Retinitis pigmentosa
Conditions
Interventions
Treatment group:Subjects who were successfully enrolled were given 0.5mg/kg body weight/day orally one week before receiving study treatment, and in the first week after treatment, the dose was increa
At the 6~8th week after treatment, the dose is reduced to 0.125mg/kg body weight/day. Patients with oral corticosteroids should have a daily dose of < = 60 mg to prevent corticosteroid overdose.
On th
Sponsors
The First Affiliated Hospital of Suzhou University
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. Voluntarily sign an informed consent form (ICF); 2. Subject age>=18 and = 0.02, < = 0.12 (best corrected visual acuity is examined using an international standard eye chart); 5. Optical coherence tomography (OCT) was used to measure the relative retention of ganglion cell complex thickness and retinal nerve fiber layer thickness, and the investigator was invited to evaluate and enroll; 6. Preserve previously available visual memory; 7. The pupils are sufficiently dilated for a comprehensive eye examination and testing; 8. Negative serum pregnancy test result for females of childbearing age; 9. Ocular refractive error between -9D and +6D.
Exclusion criteria
Exclusion criteria: Subjects who meet any of the following exclusion criteria are considered ineligible for enrollment: 1. Previous receipt of any gene therapy; 2. Subjects have received other interventional trial drug clinical trials within 90 days prior to screening; 3. Subject has symptoms and/or related treatments that in the opinion of the investigator can alter visual function, including but not limited to glaucoma and central nervous system lesions (patients with RP with cataracts that do not seriously affect vision can be included); 4. Subject has a history of other malignancies within 5 years, except: subjects with squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ, and papillary thyroid carcinoma of the skin that has been thoroughly treated and has no signs of recurrence; 5. Any serious systemic disease judged by the investigator, whose symptoms or relevant treatments received may affect the results of this study trial (such as safety, visual acuity); 6. Significant ocular surgery (as determined by the investigator) within 3 months prior to screening; 7. Presence of ocular refractive media disease interfering with visual acuity and other ocular assessments (including OCT/OCTA) during the study; 8. Patient is receiving or may be receiving immunosuppressive therapy other than this study; 9. Patients of childbearing potential who refuse to use effective contraception for 12 months after AAV injection; 10. Pregnant or lactating subjects; 11. Subjects who are unwilling or unable to comply with this study protocol; 12. Subject has any condition that, in the opinion of the investigator, does not allow him to complete follow-up examinations during the study and makes him unsuitable for continued participation in the study; 13. Subjects who are HIV antibody positive and Treponema pallidum antibody positive, as well as subjects with active hepatitis B infection and active hepatitis C infection; 14. Subject is allergic to the study treatment drug or its excipient components; 15. Subjects who are allergic to glucocorticoids, or unable to tolerate glucocorticoid regimens, or have active intercurrent infections for which treatment is contraindicated; 16. Suffering from retinal detachment and other fundus diseases that are not suitable for this study; 17. There are contraindications to pupil dilation, such as iris corneal angle (anterior chamber angle) stenosis, etc.; 18. Prior vitrectomy or vitreomacular surgery; 19. Subjects who, in the opinion of the investigator, have any other factors that are not suitable for participating in 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;Best Corrected Visual Acuity, Visual Field, Visual Electrophysiology (FST, ERG, VEP); | — |
Countries
China
Contacts
Public ContactLu Peirong
The first affiliated hospital of Suzhou university
Outcome results
None listed