Actively Recruiting
An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Led by Biocad · Updated on 2025-11-18
24
Participants Needed
14
Research Sites
256 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, effectiveness, and pharmacodynamics of a single dose of ANB-002, a gene therapy, in adult men with hemophilia B who have low factor IX (FIX) activity and no FIX inhibitor. The study aims to show that ANB-002 is not less effective than standard preventive treatment using FIX concentrates. This is an open-label, phase 3 trial sponsored by Biocad. Participants first undergo a non-interventional lead-in period lasting at least six months during which they receive standard FIX preventive treatment. After this, they enter the main interventional period where they receive a single infusion of ANB-002. This main period lasts 18 months, followed by a follow-up period of up to five years after the ANB-002 infusion. During the study, researchers will monitor participants' bleeding rates before and after treatment, FIX activity levels, and FIX concentrate use. They will also assess the proportion of subjects not needing FIX prevention, and track various bleeding events including spontaneous bleedings and hemarthrosis. Safety and treatment effects will be evaluated over the long-term follow-up, with study participation lasting up to five years after treatment.
CONDITIONS
Brief Title
An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Men diagnosed with hemophilia B aged 18 or older
- Factor IX (FIX) activity 2% or lower
- Absence of FIX inhibitor
- At least 150 previous exposure days of treatment with FIX concentrates
You will not qualify if you...
- Any blood or hematopoietic organ diseases other than hemophilia B
- History of any gene therapy, including ANB-002
- Diagnosed HIV infection not controlled with antiviral therapy
- Active hepatitis B or hepatitis C infection
- Presence of anti-AAV5 antibodies
- Any active or recurrent infections requiring systemic therapy
- Disorders causing severe immunodeficiency
- Relevant liver diseases or symptoms
- Malignancies with less than 5 years remission
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 6 months
Participants receive standard Factor IX (FIX) prevention to collect information on individual bleeding rate and FIX concentrate consumption.
Regular visits during lead-in period
Duration - Single day infusion
Participants receive a single infusion of investigational product ANB-002 (arvenacogene sanparvovec).
1 treatment visit (in-person)
Duration - 18 months after treatment
Participants are monitored for safety, efficacy, and pharmacodynamics after the ANB-002 infusion.
Visits scheduled periodically up to 18 months
Duration - Up to 5 years after treatment
Participants are evaluated up to 5 years after the ANB-002 infusion to assess long-term outcomes and safety.
Periodic visits for long-term evaluation
Trial Site Locations
Total: 14 locations
1
Chelyabinsk Regional Clinical Hospital
Chelyabinsk, Russia
Actively Recruiting
2
Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
Kirov, Russia, 610027
Actively Recruiting
3
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, 125167
Actively Recruiting
4
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, 125167
Actively Recruiting
5
Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
Moscow, Russia, 125284
Actively Recruiting
6
State Novosibirsk Regional Clinical Hospital
Novosibirsk, Russia, 630087
Actively Recruiting
7
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Saint Petersburg, Russia, 188663
Actively Recruiting
8
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
Saint Petersburg, Russia, 193024
Actively Recruiting
9
Almazov National Medical Research Centre
Saint Petersburg, Russia, 197341
Actively Recruiting
10
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
Samara, Russia, 443099
Actively Recruiting
11
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
Ufa, Russia, 450008
Actively Recruiting
12
King Chulalongkorn Memorial Hospital
Bangkok, Thailand, 10330
Not Yet Recruiting
13
Ramathibodi Hospital
Bangkok, Thailand, 10400
Not Yet Recruiting
14
Siriraj Hospital
Bangkok, Thailand, 10700
Not Yet Recruiting
Research Team
A
Anna Eremeeva, MD PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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