Safety and Efficacy of ZVS203e in the Treatment of Retinitis Pigmentosa Caused by RHO Gene Mutation
A Single-Arm, Open-Label, Phase 1/2 Clinical Trial of ZVS203e in Subjects With Retinitis Pigmentosa Associated With RHO Mutation
Chigenovo Co., Ltd
18 participants
Aug 4, 2025
INTERVENTIONAL
Conditions
Summary
This trial employs a single-arm, open-label seamless Phase I/II design, consisting of two stages: Phase I dose exploration and Phase II dose expansion.The primary objective of this trial is to evaluate the safety, tolerability, and efficacy of subretinal injection of ZVS203e solution.
Eligibility
Inclusion Criteria6
- Patients with a clinical diagnosis of retinitis pigmentosa (RP) (aged 18 years or older);
- RHO (c.403C>T, p.R135W) gene site-specific mutation was confirmed by genetic testing, and no other ophthalmic genetic diseases were complicated;
- The researchers judged that the target eye had viable retinal photoreceptor cells and retinal pigment epithelial cells;
- 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);
- The subject and his or her spouse agree to use effective contraception during the trial period and for at least 1 year after dosing;
- Voluntarily participate in clinical trials and sign informed consent, and can complete the whole test process according to the protocol requirements.
Exclusion Criteria8
- The researcher determined that the target eye currently has or had macular lesions such as macular hiatal hole or macular neovascularization;
- 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.
- Within 3 months prior to enrollment, the study eye had received any intraocular surgery, such as phacoemulsification cataract extraction.
- The study eye had undergone retinal reattachment or vitrectomy.
- Participants who had participated in any drug or medical device clinical trial within 3 months before enrollment;
- 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.
- 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).
- Known allergy to the drug planned to be used in the study.
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Interventions
ZVS203e injection is a clear, transparent liquid containing a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses humanized SauriCas9 protein and single guide RNA (sgRNA) targeting specific mutations in the RHO gene.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT06952842