[Translation] An open-label, dose-escalation, and dose-expansion Phase I study evaluating the tolerability, safety, and efficacy of VGM-R02b in patients with glutaric acidemia type I
主要目的:
评估VGM-R02b治疗GA-I患者的安全性和耐受性。
次要目的:
1.评估VGM-R02b治疗GA-I患者的初步有效性,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VGM-R02b治疗GA-I患者的头颅影像学变化;
3.评估VGM-R02b治疗GA-I患者体内相关生物标志物的变化;
4.评估VGM-R02b的免疫原性;
[Translation] Main purpose:
To evaluate the safety and tolerability of VGM-R02b in the treatment of GA-I patients.
Secondary purpose:
1. To evaluate the preliminary effectiveness of VGM-R02b in the treatment of GA-I patients, explore the best efficacy indicators for evaluating effectiveness, and provide scientific basis for subsequent research;
2. To evaluate the changes in cranial imaging of GA-I patients treated with VGM-R02b;
3. To evaluate the changes in relevant biomarkers in GA-I patients treated with VGM-R02b;
4. To evaluate the immunogenicity of VGM-R02b;