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 participant voluntarily signs the informed consent form (ICF).
The participant is older than 18 years at the time of signing the ICF.
The participant has a confirmed diagnosis of non-syndromic retinitis pigmentosa (RP)
Best-corrected visual acuity (BCVA) in the study eye is <0.05, or the ETDRS letter score is ≤19 letters.
The study eye has previously received any form of gene therapy.
The participant has participated in another interventional clinical trial within 90 days before screening.
The participant has a disease of the study eye or a systemic disease that, in the investigator's opinion, may affect visual function assessment, such as macular degeneration, retinal detachment, retinal arteriolar or venous occlusion, glaucoma, or a central nervous system disorder affecting visual function.
The study eye has an abnormality that may interfere with visual acuity or other ophthalmic assessments, such as opacity of the ocular media that affects fundus examination. At the investigator's discretion, participants with cataracts that do not significantly impair visual acuity may be enrolled.
Single group
UGX202 Injection
DrugThis 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 for details), each dose group comprised 3-6 study participants; a total of 6-12 study participants were enrolled in this phase.
Safety and tolerability
From baseline until week 52 after administration of UGX202 Injection: incidence rates of ocular and non-ocular adverse events (AEs), treatment-emergent adverse events (TEAEs), treatment-related adverse events (TRAEs), adverse events related to study procedures, serious adverse events (SAEs), treatment-related serious adverse events (TRSAEs), and adverse events related to study procedures, etc.
Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye
Change from baseline in BCVA in the study eye, measured in logMAR using the \[FrACT\] method.
Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye
Change from baseline in BCVA in the study eye, measured in logMAR using the ETDRS method.
Change From Baseline in CLVQOL Total Score
Change from baseline in the total score of the Chinese-version Low Vision Quality of Life (CLVQOL) questionnaire.The total score ranges from 0 to 100. Higher scores indicate better vision-related quality of life.
Change From Baseline in [Prespecified VEP Parameter] in the Study Eye
Change from baseline in \[parameter, e.g., P100 amplitude\], measured by flash visual evoked potentials in the study eye.
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