NCT06185335

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

An Open-Label Two-Stage Trial of the Safety, Pharmacodynamics, Biodistribution, Immunogenicity and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Biocad · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
GENETIC	ANB-010, dose 1	Adeno-associated viral vector carrying the FVIII gene single infusion at dose 1.
GENETIC	ANB-010, dose 2	Adeno-associated viral vector carrying the FVIII gene single infusion at dose 2.
GENETIC	ANB-010, dose 3	Adeno-associated viral vector carrying the FVIII gene single infusion at dose 3.

Timeline

• Start date: 2023-07-26
• Primary completion: 2025-12-01
• Completion: 2033-06-01
• First posted: 2023-12-29
• Last updated: 2025-04-24

Locations

17 sites across 1 country: Russia