 188300
Kuzbass Clinical Hospital named after S.V. Belyaev
Kemerovo, 650066
Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
Kirov, 610027
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
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
Research Center for Hematology MHSD RF
Moscow, 125167
Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
Moscow, 125284
LLC "Medis"
Nizhny Novgorod, 603137
State Novosibirsk Regional Clinical Hospital
Novosibirsk, 630087
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
Saint Petersburg, 191024
City Polyclinic №37
Saint Petersburg, 191186
Almazov National Medical Research Centre
Saint Petersburg, 197341
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
Samara, 443099
State Institution "Komi Republican Oncological Dispensary"
Syktyvkar, 167904
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
Ufa, 450008
State Autonomous Institution for Healthcare of Sverdlovsk region "Sverdlovsk Regional Clinical Hospital №1"
Yekaterinburg, 620102
About this study
Official title: An Open-Label Two-Stage Trial of the Safety, Pharmacodynamics, Biodistribution, Immunogenicity and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
Read the detailed description
The study will be conducted in 2 stages:
Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study.
Stage 2: study of the efficacy and safety of ANB-010 at the selected potentially therapeutic dose.
The stage 1 design is typical of phase I clinical trials with a modified "3+3" design and dose escalation. Three subjects are to be sequentially included in each cohort, each of whom will recieved a pre-specified cohort dose of ANB-010 as a single inravenous infusion.
Subjects will be monitored for dose-limiting toxicity (DLT) events for 4 weeks after the drug infusion. The decision concerning dose escalation will be made at the Independent Data Monitoring Committee (IDMC) meetings.
At the second stage the main study period will include 6 subjects who will receive ANB-010 at the optimal dose selected based on the results of stage 1 data analysis.
- Conditions
- Hemophilia A
- Being tested
- ANB-010, dose 1, ANB-010, dose 2, ANB-010, dose 3
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANB-010, dose 1Genetic
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 1.
ANB-010, dose 2Genetic
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 2.
ANB-010, dose 3Genetic
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 3.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Subjects in Cohort 1 will receive ANB-010 at a dose 1. ANB-010 will be administered to the first subject in group 1. Not earlier than in 28 days, the investigational product will be administered to the next two subjects in Cohort 1 (with an interval of at least 24 hours). Receives: Genetic: ANB-010, dose 1 |
| Cohort 2 | Experimental | Subjects in Cohort 2 will receive ANB-010 at a dose 2. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 1, the investigational product will be administered to the first subject in Cohort 2. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 2, the investigational product will be administered to the next two subjects in Cohort 2 (with an interval of at least 24 hours). Receives: Genetic: ANB-010, dose 2 |
| Cohort 3 | Experimental | Subjects in Cohort 3 will receive ANB-010 at a dose 3. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 2, the investigational product will be administered to the first subject in Cohort 3. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 3, the investigational product will be administered to the next two subjects in Cohort 3 (with an interval of at least 24 hours). Receives: Genetic: ANB-010, dose 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in FVIII activity from baseline to Week 52
Measured: 12 months
Assessment of ANB-010 safety
Proportion and characteristics of adverse events
Measured: 12 months
Secondary outcomes (7)
Change in FVIII activity from baseline to scheduled assessment visits
FVIII activity will be assessed at every scheduled visits and compared to baseline
Measured: 12 months
Proportion of subjects achieving clinical response
Clinical response is formulated as FVIII activity of 5-150%
Measured: 12 months
Proportion of subjects who achieved normalized response
Normalized response is formulated as FVIII activity of 50-150%
Measured: 12 months
Annualized consumption of FVIII concentrates by a subject
Measured: 12 months
Annualized bleeding rate
Measured: 12 months
Annualized rate of bleeding requiring therapy with FVIII concentrates
Measured: 12 months
Duration of response based on activity FVIII
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2023
- Study start26 Jul 2023
- Primary completionDec 2025 (estimated)
- Study completionJun 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biocad
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arina V Zinkina-Orikhan, PhD, Study director, Director of Clinical Development Department, BIOCAD
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06185335
- Sponsor's protocol ID
- ANB-010-1
- Acronym
- EDELWEISS
- Status last verified
- Apr 2025
- First submitted
- 1 Dec 2023
- Last updated
- 24 Apr 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 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Two-Stage Trial of the Safety, Pharmacodynamics, Biodistribution, Immunogenicity and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
- ClinicalTrials.gov ID
- NCT06185335
- Acronym / sponsor ID
- EDELWEISS / ANB-010-1
- Conditions
- Hemophilia A
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- BiocadIndustry (pharma, biotech, devices)
- Study start
- 26 Jul 2023 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Jun 2033 (estimated)
- First posted
- 29 Dec 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-010 in subjects with hemophilia A. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
Detailed description
The study will be conducted in 2 stages:
Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study.
