[Translation] Safety, tolerability and efficacy of RGL-193 administered via bilateral putamen injection via intracranial flexible catheter system in patients with advanced Parkinson's disease: a randomized, double-blind, sham-controlled, dose-escalation phase I clinical trial
主要研究目的:1.评估中晚期帕金森病患者接受RGL-193双侧壳核注射给药后 52周内的安全性和耐受性;
2.确定RGL-193在中晚期PD患者壳核注射给药的Ⅱ期推荐剂量。
次要研究目的:1.评估中晚期 PD 患者术后第26周、第52周,相关酶的代谢活性;2.评价中晚期PD患者接受RGL-193壳核注射给药后第26周、第52周,脑多巴胺能神经元的完整性;3.评估中晚期 PD 患者术后第26周、第52周的临床症状改善。
探索性研究目的:评估中晚期PD患者给药后52周内的生物分布。
[Translation] Main study objectives: 1. To evaluate the safety and tolerability of RGL-193 in patients with advanced Parkinson's disease within 52 weeks after bilateral putamen injection;
2. To determine the recommended Phase II dose of RGL-193 in patients with advanced PD.
Secondary study objectives: 1. To evaluate the metabolic activity of related enzymes in patients with advanced PD at 26 weeks and 52 weeks after surgery; 2. To evaluate the integrity of brain dopaminergic neurons in patients with advanced PD at 26 weeks and 52 weeks after RGL-193 putamen injection; 3. To evaluate the improvement of clinical symptoms in patients with advanced PD at 26 weeks and 52 weeks after surgery.
Exploratory study objectives: To evaluate the biodistribution of patients with advanced PD within 52 weeks after administration.