Autosomal dominant retinitis pigmentosa
Conditions
Interventions
I Phase:Three preset dosage groups of low, medium, and high (4.5×10^10 vg/eye, 7.5×10^10 vg/eye, 1.5×10^11 vg/eye) are established, with the initial dosage set as the medium dose. After all subjects i
if significant toxic side effects are observed, they will move to the low dose group. Each dose group is expected to enroll 3 cases, referencing the traditional 3+3 dose escalation principle with appr
II Phase:The subjects were administered ZVS203e injection after preventive hormone use by intraretinal injection.
Sponsors
Peking University Third Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with a clinical diagnosis of retinitis pigmentosa (RP) (aged 18 years or older); 2. RHO (c.403C>T, p.R135W) gene site-specific mutation was confirmed by genetic testing, and no other ophthalmic genetic diseases were complicated; 3. The researchers judged that the target eye had viable retinal photoreceptor cells and retinal pigment epithelial cells; 4. The best corrected visual acuity of the target eye is between 2.0 LogMAR and 0.5 LogMAR (including 2.0 LogMAR and 0.5 LogMAR, which is equivalent to a number of fingers to 60 letters); 5. The subject and his or her spouse agree to use effective contraception during the trial period and for at least 1 year after dosing; 6. Voluntarily participate in clinical trials and sign informed consent, and can complete the whole test process according to the protocol requirements.
Exclusion criteria
Exclusion criteria: 1. The researcher determined that the target eye currently has or had macular lesions such as macular hiatal hole or macular neovascularization; 2. Have other eye conditions that may prevent surgery or interfere with interpretation of the study endpoint, such as glaucoma, diabetic retinopathy, eye or periocular infections, active endophthalmitis, etc. 3.Within 3 months prior to enrollment, the study eye had received any intraocular surgery, such as phacoemulsification cataract extraction. 4.The study eye had undergone retinal reattachment or vitrectomy. 5. Had a viral infectious disease that may affect the efficacy and safety evaluation of the experimental drug within 1 month before enrollment; 6. Participants who had participated in any drug or medical device clinical trial within 3 months before enrollment; 7.Previously treatment of either eye with gene therapy or stem cell therapy for RP and other ocular diseases, including but not limited to viral vector gene therapy, RNA therapy. 8.Treatment with medications that may affect the efficacy and safety evaluation of the investigational product within 3 months prior to enrollment (e.g., ranibizumab, bevacizumab, aflibercept, conbercept). 9.Currently taking or likely to require systemic medications that can cause ocular toxicity, such as psoralen, risedrin, or tamoxifen. 10. Had used systemic corticosteroids within 1 month before enrollment, with a course longer than 7 days or a dose greater than 1.5 mg/Kg/ day; 11. Patients with severe gastrointestinal ulcers, osteoporosis and other diseases that are not suitable for systemic corticosteroid treatment; 12.Known allergy to the drug planned to be used in the study. 13.Clinically significant laboratory abnormalities: ALT or AST > 2 times upper limit of normal; Abnormal coagulation function (prothrombin time>=upper limit of normal 3 seconds, activated partial thromboplastin time>= upper limit of normal 10 seconds); Serum virological examination: active hepatitis B, hepatitis C virus antibody (HCV-Ab) or human immunodeficiency virus antibody (HIV-Ab) or syphilis antibody positive. 14.Having any past or present medical history that may affect the safety evaluation of the trial drug or the in vivo process of the trial drug, especially any clinically significant cardiovascular, hepatic, renal, endocrine, digestive, respiratory, nervous, hematological, immune, metabolic system and other diseases or tumor history as considered by the investigator. 15.Have or have had systemic damaging immune diseases. 16.Pregnant or lactating women. 17.Patients who are considered unsuitable for participating in this clinical trial for other reasons by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in BCVA at Week 24;The types, severity, and incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) within 24 weeks after treatment (including dose-limiting toxicities during the dose escalation phase).; | — |
Countries
China
Contacts
Public ContactDou Hongliang
Peking University Third Hospital
Outcome results
None listed