NCT05441553

A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

A Phase 1/2 Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 26 (estimated)
• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. · Industry
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

A multicenter, open, non-randomized, phase I/II, two-phase clinical study. The dose exploration phase was phase I, and the dose extension phase was phase II.

Detailed description

Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain from chronic haemarthropathy. Current replacement therapy needs regular treatment in the life-long time, bringing heavy economic and social burdens. VGB-R04 is a novel AAV vector carrying a high specific activity factor IX variant. This study is intended to evaluate the safety, tolerability and efficacy of a single IV infusion of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 6 weeks before administration of VGB-R04. All subjects will undergo 52 weeks of safety observation and will be encouraged to enroll in an Long-term follow-up study to evaluate the long-term safety of VGB-R04 for a total of five years.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	VGB-R04	A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant

Timeline

• Start date: 2022-07-01
• Primary completion: 2025-01-01
• Completion: 2025-01-01
• First posted: 2022-07-01
• Last updated: 2022-07-01

Locations

1 site across 1 country: China