UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
NCT ID: NCT07311863
Last Updated: 2025-12-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
EARLY_PHASE1
6 participants
INTERVENTIONAL
2026-01-31
2027-03-31
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Low dose of UGX202 group
UGX202, 4.2E+10 vg per eye, administered as a single intravitreal injection
UGX202 injection
Comparison of different dosages of UGX202
High dose of UGX202 group
UGX202, 1.2E+11 vg/eye, administered as a single intravitreal injection
UGX202 injection
Comparison of different dosages of UGX202
Interventions
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UGX202 injection
Comparison of different dosages of UGX202
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
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Suzhou UgeneX Therapeutics Co., Ltd.
OTHER
Eye & ENT Hospital of Fudan University
OTHER
Responsible Party
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Locations
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Eye & ENT Hospital of Fudan University
Shanghai, Shanghai Municipality, China
Countries
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Central Contacts
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Facility Contacts
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Jihong Wu, MD, Phd
Role: primary
Other Identifiers
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UGENEXIIT002
Identifier Type: -
Identifier Source: org_study_id