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

NCT ID: NCT06185335

Last Updated: 2025-04-24

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-07-26
• Study Completion Date: 2033-06-30

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.

Detailed Description

The study will be conducted in 2 stages:

Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study.

Stage 2: study of the efficacy and safety of ANB-010 at the selected potentially therapeutic dose.

The stage 1 design is typical of phase I clinical trials with a modified "3+3" design and dose escalation. Three subjects are to be sequentially included in each cohort, each of whom will recieved a pre-specified cohort dose of ANB-010 as a single inravenous infusion.

Subjects will be monitored for dose-limiting toxicity (DLT) events for 4 weeks after the drug infusion. The decision concerning dose escalation will be made at the Independent Data Monitoring Committee (IDMC) meetings.

At the second stage the main study period will include 6 subjects who will receive ANB-010 at the optimal dose selected based on the results of stage 1 data analysis.

Conditions

Hemophilia A

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

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).

Group Type: EXPERIMENTAL

ANB-010, dose 1

Intervention Type: GENETIC

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

Cohort 2

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).

Group Type: EXPERIMENTAL

ANB-010, dose 2

Intervention Type: GENETIC

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

Cohort 3

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).

Group Type: EXPERIMENTAL

ANB-010, dose 3

Intervention Type: GENETIC

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

Interventions

ANB-010, dose 1

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

Intervention Type: GENETIC

ANB-010, dose 2

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

Intervention Type: GENETIC

ANB-010, dose 3

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

Intervention Type: GENETIC

Eligibility Criteria

Inclusion Criteria

1. Male subjects aged ≥18 years at the time of signing the informed consent form.

3. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.

4. Therapy with FVIII concentrates for at least 150 exposure days.

Exclusion Criteria

1. History of use of any gene therapy product.

2. Use of emicizumab within less than 6 months before the date of signing the ICF.

3. The presence of other blood or hematopoietic disorders other than hemophilia A.

4. Presence of AAV6 antibodies detected by ELISA.

5. BMI <16 kg/m² or ≥35 kg/m².

6. Diagnosis of HIV infection.

7. HBV infection.

8. HCV infection.

9. Any active systemic infections or recurrent infections requiring systemic therapy at screening.

10. Any other disorders associated with severe immunodeficiency.

11. Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.

12. Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.

• Minimum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Biocad

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Arina V Zinkina-Orikhan, PhD

Role: STUDY_DIRECTOR

Director of Clinical Development Department, BIOCAD

Locations

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"

Chelyabinsk, , Russia

State budgetary healthcare institution Leningrad Regional Clinical Hospital

Gatchina, , Russia

Kuzbass Clinical Hospital named after S.V. Belyaev

Kemerovo, , Russia

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

Kirov, , Russia

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

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

Research Center for Hematology MHSD RF

Moscow, , Russia

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

Moscow, , Russia

LLC "Medis"

Nizhny Novgorod, , Russia

State Novosibirsk Regional Clinical Hospital

Novosibirsk, , Russia

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

Saint Petersburg, , Russia

City Polyclinic №37

Saint Petersburg, , Russia

Almazov National Medical Research Centre

Saint Petersburg, , Russia

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

Samara, , Russia

State Institution "Komi Republican Oncological Dispensary"

Syktyvkar, , Russia

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

Ufa, , Russia

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

Yekaterinburg, , Russia

Countries

Russia

Other Identifiers

ANB-010-1

Identifier Type: -

Identifier Source: org_study_id