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

Phase 1

Active, not recruiting

• Conditions: Hemophilia A

• Registration Number: NCT06185335
• Lead Sponsor: Biocad

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.

Study Timeline

• Study Start: 2023-07-26
• Study First Submitted: 2023-12-01
• Study First Submitted QC: 2023-12-14
• Study First Posted: 2023-12-29
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-22
• Last Update Posted: 2025-04-24
• Primary Completion: 2025-12
• Study Completion: 2033-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

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

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

3. 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Change in FVIII activity from baseline to Week 52	12 months
Assessment of ANB-010 safety	12 months	Proportion and characteristics of adverse events

Secondary Outcome Measures

Name	Time	Method
Change in FVIII activity from baseline to scheduled assessment visits	12 months	FVIII activity will be assessed at every scheduled visits and compared to baseline
Proportion of subjects achieving clinical response	12 months	Clinical response is formulated as FVIII activity of 5-150%
Annualized consumption of FVIII concentrates by a subject	12 months
Annualized bleeding rate	12 months
Annualized rate of bleeding requiring therapy with FVIII concentrates	12 months
Duration of response based on activity FVIII	12 months
Proportion of subjects who achieved normalized response	12 months	Normalized response is formulated as FVIII activity of 50-150%

Trial Locations

Locations (17)

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"; 🇷🇺 Chelyabinsk, Russian Federation

State Autonomous Institution for Healthcare of Sverdlovsk region "Sverdlovsk Regional Clinical Hospital №1"; 🇷🇺 Ekaterinburg, Russian Federation

State budgetary healthcare institution Leningrad Regional Clinical Hospital; 🇷🇺 Gatchina, Russian Federation

Kuzbass Clinical Hospital named after S.V. Belyaev; 🇷🇺 Kemerovo, Russian Federation

Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"; 🇷🇺 Kirov, Russian Federation

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, Russian Federation

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, Russian Federation

Research Center for Hematology MHSD RF; 🇷🇺 Moscow, Russian Federation

Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"; 🇷🇺 Moscow, Russian Federation

LLC "Medis"; 🇷🇺 Nizhny Novgorod, Russian Federation

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