评价纹状体内注射VGN-R09b在帕金森病患者中的耐受性、安全性和有效性的Ⅰ/Ⅱ期临床研究
[Translation] Phase I/II clinical study to evaluate the tolerability, safety and efficacy of intrastriatal injection of VGN-R09b in patients with Parkinson's disease
评估VGN-R09b治疗帕金森病患者的耐受性和安全性。
[Translation] To evaluate the tolerability and safety of VGN-R09b in patients with Parkinson's disease.
评价壳核内注射VGN-R09b在重度芳香族L-氨基酸脱羧酶(AADC)缺乏症患者中的安全性和有效性的临床研究(当前仅开展I期研究)
[Translation] A clinical study to evaluate the safety and efficacy of intra-putaminal injection of VGN-R09b in patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency (currently only Phase I study is underway)
第一部分
主要目的:评估VGN-R09b治疗AADC缺乏症儿童的安全性、初步有效性。
次要目的:评估VGN-R09b治疗AADC缺乏症儿童的有效性、药代动力学、药效学、免疫原性等特征。
第二部分
主要目的:评估VGN-R09b治疗AADC缺乏症儿童的有效性。
次要目的:评估VGN-R09b治疗AADC缺乏症儿童的安全性。
[Translation] Part I
Main purpose: To evaluate the safety and preliminary efficacy of VGN-R09b in the treatment of children with AADC deficiency.
Secondary purpose: To evaluate the efficacy, pharmacokinetics, pharmacodynamics, immunogenicity and other characteristics of VGN-R09b in the treatment of children with AADC deficiency.
Part II
Main purpose: To evaluate the efficacy of VGN-R09b in the treatment of children with AADC deficiency.
Secondary purpose: To evaluate the safety of VGN-R09b in the treatment of children with AADC deficiency.
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