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

NCT07847723 | Not Yet Recruiting Phase 1

• 1 other identifier: UGX202-S101
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Retinitis Pigmentosa (RP)

Keywords

RP; Retinitis Pigmentosa; UGX202; UGX202-S101

Outcome Measures

Primary Outcomes (1)

• 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.

  From baseline until week 52

Secondary Outcomes (6)

• Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye

  Baseline and Week 4, 8, 12, 24, 36 and W52 after administration

• Change From Baseline in Best-Corrected Visual Acuity (BCVA) in the Study Eye

  Baseline and Weeks 4,8,12,24,36 and 52 after administration

• Change From Baseline in CLVQOL Total Score

  Baseline and Week 12,24,36 and 52 after administration

• Change From Baseline in [Prespecified VEP Parameter] in the Study Eye

  Baseline and [protocol-specified visits] through Week 52

• Change From Baseline in [Prespecified ERG Parameter] in the Study Eye

  Baseline and [protocol-specified visits] through Week 52

Study Arms (1)

a 3+3 dose escalation design to evaluate two UGX202 dose groups

EXPERIMENTAL

Drug: UGX202 Injection

Interventions

UGX202 Injection DRUG

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 for details), each dose group comprised 3-6 study participants; a total of 6-12 study participants were enrolled in this phase.

a 3+3 dose escalation design to evaluate two UGX202 dose groups

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Suzhou UgeneX Therapeutics Co., Ltd.: lead

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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 for details), each dose group comprised 3-6 study participants; a total of 6-12 study participants were enrolled in this phase.

Study Record Dates

• First Submitted: September 17, 2026
• First Posted: September 29, 2026
• Study Start: September 28, 2026
• Primary Completion (Estimated): February 15, 2029
• Study Completion (Estimated): June 15, 2029
• Last Updated: October 2, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share