An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ANB-002.
- Who it may be relevant to
- Registry conditions: Hemophilia B. Basic parameters: from 18 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Russia, Thailand
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Interventions
- Genetic ANB-002
Adeno-associated viral vector carrying the FIX gene single infusion
Primary outcome measures
- 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)]
Secondary outcome measures (12)
- 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]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Russia · 11 centers
- Chelyabinsk Regional Clinical Hospital — Chelyabinsk
- Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology an — Kirov
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin" — Moscow
- State Novosibirsk Regional Clinical Hospital — Novosibirsk
- State budgetary healthcare institution Leningrad Regional Clinical Hospital — Saint Petersburg
- Russian Research Institute of Hematology and Transfusiology Federal State Institution of F — Saint Petersburg
- … and 3 more centers
Thailand · 3 centers
- King Chulalongkorn Memorial Hospital — Bangkok
- Ramathibodi Hospital — Bangkok
- Siriraj Hospital — Bangkok
Identifiers
NCT: NCT06700096 · ANB-002-2