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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ANB-002 | Genetic | Adeno-associated viral vector carrying the FIX gene single infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| FIX activity | 18 months after drug administration; final assessment - 5 years | |
| Annualized FIX consumption | 18 months after drug administration; final assessment - 5 years | |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects with adverse reactions | 18 months after drug administration; final assessment - 5 years | |
| Haemo-A-QoL (Hemophilia-Specific Quality of Life) score | 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. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Anna Eremeeva, MD PhD | Contact | +7 (812) 380 49 34 | 6963 | eremeevaav@biocad.ru |
| Name | Affiliation | Role |
|---|---|---|
| Arina Arina V Zinkina-Orikhan | Biocad | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chelyabinsk Regional Clinical Hospital | Recruiting | Chelyabinsk | Russia |
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| Proportion of subjects not receiving FIX prevention |
| 18 months after drug administration |
| Annualized bleeding rate (ABR) | 18 months after drug administration; final assessment - 5 years |
| Proportion of subjects with spontaneous bleedings | 18 months after drug administration; final assessment - 5 years |
| Annualized rate of spontaneous bleedings | 18 months after drug administration; final assessment - 5 years |
| Proportion of subjects with hemarthrosis | 18 months after drug administration; final assessment - 5 years |
| Annualized hemarthrosis rate | 18 months after drug administration; final assessment - 5 years |
| Proportion of subjects with bleedings requiring FIX concentrate therapy | 18 months after drug administration; final assessment - 5 years |
| Annualized rate of bleedings requiring therapy with FIX concentrates | 18 months after drug administration; final assessment - 5 years |
| Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity | 12 months after stabilization of endogenous FIX activity |
| Proportion of subjects achieving clinical response | Clinical Response is defined as FIX activity 5-150% | 18 months after drug administration |
| Proportion of subjects achieving a normalized response | Normalized response is defined as FIX activity 50-150% | 18 months after drug administration |
| 18 months after drug administration; final assessment - 5 years |
| EuroQol-5D-3L (European Quality of Life Questionnaire) score | 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. | 18 months after drug administration; final assessment - 5 years |
| SF-36 (Short Form-36) score | 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 | 18 months after drug administration; final assessment - 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. | 18 months after drug administration; final assessment - 5 years |
| Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency" | Recruiting | Kirov | 610027 | 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) | Recruiting | Moscow | 125167 | 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) | Recruiting | Moscow | 125167 | Russia |
|
| Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin" | Recruiting | Moscow | 125284 | Russia |
|
| State Novosibirsk Regional Clinical Hospital | Recruiting | Novosibirsk | 630087 | Russia |
|
| State budgetary healthcare institution Leningrad Regional Clinical Hospital | Recruiting | Saint Petersburg | 188663 | Russia |
|
| Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency | Recruiting | Saint Petersburg | 193024 | Russia |
|
| Almazov National Medical Research Centre | Recruiting | Saint Petersburg | 197341 | Russia |
|
| Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation | Recruiting | Samara | 443099 | Russia |
|
| Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation | Recruiting | Ufa | 450008 | Russia |
|
| King Chulalongkorn Memorial Hospital | Not yet recruiting | Bangkok | 10330 | Thailand |
|
| Ramathibodi Hospital | Not yet recruiting | Bangkok | 10400 | Thailand |
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| Siriraj Hospital | Not yet recruiting | Bangkok | 10700 | Thailand |
|
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
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