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Clinical Trials associated with VGB-R04评价VGB-R04在成人血友病B患者中的安全性、耐受性I/II期临床研究
[Translation] Phase I/II clinical study to evaluate the safety and tolerability of VGB-R04 in adult patients with hemophilia B
剂量探索阶段:
主要目的:
1.评估VGB-R04治疗成人血友病B(HB)患者的耐受性和安全性。
2.评估VGB-R04给药后,稳态FIX活性水平。
次要目的:
1.评估VGB-R04治疗成人HB患者的有效性,探索评估有效性的最佳疗效指标,为后续研究提供科学依据;
2.评估VGB-R04的免疫原性;
3.评估VGB-R04给药后载体基因组脱落情况。
剂量扩展阶段:
主要目的:
评估VGB-R04治疗成人血友病B(HB)患者的有效性。
次要目的:
1.评估VGB-R04治疗成人HB患者的安全性;
2.评估VGB-R04的免疫原性;
3.评估VGB-R04给药后载体基因组脱落情况。
[Translation] Dose exploration phase:
Main objectives:
1. Evaluate the tolerability and safety of VGB-R04 in the treatment of adult patients with hemophilia B (HB).
2. Evaluate the steady-state FIX activity level after VGB-R04 administration.
Secondary objectives:
1. Evaluate the effectiveness of VGB-R04 in the treatment of adult patients with HB, explore the best efficacy indicators for evaluating effectiveness, and provide scientific basis for subsequent studies;
2. Evaluate the immunogenicity of VGB-R04;
3. Evaluate the shedding of vector genomes after VGB-R04 administration.
Dose expansion phase:
Main objectives:
Evaluate the effectiveness of VGB-R04 in the treatment of adult patients with hemophilia B (HB).
Secondary objectives:
1. Evaluate the safety of VGB-R04 in the treatment of adult patients with HB;
2. Evaluate the immunogenicity of VGB-R04;
3. Evaluate the shedding of vector genomes after VGB-R04 administration.
A Phase 1/2 Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients
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.
Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.
Start Date28 Dec 2021 |
Sponsor / Collaborator- |
100 Clinical Results associated with VGB-R04
100 Translational Medicine associated with VGB-R04
100 Patents (Medical) associated with VGB-R04
100 Deals associated with VGB-R04