Stage 2: study of the efficacy and safety of ANB-010 at the selected potentially therapeutic dose.
The stage 1 design is typical of phase I clinical trials with a modified "3+3" design and dose escalation. Three subjects are to be sequentially included in each cohort, each of whom will recieved a pre-specified cohort dose of ANB-010 as a single inravenous infusion.
Subjects will be monitored for dose-limiting toxicity (DLT) events for 4 weeks after the drug infusion. The decision concerning dose escalation will be made at the Independent Data Monitoring Committee (IDMC) meetings.
At the second stage the main study period will include 6 subjects who will receive ANB-010 at the optimal dose selected based on the results of stage 1 data analysis.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: ANB-010, dose 1Subjects in Cohort 1 will receive ANB-010 at a dose 1. ANB-010 will be administered to the first subject in group 1. Not earlier than in 28 days, the investigational product will be administered to the next two subjects in Cohort 1 (with an interval of at least 24 hours). |
| Cohort 2 | Experimental | Genetic: ANB-010, dose 2Subjects in Cohort 2 will receive ANB-010 at a dose 2. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 1, the investigational product will be administered to the first subject in Cohort 2. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 2, the investigational product will be administered to the next two subjects in Cohort 2 (with an interval of at least 24 hours). |
| Cohort 3 | Experimental | Genetic: ANB-010, dose 3Subjects in Cohort 3 will receive ANB-010 at a dose 3. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 2, the investigational product will be administered to the first subject in Cohort 3. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 3, the investigational product will be administered to the next two subjects in Cohort 3 (with an interval of at least 24 hours). |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 1. Male subjects aged ≥18 years at the time of signing the informed consent form.
- 3. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
- 4. Therapy with FVIII concentrates for at least 150 exposure days.
Key exclusion criteria (12 listed)
- History of use of any gene therapy product.
- Use of emicizumab within less than 6 months before the date of signing the ICF.
- The presence of other blood or hematopoietic disorders other than hemophilia A.
- Presence of AAV6 antibodies detected by ELISA.
- BMI <16 kg/m² or ≥35 kg/m².
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-010, dose 1Adeno-associated viral vector carrying the FVIII gene single infusion at dose 1. | Genetic | Cohort 1 |
| ANB-010, dose 2Adeno-associated viral vector carrying the FVIII gene single infusion at dose 2. | Genetic | Cohort 2 |
| ANB-010, dose 3Adeno-associated viral vector carrying the FVIII gene single infusion at dose 3. | Genetic | Cohort 3 |
Primary outcome
Change in FVIII activity from baseline to Week 52
Time frame: 12 months
Assessment of ANB-010 safety
Time frame: 12 months
Proportion and characteristics of adverse events
7 secondary outcomes
Change in FVIII activity from baseline to scheduled assessment visits
Time frame: 12 months
Proportion of subjects achieving clinical response
Time frame: 12 months
Proportion of subjects who achieved normalized response
Time frame: 12 months
Annualized consumption of FVIII concentrates by a subject
Time frame: 12 months
Annualized bleeding rate
Time frame: 12 months
Annualized rate of bleeding requiring therapy with FVIII concentrates
Time frame: 12 months
Duration of response based on activity FVIII
Time frame: 12 months
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 24 Apr 2025.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Arina V Zinkina-Orikhan, PhD study director
Director of Clinical Development Department, BIOCAD
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ANB-010, dose 1, ANB-010, dose 2 and ANB-010, dose 3 in people with Hemophilia A, run by Biocad at 17 sites in Russia.
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 3 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 24 Apr 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. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in FVIII activity from baseline to Week 52 (time frame: 12 months). 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 24 Apr 2025.