Clinical trial NCT06700096: An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Official title: An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hemophilia B
Lead sponsor: Biocad
Sponsor class: INDUSTRY
First posted: 2024-11-21
Enrollment: 24
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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

Primary outcomes:
measure: Annualized bleeding rate (ABR) before and after a single dose of ANB-002
timeFrame: 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 outcomes:
measure: FIX activity
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Annualized FIX consumption
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Proportion of subjects not receiving FIX prevention
timeFrame: 18 months after drug administration
measure: Annualized bleeding rate (ABR)
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Proportion of subjects with spontaneous bleedings
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Annualized rate of spontaneous bleedings
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Proportion of subjects with hemarthrosis
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Annualized hemarthrosis rate
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Proportion of subjects with bleedings requiring FIX concentrate therapy
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Annualized rate of bleedings requiring therapy with FIX concentrates
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity
timeFrame: 12 months after stabilization of endogenous FIX activity
measure: Proportion of subjects achieving clinical response
timeFrame: 18 months after drug administration
measure: Proportion of subjects achieving a normalized response
timeFrame: 18 months after drug administration
Other outcomes:
measure: Proportion of subjects with adverse reactions
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Haemo-A-QoL (Hemophilia-Specific Quality of Life) score
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: EuroQol-5D-3L (European Quality of Life Questionnaire) score
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: SF-36 (Short Form-36) score
timeFrame: 18 months after drug administration; final assessment - 5 years
measure: Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)
timeFrame: 18 months after drug administration; final assessment - 5 years
Locations:
city: Chelyabinsk
country: Russian Federation
facility: Chelyabinsk Regional Clinical Hospital
city: Kirov
country: Russian Federation
facility: Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
city: Moscow
country: Russian Federation
facility: 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)
city: Moscow
country: Russian Federation
facility: 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)
city: Moscow
country: Russian Federation
facility: Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
city: Novosibirsk
country: Russian Federation
facility: State Novosibirsk Regional Clinical Hospital
city: Saint Petersburg
country: Russian Federation
facility: State budgetary healthcare institution Leningrad Regional Clinical Hospital
city: Saint Petersburg
country: Russian Federation
facility: Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
city: Saint Petersburg
country: Russian Federation
facility: Almazov National Medical Research Centre
city: Samara
country: Russian Federation
facility: Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
city: Ufa
country: Russian Federation
facility: Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
Study officials:
affiliation: Biocad
name: Arina Arina V Zinkina-Orikhan
role: STUDY_DIRECTOR
References: