ZVS203e eye gene-editing therapy for a specific RHO-related retinitis pigmentosa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a two-stage, open-label, single-arm Phase I/II trial to find a safe dose and then see if ZVS203e can help improve vision in adults with RHO-related retinitis pigmentosa. ZVS203e uses an AAV8 vector delivering CRISPR/Cas9 to silence the mutant RHO gene so retinal cells express normal protein. The study plans a small total enrollment (9–18 participants) with closely monitored safety and any signs of improvement in vision. Participants receive one subretinal injection and follow-up visits to assess tolerability, ocular health, and functional outcomes.

Why it matters

Who can join: the full rules

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:

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. Participants who had participated in any drug or medical device clinical trial within 3 months before enrollment;
6. 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.
7. 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).
8. Known allergy to the drug planned to be used in the study.

Where it's running

• Peking University Third Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Principal investigator: Hongliang Dou, M.D. — Peking University Third Hospital
• Study coordinator: Jinlu Zhang, MD
• Email: zhangjinlu@chinagene.cc
• Phone: 15810570898

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.