RECRUITINGPhase 1 / Phase 2INTERVENTIONAL
Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
1. Male with hemophilia B.
2. Age ≥18 years.
3. FIX activity at screening ≤2% without FIX inhibitor.
4. ≥150 previous exposure days of treatment with FIX concentrates.
Who Should NOT Join This Trial:
1. Previous gene therapy.
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 weakened immune system.
8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
9. Malignancies with remission \<5 years.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
1. Male with hemophilia B.
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:
1. Previous gene therapy.
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.
Treatments Being Tested
GENETIC
ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
GENETIC
ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
GENETIC
ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Locations (17)
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 №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