Clinical trials

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.

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.