Phase 1Not yet recruiting
Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis Pigmentosa
An Open-label, Dose-escalation and Expanded Phase I/II Clinical Trial to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
- Sponsor
- Suzhou UgeneX Therapeutics Co., Ltd. (Academic or other)
- Enrolment
- 12 planned
- Conditions
- Retinitis Pigmentosa (RP)
- Interventions
- UGX202 Injection
- Ages
- adults, older adults
- Registry
- NCT07847723
From the registry
This is a single-arm, open-label study employing a 3+3 dose escalation design to evaluate two UGX202 dose groups: the low-dose group (1.2×10¹¹ vg/eye) and the high-dose group (3.5×10¹¹ vg/eye). According to the "Dose Escalation Rule" (see below), each dose group comprised 3-6 study participants; thus, a total of 6-12 study participants were enrolled in this phase. Participants were enrolled in ascending order from the lowest to the highest dose; each study participant could participate in only one dose group (defined as having received the investigational drug - participants who had not received the drug were not considered to have participated in any dose group), and could not participate in multiple dose groups sequentially.
Primary outcomes
- Safety and tolerability