UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

NCT07311863 | Not Yet Recruiting Early Phase 1

• 1 other identifier: UGENEXIIT002
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Retinitis Pigmentosa (RP)

Keywords

UGX202; UGENEXIIT002; retinitis pigmentosa; RP; AAV

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• The changes in BCVA

  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

  baseline to Week 4, Week 12, Week 24, Week 52

• The changes in the visual function questionnaire (VFQ-25) scores

  baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52

Other Outcomes (11)

• The change in Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP

  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

  Baseline, week4, week 8, week 12, week 24, week 52/EoS

• Change of mean defect (MD) in the visual fields

  Baseline, week 24, week 52/EoS

Study Arms (2)

Low dose of UGX202 group

EXPERIMENTAL

UGX202, 4.2E+10 vg per eye, administered as a single intravitreal injection

Genetic: UGX202 injection

High dose of UGX202 group

EXPERIMENTAL

UGX202, 1.2E+11 vg/eye, administered as a single intravitreal injection

Genetic: UGX202 injection

Interventions

UGX202 injection GENETIC

Comparison of different dosages of UGX202

High dose of UGX202 group; Low dose of UGX202 group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (1)

Eye & ENT Hospital of Fudan University

Shanghai, Shanghai Municipality, 200031, China

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Jihong Wu, MD, PHD

CONTACT

+86 21 6437 7134; 1217586177@qq.com

Xiuqian Yi, MD, PHD

CONTACT

+86 21 6437 7134; 1217586177@qq.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2025
• First Posted: December 31, 2025
• Study Start: January 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: December 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)