Gene therapy injection aims to restore sight in rare blindness
NCT ID NCT06952842
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called ZVS203e for people with retinitis pigmentosa caused by a specific RHO gene mutation. The therapy is injected into the eye and uses a harmless virus to deliver a gene-editing tool. The trial includes 18 adults and will check safety and whether it improves vision over 24 weeks.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ZVS203e (a gene therapy using a harmless virus to deliver a gene-editing tool into the eye)
- What this could lead to
- If successful, this could slow or partially reverse vision loss in people with a specific genetic form of retinitis pigmentosa.
- What could go wrong
- This is an early-phase trial with only 18 participants, so results may not apply to everyone. Gene therapies can cause inflammation or other side effects, and vision improvement may be limited or temporary.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2025
- Expected to finish
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Jun 2045
An estimate. End dates often move.
- Lead sponsor
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A research network
The lead sponsor is a research network or cooperative group.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Peking University Third Hospital
RECRUITINGBeijing, Beijing Municipality, 100191, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single eye injection restore sight in genetic blindness?
- A sharper look at the retina: new imaging technology may spot eye disease earlier
- Stem cell eye transplant aims to halt blindness from rare genetic disease
- Could stem cells restore sight in damaged eyes?
- Ground-Level vision test could help retinitis pigmentosa patients walk better
- New eye camera could unlock secrets of inherited blindness