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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Comparison of different dosages of UGX202
Eye & ENT Hospital of Fudan University
Shanghai, Shanghai Municipality, China
Incidence of adverse events and serious adverse events
From the time of administration of UGX202 injection until the 52nd week, based on the topical and systemic safety data, the incidence rates of AEs, TEAEs during treatment, TRAEs related to the study drug, TRAEs related to the study procedures, SAEs, TRSAEs related to the study drug, and TRSAEs related to the study procedures during the study period were summarized by the investigators, and the correlations between AEs and the study drug and study procedures were determined.
Time frame: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52
The average change in IOP
The average change in IOP of the study eyes and non-study eyes from after treatment to the 52nd week compared to the baseline. The IOP was measured three times consecutively at each visit and the average value was taken.
Time frame: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52
The changes in BCVA
The changes in BCVA of the study eyes and non-study eyes at each follow-up visit compared to the baseline were evaluated. BCVA was assessed using the Freiburg Vision Test (FrACT) system. If the subjects had no light perception at the baseline: the proportion of subjects whose BCVA improved to having light perception after treatment was evaluated, and the change in their vision compared to the baseline was assessed;
Time frame: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52
The changes in the average stimulus threshold
The changes in the average stimulus threshold measured by the full-field stimulus threshold test (FST) of the study eyes and/or non-study eyes at each follow-up visit compared to the baseline;
Time frame: baseline to Week 4, Week 12, Week 24, Week 52
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The changes in the visual function questionnaire (VFQ-25) scores
The changes in the visual function questionnaire (VFQ-25) scores of the subjects at each follow-up visit after the treatment compared to the baseline.
Time frame: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52