A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A

NCT06185335 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: ANB-010-1
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 17

Brief Summary

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (2)

• Change in FVIII activity from baseline to Week 52

  12 months

• Assessment of ANB-010 safety

  Proportion and characteristics of adverse events

  12 months

Secondary Outcomes (7)

• Change in FVIII activity from baseline to scheduled assessment visits

  12 months

• Proportion of subjects achieving clinical response

  12 months

• Proportion of subjects who achieved normalized response

  12 months

• Annualized consumption of FVIII concentrates by a subject

  12 months

• Annualized bleeding rate

  12 months

Other Outcomes (14)

• Pharmacodynamics of ANB-010

  12 months

• Proportion of subjects with FVIII inhibitor

  12 months

• Proportion of subjects with antibodies to capsid

  12 months

Study Arms (3)

Cohort 1

EXPERIMENTAL

Subjects in Cohort 1 will receive ANB-010 at a dose 1. ANB-010 will be administered to the first subject in group 1. Not earlier than in 28 days, the investigational product will be administered to the next two subjects in Cohort 1 (with an interval of at least 24 hours).

Genetic: ANB-010, dose 1

Cohort 2

EXPERIMENTAL

Subjects in Cohort 2 will receive ANB-010 at a dose 2. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 1, the investigational product will be administered to the first subject in Cohort 2. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 2, the investigational product will be administered to the next two subjects in Cohort 2 (with an interval of at least 24 hours).

Genetic: ANB-010, dose 2

Cohort 3

EXPERIMENTAL

Subjects in Cohort 3 will receive ANB-010 at a dose 3. Not earlier than 28 days after the ANB-010 administration to the third subject in Cohort 2, the investigational product will be administered to the first subject in Cohort 3. Not earlier than 28 days after the ANB-010 administration to the first subject in Cohort 3, the investigational product will be administered to the next two subjects in Cohort 3 (with an interval of at least 24 hours).

Genetic: ANB-010, dose 3

Interventions

ANB-010, dose 1 GENETIC

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

Cohort 1

ANB-010, dose 2 GENETIC

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

Cohort 2

ANB-010, dose 3 GENETIC

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

Cohort 3

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Male subjects aged ≥18 years at the time of signing the informed consent form.
• \. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
• \. Therapy with FVIII concentrates for at least 150 exposure days.

You may not qualify if:

• History of use of any gene therapy product.
• Use of emicizumab within less than 6 months before the date of signing the ICF.
• The presence of other blood or hematopoietic disorders other than hemophilia A.
• Presence of AAV6 antibodies detected by ELISA.
• BMI \<16 kg/m² or ≥35 kg/m².
• Diagnosis of HIV infection.
• HBV infection.
• HCV infection.
• Any active systemic infections or recurrent infections requiring systemic therapy at screening.
• Any other disorders associated with severe immunodeficiency.
• Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.
• Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.

Sponsors & Collaborators

• Biocad: lead

Study Sites (17)

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"

Chelyabinsk, 454048, Russia

Location

State budgetary healthcare institution Leningrad Regional Clinical Hospital

Gatchina, 188300, Russia

Location

Kuzbass Clinical Hospital named after S.V. Belyaev

Kemerovo, 650066, Russia

Location

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

Kirov, 610027, Russia

Location

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

Location

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

Location

Research Center for Hematology MHSD RF

Moscow, 125167, Russia

Location

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

Moscow, 125284, Russia

Location

LLC "Medis"

Nizhny Novgorod, 603137, Russia

Location

State Novosibirsk Regional Clinical Hospital

Novosibirsk, 630087, Russia

Location

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

Saint Petersburg, 191024, Russia

Location

City Polyclinic №37

Saint Petersburg, 191186, Russia

Location

Almazov National Medical Research Centre

Saint Petersburg, 197341, Russia

Location

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

Samara, 443099, Russia

Location

State Institution "Komi Republican Oncological Dispensary"

Syktyvkar, 167904, Russia

Location

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

Ufa, 450008, Russia

Location

State Autonomous Institution for Healthcare of Sverdlovsk region "Sverdlovsk Regional Clinical Hospital №1"

Yekaterinburg, 620102, Russia

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Arina V Zinkina-Orikhan, PhD

  Director of Clinical Development Department, BIOCAD

  STUDY DIRECTOR

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: December 1, 2023
• First Posted: December 29, 2023
• Study Start: July 26, 2023
• Primary Completion: December 1, 2025
• Study Completion (Estimated): June 1, 2033
• Last Updated: April 24, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

RU (17)