NCT06120582

Brief Summary

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
42mo left

Started May 2023

Longer than P75 for phase_1

Geographic Reach
2 countries

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress46%
May 2023Nov 2029

Study Start

First participant enrolled

May 2, 2023

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

October 27, 2023

Completed
11 days until next milestone

First Posted

Study publicly available on registry

November 7, 2023

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2025

Completed
4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2029

Expected
Last Updated

June 13, 2025

Status Verified

June 1, 2025

Enrollment Period

2.5 years

First QC Date

October 27, 2023

Last Update Submit

June 10, 2025

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (2)

  • Change in FIX activity from baseline

    12 months; final assessment - 5 years

  • Proportion of subjects with adverse reactions

    12 months; final assessment - 5 years

Secondary Outcomes (9)

  • Changes in FIX activity

    5 years

  • Annualized use of FIX concentrates

    5 years

  • Annualized bleeding rate

    5 years

  • Annualized rate of bleedings requiring therapy with FIX concentrates

    5 years

  • Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline

    5 years

  • +4 more secondary outcomes

Study Arms (4)

Cohort 1

EXPERIMENTAL

Subjects in Cohort 1 will receive ANB-002 (arvenacogene sanparvovec) at a dose 1. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.

Genetic: ANB-002, dose 1

Cohort 2

EXPERIMENTAL

Subjects in Cohort 2 will receive ANB-002 (arvenacogene sanparvovec) at a dose 2. The decision to continue enrolling in Cohort 1 or dosing in Cohort 2 will be made at the IDMC meeting. After the IDMC makes a decision regarding the dosing in Cohort 2 subjects, the next subject will be included in Cohort 2. If no DLT events are observed in the subject of Cohort 2, the following subjects will be included in Cohort 2.

Genetic: ANB-002, dose 2

Cohort 3

EXPERIMENTAL

Subjects in Cohort 3 will receive ANB-002 (arvenacogene sanparvovec) at a dose 3. The decision to continue enrolling in Cohort 2 or dosing in Cohort 3 will be made at the IDMC meeting.

Genetic: ANB-002, dose 3

Cohort 4

EXPERIMENTAL

Exploratory cohort. Subjects in Cohort 4 (with anti-AAV5 antibodies and/or hepatitis B in anamnesis) will receive ANB-002 (arvenacogene sanparvovec) at the dose 3.

Genetic: ANB-002, dose 3

Interventions

ANB-002, dose 1GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.

Also known as: arvenacogene sanparvovec
Cohort 1
ANB-002, dose 2GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.

Also known as: arvenacogene sanparvovec
Cohort 2
ANB-002, dose 3GENETIC

Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.

Also known as: arvenacogene sanparvovec
Cohort 3Cohort 4

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male with hemophilia B.
  • Age ≥18 years.
  • FIX activity at screening ≤2% without FIX inhibitor.
  • ≥150 previous exposure days of treatment with FIX concentrates.

You may not qualify if:

  • Previous gene therapy.
  • Other blood or hematopoietic disorders.
  • Positive Anti-AAV5 antibodies (for Cohorts 1-3).
  • Diagnosed HIV-infection, not controlled with anti-viral therapy.
  • Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
  • Any active systemic infections or recurrent infections requiring systemic therapy at screening.
  • Any other disorders associated with severe immunodeficiency.
  • Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
  • Malignancies with remission \<5 years.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Republican Scientific and Practical Center for Radiation Medicine and Human Ecology

Homyel, 246040, Belarus

RECRUITING

Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology

Minsk, 220089, Belarus

RECRUITING

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"

Chelyabinsk, 454048, Russia

RECRUITING

State budgetary healthcare institution Leningrad Regional Clinical Hospital

Gatchina, 188300, Russia

RECRUITING

Kuzbass Clinical Hospital named after S.V. Belyaev

Kemerovo, 650066, Russia

RECRUITING

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

Kirov, 610027, Russia

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, 125167, Russia

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, 125167, Russia

RECRUITING

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

Moscow, 125284, Russia

RECRUITING

LLC "Medis"

Nizhny Novgorod, 603137, Russia

RECRUITING

State Novosibirsk Regional Clinical Hospital

Novosibirsk, 630087, Russia

RECRUITING

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

Saint Petersburg, 191024, Russia

RECRUITING

City Polyclinic №37

Saint Petersburg, 191186, Russia

RECRUITING

Almazov National Medical Research Centre

Saint Petersburg, 197341, Russia

RECRUITING

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

Samara, 443099, Russia

RECRUITING

State Institution "Komi Republican Oncological Dispensary"

Syktyvkar, 167904, Russia

RECRUITING

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

Ufa, 450008, Russia

RECRUITING

Related Links

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Arina V Zinkina-Orikhan, MD

    Director of Clinical Development Department, BIOCAD

    STUDY DIRECTOR

Central Study Contacts

Anna Eremeeva, MD PhD

CONTACT

+7 (812) 380 49 34eremeevaav@biocad.ru

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 27, 2023

First Posted

November 7, 2023

Study Start

May 2, 2023

Primary Completion

November 1, 2025

Study Completion (Estimated)

November 1, 2029

Last Updated

June 13, 2025

Record last verified: 2025-06

Locations

RU(15)BE(2)