NCT07414290

A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations

A Phase I/II Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular Injection in Parkinson's Disease Patients With GBA1 Mutations

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (estimated)
• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. · Industry
• Sex: All
• Age: 30 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

A Phase I/II Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular injection in Parkinson's Disease Patients with GBA1 Mutations

Detailed description

In the open-label dose escalation part, 3 dose cohorts will be explored, with 3 subjects per cohort. Cohort 1: 3 subjects on 4.2×10\^13 vg for at least 4 weeks post infusion Cohort 2: 3 subjects on 8.4×10\^13 vg for at least 4 weeks post infusion Cohort 3: 3 subjects on 1.68×10\^14 vg for at least 4 weeks post infusion In the dose-escalation part, each cohort follows the principle of sentinel administration (i.e., one subject will be enrolled and dosed first in each cohort). If no significant safety risk is observed within 4 weeks after administra-tion, the remaining 2 subjects will be dosed. Additional cohort(s) and/or a safe low and high dose will be determined by the safety review committee (SRC) to initiate Part II

Conditions

• Parkinson Disease (PD)

Interventions

Type	Name	Description
DRUG	VGN-R08b 4.2×10^13 vg	Intracerebroventricular injection
DRUG	VGN-R08b 8.4×10^13 vg	Intracerebroventricular injection
DRUG	VGN-R08b 1.68×10^14 vg	Intracerebroventricular injection

Timeline

• Start date: 2026-03-01
• Primary completion: 2027-09-01
• Completion: 2031-09-01
• First posted: 2026-02-17
• Last updated: 2026-03-02

Locations

1 site across 1 country: China