Gene therapy injection aims to halt vision loss in stargardt patients
NCT ID NCT07241169
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a gene therapy called ZVS106e for Stargardt disease, a genetic condition that causes vision loss. Nine participants will receive a single injection of the drug under their retina. The main goal is to check safety over 52 weeks, not yet to prove it works.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ZVS106e (a gene replacement drug delivered via a harmless virus into the eye)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops vision loss in people with Stargardt disease caused by ABCA4 mutations.
- What could go wrong
- This is a very early, small trial (9 people) focused on safety, not effectiveness. The treatment involves eye surgery, which carries risks like infection or retinal damage. Success is far from guaranteed.
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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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 9 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2025
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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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8 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. Age ≥8 years old; 2. Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases; 3. The target eye must meet the following requirements: the best corrected visual acuity is 0.5 to 2.0 LogMAR (including 0.5 and 2.0 LogMAR, equivalent to the decimal visual acuity index up to 0.3); 4. The subject and his or her spouse agree to take effective contraceptive measures during the trial period and for at least one year after administration. 5. Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol. Exclusion Criteria: 1. The researchers determined that the target eye currently has or has previously had other macular lesions such as retinal schisis or epiretinal membrane. Or have other eye diseases that may hinder the surgery or interfere with the interpretation of the study endpoint; 2. Having received drug treatment that may affect the observation of the trial within the three months prior to screening; 3. The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy; 4. There are known eye/visual diseases, disorders or lesions that cause or are related to vision loss, or whose related treatments or therapies are known to cause or are related to vision loss; 5. Having suffered from a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or having received an antiviral vaccine within one month prior to enrollment; 6. Systemic medications that are currently in use or may be required to cause eye toxicity, such as psoralen, risselinic acid or tamoxifen, etc. 7. Known to be allergic to the drugs planned to be used in the study; 8. Suffering from poorly drug-controlled hypertension: systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg; 9. The following laboratory test abnormalities have clinical significance: Liver function: Chronic liver disease, elevation of ALT or AST \>2 times the upper limit of the normal value; Those with abnormal coagulation function (prothrombin time ≥ 3 seconds above the upper limit of the normal value, activated partial thromboplastin time ≥ 10 seconds above the upper limit of the normal value); Serum virological examination: Positive for active hepatitis B, hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), or syphilis antibody; 10. There is any past or current medical history that may affect the safety of the trial or the in vivo process of the drug, especially a history of diseases such as cardiovascular, liver, kidney, endocrine, digestive tract, lung, nervous, hematological, tumor, immune or metabolic disorders that the investigators consider to have clinical significance; 11. Those who have participated in any clinical trials of drugs or medical devices within the three months prior to screening; 12. Pregnant or lactating women; According to the researcher's judgment, those who are deemed unsuitable to participate in this clinical trial for other reasons
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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
2 sites. 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, China
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Zhongshan Ophthalmic Center, Sun Yat-sen University
NOT_YET_RECRUITINGGuangzhou, Guangdong, China
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