RHO-adRP
Conditions
Interventions
Low-dose group:Low dose of OPGx-RHO injection
High-dose group:High dose of OPGx-RHO injection
Sponsors
Peking Union Medical College Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 100 Years
Inclusion criteria
Inclusion criteria: 1.Willingness to provide informed consent and adhere to schedule of study assessments; 2.Age >=18 years at the time of informed consent; 3.Diagnosis of RHO-adRP based on the following: a. Clinical diagnosis of retinitis pigmentosa (RP) based on history and visual function. b. Genetic testing for RHO mutations associated with adRP; 4.Ability to perform tests of visual and retinal function and structure; 5.Detectable rod photoreceptor–mediated vision in the intended treatment region, assessed by microperimetry; 6.For the sentinel participant in each cohort, BCVA 20/80 or worse on the ETDRS visual acuity chart in the eye to be treated. For all subsequent participants in each cohort, BCVA 20/40 or worse in the eye to be treated; 7.Participants and their partners agreed to take effective contraceptive measures throughout the entire study period and for at least 12 months after taking the drug;
Exclusion criteria
Exclusion criteria: 1. The study eye has opacity of the refractive medium that significantly interferes with visual acuity detection, anterior segment or fundus assessment, or the pupil cannot be dilated; 2. Diabetic retinopathy, retinal vein occlusion, pathological myopia, retinal detachment, macular hole, epimacular membrane, macular retinal atrophy and other diseases in the study eye, which are assessed by the investigator to affect the safety of the subjects or the evaluation of study effectiveness; 3. Active intraocular or periocular infection (such as blepharitis, conjunctivitis, keratitis, scleritis, etc.) in the study eye; 4. History of vitreous hemorrhage in the study eye within 6 months before screening; 5. Significant (as judged by the investigator) ocular surgery in the study eye within 6 months prior to screening; 6. History of glaucoma in any eye; 7. History of uveitis in any eye; 8. BCVA detected by ETDRS eye chart in the monocular or non-study eye is less than 4 letters (equivalent to 20/800 of the Snellen eye chart); 9. Those with diffuse intravascular coagulation and obvious bleeding tendency (such as hemoptysis, hematemesis, severe purpura, etc.) within 3 months before screening; 10. History of myocardial infarction, unstable angina, coronary revascularization, cerebrovascular accident (including TIA), history of other thromboembolic diseases (such as thromboembolic vasculitis, pulmonary embolism, deep vein thrombosis, portal vein thrombosis, etc.) within 6 months before screening, New York Heart Association (NYHA) grade >= Class II cardiac insufficiency, severe unstable ventricular arrhythmia; 11. Those with systemic immune diseases (including systemic lupus erythematosus, ankylosing spondylitis, rheumatoid arthritis, etc.); 12. Diabetic patients with any of the following conditions: known macrovascular complications; Glycated hemoglobin (HbA1c) > 7.5% at screening; Those who are receiving more than two oral hypoglycemic drugs or receiving insulin or GLP-1 receptor agonists; 13. Hypertension and poor blood pressure control (defined as: systolic blood pressure >= 160 mmHg or diastolic blood pressure >= 100 mmHg when sitting after receiving antihypertensive drugs); 14. Any uncontrollable clinical disease (such as severe psychiatric, respiratory and other system diseases and history of malignant tumors); 15. Those with abnormal liver and kidney function: alanine aminotransferase (ALT)/aspartate aminotransferase (AST) > = 2 times the upper limit of normal value; Total bilirubin >= 1.5 times the upper limit of normal; Creatinine, urea/urea nitrogen >= 1.5 times the upper limit of normal; 16. Abnormal coagulation function: prothrombin time (PT) > upper limit of normal value 3 seconds or activated partial thromboplastin time (APTT) > upper limit of normal value 10 seconds; Hemoglobin (Hb) < 10 g/dL; 17. Individuals positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, syphilis treponemal antibody, or human immunodeficiency virus (HIV) antibody; 18. Individuals known to be allergic to the therapeutic or diagnostic drugs used in the study, including study drugs; 19. Individuals who have used systemic corticosteroids or other immunosuppressive drugs within 3 months prior to screening; 20. Individuals who have received vaccinations within 2 weeks prior to dosing, or are expected to require vaccination while using glucocorticoids; 21. Individuals who have used anticoagulant drugs within 14 days prior to dosing; 22
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number and severity of adverse events (AE);Number of DLT events; | — |
Secondary
| Measure | Time frame |
|---|---|
| the Michigan Retinal Degeneration Questionnaire (MRDQ);Mean sensitivity;Retinal morphology: thickness of the ellipsoid zone, thickness and segmentation of the outer nuclear layer;BCVA and LLVA; | — |
Countries
China
Contacts
Public ContactRuifang Sui
Peking Union Medical College Hospital
Outcome results
None listed