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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Retinitis Pigmentosa (RP)
• Intervention / Treatment: Genetic: UGX202 injection

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.

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Jihong Wu, MD, Phd; Phone Number: +86 21 6437 7134; Email: 1217586177@qq.com
• Study Contact Backup: Name: Xiuqian Yi, MD, PHD; Phone Number: +86 21 6437 7134; Email: 1217586177@qq.com

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200031
      • Eye & ENT Hospital of Fudan University
      • Contact: Jihong Wu, MD, Phd; Phone Number: +86 21 6437 7134; Email: 1217586177@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Provide written informed consent form (ICF).
• Age ≥18 years at ICF signing.
• Diagnosed as non-syndromic RP；
• BCVA > logMAR 1.9 (assessed by FrACT) in the study eye.
• Confirmation of preserved memory of visual experience
• Spherical equivalent between -9D and +6D.

Exclusion Criteria:

• Prior gene therapy in either eye.
• Received any interventional investigational drug within 90 days prior to screening.
• Any Study eye disease or systemic disease judged by the investigator to affect visual function assessment.
• Hypersensitivity to corticosteroids, intolerance to corticosteroid regimen, active concurrent infection contraindicating treatment.
• History or tendency of psychiatric disorders impacting safety and/or efficacy assessment.
• Any other factor deemed unsuitable by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low dose of UGX202 group; UGX202, 4.2E+10 vg per eye, administered as a single intravitreal injection	Genetic: UGX202 injection; Comparison of different dosages of UGX202
Experimental: High dose of UGX202 group; UGX202, 1.2E+11 vg/eye, administered as a single intravitreal injection	Genetic: UGX202 injection; Comparison of different dosages of UGX202

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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;	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;	baseline to Week 4, Week 12, Week 24, Week 52
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.	baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
The change in Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP	Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP both in the study eye and non-study eyes at each visits compared with baseline.	Baseline, week4, week 8, week 12, week 24, week 52/EoS
The change in dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG	Dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG will be evaluated in electroretinogram both in the study eye and non-study eyes at each visits compared with baseline.	Baseline, week4, week 8, week 12, week 24, week 52/EoS
Change of mean defect (MD) in the visual fields	Change of mean defect (MD) in the visual fields of the study eyes and non-study eyes	Baseline, week 24, week 52/EoS
Change of visual field index (VFI) in the visual fields	Change of visual field index (VFI) in the visual fields of the study eyes and non-study eyes	Baseline, week 24, week 52/EoS
The benefit outcomes of patients with retinitis pigmentosa (RP) of different genotypes in either best-corrected visual acuity (BCVA) or the multi-luminance mobility test (MLMT)	The benefit outcomes of patients with retinitis pigmentosa (RP) of different genotypes either in best-corrected visual acuity (BCVA) or the multi-luminance mobilitytest (MLMT), and will conduct correlation the above analysis between factors.	Baseline
Changes in the scores of MLMT	Changes in the scores of multi-luminance mobility test (MLMT)	Baseline，week4， week 8, week 12, week 24, week 52/EoS
Change of Color Vision Test score	Color vision test will be conducted to patients' assess judgment and discrimination abilities for different channels, with comparisons of the changes in scores at different study visits related to the baseline scores.	Baseline, week4, week 12, week 24, week 52/EoS
Titer of viral vector DNA detected in blood, tears, and urine	Detection of viral vector DNA in blood, tears, and urine	Baseline，Day3， Day7, week2, week 12, week 24, week 52/EoS
Number of participants with positive Anti-drug antibodies(ADA) and neutralizing antibodies(Nab)	From the time of administration of UGX202 injection until the 52nd cycle, the detection of anti-drug antibodies (ADA) and neutralizing antibodies (Nab) for the viral vector capsid protein was carried out.	Baseline, week2, week4, week 12, week 24, week 36, week 52/EoS
Number of participants with positive anti-target photosensitive protein	From the time of administration of UGX202 injection until the 52nd cycle, ADA (anti-target photosensitive protein) detection was conducted.	Baseline, week2, week4, week 12, week 24, week 36, week 52/EoS
Concentration of T-cell immune responses against the viral vector capsid protein and the target photosensitive protein.	From the time of UGX202 injection treatment until the 52nd cycle, ELISpot was used to detect T-cell immune responses against the viral vector capsid protein and the target photosensitive protein.	Baseline, week 12, week 24

Collaborators and Investigators

• Sponsor: Suzhou UgeneX Therapeutics Co., Ltd.
• Collaborators: Eye & ENT Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: November 14, 2025
• First Submitted That Met QC Criteria: December 16, 2025
• First Posted (Actual): December 31, 2025

Study Record Updates

• Last Update Posted (Actual): December 31, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: AAV; RP; retinitis pigmentosa; UGX202; UGENEXIIT002
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Dystrophies; Retinal Degeneration; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinitis Pigmentosa
• Other Study ID Numbers: UGENEXIIT002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No