NCT07311863

UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

Study to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Suzhou UgeneX Therapeutics Co., Ltd. · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this clinical trial is to evaluate the safety and tolerability of a single intravitreal injection of the gene therapy drug UGX202 in patients with advanced RP. The secondary objective is, to assess the preliminary efficacy of a single intravitreal injection of the gene therapy drug UGX202 in treating patients with advanced RP.

Detailed description

This study is a non-randomized, open-label investigator-initiated trial (IIT). It plans to enroll approximately 6 subjects with non-syndromic retinitis pigmentosa (RP) who have extremely low vision (the study eye is the eye with lower vision, and the best corrected visual acuity \[BCVA\] \> logMAR 1.9). The study drug is divided into two dose groups: low dose and high dose. A modified "3+3" dose escalation approach is adopted. The low-dose group (4.2E+10 vg/eye) is planned to include 3 subjects. First, 1 subject (sentinel) will be enrolled and observed for 28 days. If no dose-limiting toxicity (DLT) occurs, 2 more subjects (non-sentinel) will be enrolled and observed for 28 days. The second and third subjects will be enrolled with a 7-day interval. The high-dose group (1.2E+11 vg/eye) is planned to include 3 subjects. Subjects in the high-dose group will be enrolled and administered the drug in sequence after passing the screening. There will be at least a 1-week interval between each subject. The timing of enrolling the full 3 subjects or stopping enrollment will be determined by the investigator's assessment of safety.All subjects will receive intravitreal injection of the study drug UGX202 after enrollment and will be followed up for 52 weeks to evaluate the safety, tolerability, and preliminary efficacy of UGX202.

Conditions

• Retinitis Pigmentosa (RP)

Interventions

Type	Name	Description
GENETIC	UGX202 injection	Comparison of different dosages of UGX202

Timeline

• Start date: 2026-01-01
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2025-12-31
• Last updated: 2025-12-31

Locations

1 site across 1 country: China