Retinitis pigmentosa
Conditions
Interventions
Intervention/Treatment :Intravitreal injection
Sponsors
The First Affiliated Hospital of Soochow University
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. voluntarily sign the informed consent form (ICF); 2. subjects aged = 18 and = 60 years at the time of signing the ICF; 3. Confirmed diagnosis of nonsyndromic RP, defined as: (1)Clinically diagnosed as nonsyndromic RP based on history, visual dysfunction, and funduscopy examination. (2) Confirmed diagnosis of nonsyndromic RP by OCT, FFA, and/or full-field ERG (any previously performed ERG is acceptable). 4. visual acuity: best-corrected visual acuity < 0.02 in the eye with lower visual acuity; 5. measure the preserved ganglion cell layer volume and retinal nerve fiber layer thickness using optical coherence tomography (OCT), enrollment is based on investigator's decision; 6. preserve previously available visual memory; 7. The pupils can be fully dilated for comprehensive ophthalmic examination and testing; 8. negative serum pregnancy test in women of childbearing age; 9. refractive error between -9D and + 6D.
Exclusion criteria
Exclusion criteria: 1. Previously received any prior gene therapy; 2. the subject has received other interventional investigational drug clinical trials within 90 days before screening; 3. Subjects with diseases or conditions that, in the opinion of the investigator, can alter visual function and/or related treatments, including but not limited to glaucoma and central nervous system lesions (RP patients with cataracts that do not seriously affect vision may be included); 4. the subject has a history of other malignancies within 5 years, except: thoroughly treated skin squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ and papillary thyroid carcinoma without signs of recurrence; 5. any serious systemic disease judged by the investigator, whose symptoms or relevant treatment received may affect the test results of this study (such as safety, vision); 6. major ocular surgery (as determined by the investigator) within 3 months before screening; 7. Ocular refractive media disease that interferes with visual acuity and other ocular assessments (including OCT/OCTA) during the study; 8. patients are receiving or may receive immunosuppressive therapy other than this study; 9. patients of childbearing potential refuse to take effective contraceptive measures within 12 months after injection of AAV; 10. subjects who are pregnant or lactating; 11. subjects who are unwilling or unable to comply with this study protocol; 12. subjects have any condition that, in the opinion of the investigator, does not allow them to complete follow-up examinations during the study period and makes them unsuitable for continued participation in the study; 13. subjects that are HIV antibody positive and Treponema pallidum antibody positive, as well as subjects with active hepatitis B infection or active hepatitis C infection ; 14. subjects allergic to the study treatment or its excipients; 15. subjects allergic to glucocorticoids, or unable to tolerate glucocorticoid regimens, or have active concurrent infection that contraindicates treatment; 16. subjects with retinal detachment and other fundus diseases not suitable for this study; 17. mydriatic contraindications, such as iridocorneal angle (anterior chamber angle) stenosis; 18. previous vitrectomy or vitreomacular surgery; 19. the investigator determines that the subject has any other factors that are not suitable for this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of AE;Compared to baseline, the change of central foveal thickness after the intervention tested by OCT;Compared to baseline, the change of central macular thickness after the intervention tested by OCT;Compared to baseline, the change of mean IOP of the treated and untreated eyes; | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Group with light perception at baseline: change in mean BCVA in treated and non-treated eyes after treatment relative to baseline;;2. Group with no light perception at baseline: the proportion of subjects that gain light perception of BCVA and change in their visual acuity in the treated and non-treated eyes after treatment relative to baseline.;Proportion of subjects with BCVA improvement from baseline in treated and non-treated eyes respectively following treatment;;Change from baseline in mean stimulation threshold measured by full-field threshold stimulation test (FST) in treated and/or non-treated eyes following treatment (Note: non-treated eyes may be selectively tested as clinically indicated during the study);;Change in visual function questionnaire (VFQ-25) score after study treatment from baseline; | — |
Countries
China
Contacts
Public ContactPeirong Lu
Outcome results
None listed