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NCT07847723

PHASE1

Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis Pigmentosa

Sponsor: Suzhou UgeneX Therapeutics Co., Ltd.

View on ClinicalTrials.gov

Summary

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.

Official title: An Open-label, Dose-escalation and Expanded Phase I/II Clinical Trial to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-09-28

Completion Date

2029-06-15

Last Updated

2026-10-02

Healthy Volunteers

Conditions

Retinitis Pigmentosa (RP)

Interventions

DRUG

UGX202 Injection

Inclusion Criteria * 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 participant's pupil can be sufficiently dilated to allow comprehensive ophthalmic examination and testing. * The participant agrees to use effective contraception from enrollment until at least 1 year after the last administration of study treatment. Effective methods include complete abstinence (if this is the participant's preferred and usual lifestyle), an intrauterine device, bilateral tubal ligation, a partner who has undergone vasectomy with confirmed azoospermia, or a dual-barrier method (a male condom used with a cervical cap, diaphragm, or spermicidal sponge). * The spherical equivalent refractive error of the study eye is between -6.00 D and +6.00 D, inclusive. Exclusion Criteria: * 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. * The participant has an ocular disease or abnormality that may affect safety assessment, such as an active ocular infection, chronic uveitis, or ocular trauma within the preceding 3 months. * The participant has a contraindication to pupil dilation, such as a narrow anterior chamber angle. * The study eye has undergone vitrectomy or macular surgery. * The study eye has undergone major surgery or intraocular surgery within the 3 months before screening, as determined by the investigator.