A Phase 3 interventional study of ANB-002 in Hemophilia B, sponsored by Biocad. Recruiting at 14 sites in 2 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-11-18.
Sponsored by Biocad · Phase 3, Interventional, and Treatment
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.
292 studies on the registry are indexed under Hemophilia B; 51 are open to participants now.
This study's planned enrollment of 24 is close to the median of 26 across 171 interventional studies indexed under Hemophilia B.
Browse Hemophilia B studies →Biocad is the lead sponsor of 94 studies on the registry; 12 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption
After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion.
Genetic: ANB-002
Adeno-associated viral vector carrying the FIX gene single infusion
Also known as: arvenacogene sanparvovec
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
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)
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
Clinical Response is defined as FIX activity 5-150%
Time frame: 18 months after drug administration
Proportion of subjects achieving a normalized response
Normalized response is defined as FIX activity 50-150%
Time frame: 18 months after drug administration
Proportion of subjects with adverse reactions
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: 18 months after drug administration; final assessment - 5 years
Plan to share: No
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Started Nov 2024 (actual). Primary completion is estimated for Apr 2027, 29 months after the start.
Of 35 industry-sponsored phase 3 interventional studies under Hemophilia B, half reached primary completion within 38 months of starting (the median), counting those still running; a quarter within 23 and three quarters within 70. This study's planned 29 months is below that.
Counting every sponsor, half of 35 studies reached it within 38 months; a quarter within 23 and three quarters within 70.
How these figures are worked out, and which studies are left out of them.
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