Advanced retinitis pigmentosa
Conditions
Interventions
Low-dose group (4.2E+10 vg per eye):4.2E+10 vg/eye UXG202 injection solution
High-dose group (1.2E+11 vg per eye):1.2E+11 vg/eye UXG202 injection solution
Alternate dose group (1.4E+10 vg per eye, one-third of the low dose):1.4E+10 vg/eye UXG202 injection solution
Sponsors
Fudan University Affiliated Eye, Ear and Nose Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: The participants must meet all of the following inclusion criteria: 1. Voluntary signing of the Informed Consent Form (ICF); 2. At the time of signing the ICF, the participant's age is >= 18 years old; 3. Diagnosed with non-syndromic RP, defined as: a) Clinically diagnosed as non-syndromic RP based on medical history, visual impairment, and fundus examination, etc. And b) After OCT confirmation, the diagnosis of non-syndromic RP was confirmed by fundus autofluorescence (FAF), VEP, and ERG examinations. 4. Vision: The BCVA of the study eye was > logMAR 1.9 (evaluated using the FrACT system); 5. The thickness of the retinal ganglion cell complex and the retinal nerve fiber layer was measured using optical coherence tomography (OCT), and the study investigator evaluated and enrolled the subject; 6. The previous visual memory was retained; 7. The pupil could be fully dilated to facilitate comprehensive ophthalmic examination and testing; 8. For women with potential fertility, the pregnancy test result was negative and they agreed to take effective contraceptive measures within 12 months after the injection of AAV; for men with fertility, they agreed to take effective contraceptive measures within 12 months after the injection of AAV; or for infertile women and men; 9. The refractive error of the eye was between -9D and +6D.
Exclusion criteria
Exclusion criteria: If a subject meets any of the following exclusion criteria, they will not meet the inclusion requirements: 1. Any eye has previously received any gene therapy. 2. The subject has undergone other interventional trial drug clinical trials within 90 days prior to screening. 3. The subject has a disease or systemic disease that the investigator considers may affect the assessment of visual function, including but not limited to macular degeneration, retinal detachment, retinal artery/vein occlusion, glaucoma, or central nervous system lesions that affect visual function. 4. The study eye has abnormal findings in ocular examinations that interfere with vision and other eye assessments (such as refractive media opacity that affects fundus observation (this can include patients with RP accompanied by mild vision impairment due to cataracts)). 5. Eye diseases or abnormal findings in ocular examinations that affect vision and other eye assessments, including but not limited to active ocular infections, chronic uveitis, and ocular trauma within 3 months. 6. There are mydriasis contraindications, such as narrow anterior chamber angle. 7. Previous vitrectomy or vitreomacular surgery in the study eye. 8. The study eye has undergone major surgery or internal eye surgery within 3 months prior to screening (as determined by the investigator). 9. The subject has a history of systemic malignant tumors/eye tumors within 5 years. 10. The subject has a systemic disease that the investigator assesses as poorly controlled and has an impact on the safety observation of the trial. For example: Hypertensive patients whose blood pressure is not controlled after antihypertensive treatment: systolic/diastolic blood pressure >= 160/100 mmHg; Patients with poor blood glucose control: fasting blood glucose > 10.0 mmol/L or HbA1c > 10%; Patients with abnormal coagulation function: prothrombin time >= upper limit of normal by 3 seconds, activated partial thromboplastin time >= upper limit of normal by 10 seconds; Patients with abnormal tumor markers as assessed by the investigator (alpha-fetoprotein, carcinoembryonic antigen, CA125, CA153, CA199) and with clinical significance. 11. Any immunoreaction dysfunction, including immunodeficiency diseases, and those receiving or potentially receiving immunosuppressive therapy other than this study. 12. Pregnant or lactating subjects. 13. Subjects who are unwilling or unable to comply with the study protocol. 14. Subjects with HIV antibody positive and syphilis antibody positive, as well as active hepatitis B infection and active hepatitis C infection. 15. Subjects allergic to the study treatment drug or its excipient components. 16. Subjects allergic to glucocorticoids, or unable to tolerate glucocorticoid regimens, or with active concurrent infections that are contraindicated for treatment. 17. Subjects assessed by the investigator have a history or tendency of mental disorders that may affect the assessment of trial safety and efficacy. 18. Subjects considered by the investigator to have any other factors that are not suitable for participating in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| based on local and systemic safety data;The average change in IOP (intraocular pressure) of the study eyes and non-study eyes ; | — |
Secondary
| Measure | Time frame |
|---|---|
| The changes in BCVA (Best Corrected Visual Acuity) of the study eyes and non-study eyes compared to the baseline were evaluated. ;The average stimulus threshold values measured by the full-field photopic threshold test (FST) for both the study eyes and (or) non-study eyes at each follow-up visit after treatment, compared with the baseline values;The changes in the visual function questionnaire (VFQ-25) scores of the subjects at each follow-up visit after treatment compared to the baseline values; | — |
Countries
China
Contacts
Public ContactJihong Wu
Fudan University Affiliated Eye, Ear and Nose Hospital
Outcome results
None listed