An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

NCT ID: NCT06700096

Last Updated: 2025-11-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-07

Study Completion Date

2032-03-31

Brief Summary

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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

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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.

Conditions

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Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lead-in

All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption

Group Type NO_INTERVENTION

No interventions assigned to this group

ANB-002 (arvenacogene sanparvovec)

After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion.

Group Type EXPERIMENTAL

ANB-002

Intervention Type GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion

Interventions

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ANB-002

Adeno-associated viral vector carrying the FIX gene single infusion

Intervention Type GENETIC

Other Intervention Names

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arvenacogene sanparvovec

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Biocad

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Arina Arina V Zinkina-Orikhan

Role: STUDY_DIRECTOR

Biocad

Locations

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Chelyabinsk Regional Clinical Hospital

Chelyabinsk, , Russia

Site Status RECRUITING

Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"

Kirov, , Russia

Site Status RECRUITING

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

Site Status RECRUITING

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

Site Status RECRUITING

Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"

Moscow, , Russia

Site Status RECRUITING

State Novosibirsk Regional Clinical Hospital

Novosibirsk, , Russia

Site Status RECRUITING

State budgetary healthcare institution Leningrad Regional Clinical Hospital

Saint Petersburg, , Russia

Site Status RECRUITING

Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency

Saint Petersburg, , Russia

Site Status RECRUITING

Almazov National Medical Research Centre

Saint Petersburg, , Russia

Site Status RECRUITING

Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation

Samara, , Russia

Site Status RECRUITING

Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation

Ufa, , Russia

Site Status RECRUITING

King Chulalongkorn Memorial Hospital

Bangkok, , Thailand

Site Status NOT_YET_RECRUITING

Ramathibodi Hospital

Bangkok, , Thailand

Site Status NOT_YET_RECRUITING

Siriraj Hospital

Bangkok, , Thailand

Site Status NOT_YET_RECRUITING

Countries

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Russia Thailand

Central Contacts

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Anna Eremeeva, MD PhD

Role: CONTACT

+7 (812) 380 49 34 ext. 6963

Facility Contacts

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Alexander Korobkin

Role: primary

+7 (351) 749-37-27

Margarita Timofeeva

Role: primary

+7 (8332) 25 59 19

Nadezhda Zozulya

Role: primary

+7 (800) 775 05 82

Vladimir Zorenko

Role: primary

+7 (800) 775 05 82

Vadim Ptushkin

Role: primary

+7 (499) 490 03 03

Tatyana Pospelova

Role: primary

+7 (383) 315 99 99

Sergey Voloshin

Role: primary

+7 (812) 670 18 88

Andrey Garifullin

Role: primary

+7 (812) 309 79 81

Vladimir Ivanov

Role: primary

+7 (812) 702 37 16

Igor Davydkin

Role: primary

+7 (846) 374 91 00

Bulat Bakhirov

Role: primary

+7 (347) 272 41 73

Ponlapat Rojnuckarin

Role: primary

6685-911-521

Pantep Angchaisuksiri

Role: primary

Yingyong Chinnathammamit

Role: primary

6681-426-4252

Other Identifiers

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ANB-002-2

Identifier Type: -

Identifier Source: org_study_id

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