UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: UGX202 injection.
- Who it may be relevant to
- Registry conditions: Retinitis Pigmentosa (RP). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Study to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
Overview
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.
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.
Interventions
- Genetic UGX202 injection
Comparison of different dosages of UGX202
Primary outcome measures
- Incidence of adverse events and serious adverse events [Time frame: 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 [Time frame: 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 (3)
- The changes in BCVA [Time frame: 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 [Time frame: baseline to Week 4, Week 12, Week 24, Week 52]
- The changes in the visual function questionnaire (VFQ-25) scores [Time frame: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Eye & ENT Hospital of Fudan University — Shanghai
Identifiers
NCT: NCT07311863 · UGENEXIIT002