StargardtEarly Phase 1ZVS106eNCT07241169
The Safety and Efficacy of ZVS106e in the Treatment of IRDs Caused by Biallelic Mutations in ABCA4
A Early Phase 1 study testing ZVS106e in people with Stargardt, run by Zhongshan Ophthalmic Center, Sun Yat-sen University at 2 sites in China.
Trial summary
To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Stargardt
- What is being tested?
- ZVS106e
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesChina
- Study dates
- 29 Dec 2025to 30 Dec 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥8 years old;
- Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases;
- 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);
- 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.
- Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol.
Who cannot join
- 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;
- Having received drug treatment that may affect the observation of the trial within the three months prior to screening;
- The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy;
- 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;
- 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;
- Systemic medications that are currently in use or may be required to cause eye toxicity, such as psoralen, risselinic acid or tamoxifen, etc.
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- Known to be allergic to the drugs planned to be used in the study;
- Suffering from poorly drug-controlled hypertension: systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg;
- 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;
- 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;
- Those who have participated in any clinical trials of drugs or medical devices within the three months prior to screening;
- Pregnant or lactating women; According to the researcher's judgment, those who are deemed unsuitable to participate in this clinical trial for other reasons
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Peking University Third Hospital
Beijing, Beijing Municipality
Jinlu Zhang, MD, +86-15810570898, zhangjinlu@chinagene.cc
- Not yet recruiting
Zhongshan Ophthalmic Center, Sun Yat-sen University
Guangzhou, Guangdong
Jinlu Zhang, MD, +86-15810570898, zhangjinlu@chinagene.cc
About this study
Official title: A Preliminary Clinical Study on the Safety and Efficacy of the Gene Replacement Drug ZVS106e in the Treatment of Hereditary Retinal Degeneration (IRDs) Caused by ABCA4 Biallelic Mutations
To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations.
Read the detailed description
The main research objective: To evaluate the preliminary safety of single subretinal injection of ZVS106e injection in one eye for patients with hereditary retinal degeneration (ABCA4-IRDS) caused by biallelic mutations in ABCA4. Secondary research objective: To preliminarily explore the clinical efficacy of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations. The purpose of the exploratory study is to evaluate the improvement of retinal structure and visual function in non-injection eyes compared to the baseline.
- Conditions
- Stargardt
- Being tested
- ZVS106e
- Keywords
- ZVS106e
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZVS106eDrug
Also called rAAV8
ZVS106e is A colorless, clear and transparent liquid, containing two active ingredients, ZVS106E-A and ZVS106E-B. The viral vectors used for both active ingredients are rAAV8
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | All patients enrolled in the study will receive a single subretinal injection of ZVS106e in one eye Receives: Drug: ZVS106e |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the safety and tolerability of subretinal injection of ZVS106e solution
Types, severity, and incidence of adverse events (AE) and serious adverse events (SAE) in the eyes and throughout the body within 52 weeks post-treatment, including dose-limiting toxicities (DLT) during the dose escalation phase.
Measured: 52 weeks post-treatment
Secondary outcomes (4)
Change from baseline in best-corrected visual acuity (BCVA)
Change in best-corrected visual acuity (BCVA) of the treated eye at 52 weeks compared to baseline.
Measured: 52 weeks post-treatment
Change from Baseline in Visual function metrics
Treatment outcomes for visual function metrics include changes from baseline in LLVA, dynamic visual field, microperimetry, FST, contrast sensitivity, color vision, and mfERG; as well as changes from baseline in the NEI-VFQ-25 score reported by the participants.
Measured: 52 weeks post-treatment
Change from Baseline in OCT
Compare changes in retinal morphology and alterations in cell layers of the retina before and after drug administration.
Measured: 52 weeks post-treatment
Change from Baseline in Fundus autofluorescence (FAF)
Compare the atrophy of the RPE/ photoreceptor complex in the subjects before and after administration
Measured: 52 weeks post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2025
- Study start29 Dec 2025
- Primary completion30 Nov 2028 (estimated)
- Study completion30 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for StargardtStudies of the same condition, open and under way.
- More trials run by Zhongshan Ophthalmic Center, Sun Yat-sen UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Chigenovo Co., Ltd
- Responsible party
- Principal investigator, Qingjiong Zhang, Zhongshan Ophthalmic Center, Sun Yat-sen University
- Investigators
- qingjiong zhang, M.D, Study director, PIlin Lv, M.D, Principal investigator, PI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07241169
- Sponsor's protocol ID
- ZYA-2025-002
- Acronym
- ZVS106e
- Status last verified
- Apr 2026
- First submitted
- 29 Sep 2025
- Last updated
- 16 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Preliminary Clinical Study on the Safety and Efficacy of the Gene Replacement Drug ZVS106e in the Treatment of Hereditary Retinal Degeneration (IRDs) Caused by ABCA4 Biallelic Mutations
- ClinicalTrials.gov ID
- NCT07241169
- Acronym / sponsor ID
- ZVS106e / ZYA-2025-002
- Conditions
- Stargardt
- Keywords
- ZVS106e
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Zhongshan Ophthalmic Center, Sun Yat-sen UniversityUniversity, hospital or other
- Collaborators
- Chigenovo Co., Ltd
- Study start
- 29 Dec 2025 (actual)
- Primary completion
- 30 Nov 2028 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 21 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations.
Detailed description
The main research objective: To evaluate the preliminary safety of single subretinal injection of ZVS106e injection in one eye for patients with hereditary retinal degeneration (ABCA4-IRDS) caused by biallelic mutations in ABCA4. Secondary research objective: To preliminarily explore the clinical efficacy of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations. The purpose of the exploratory study is to evaluate the improvement of retinal structure and visual function in non-injection eyes compared to the baseline.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Drug: ZVS106eAll patients enrolled in the study will receive a single subretinal injection of ZVS106e in one eye |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥8 years old;
- Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases;
- 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);
- 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.
- Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol.
Key exclusion criteria (13 listed)
- 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;
- Having received drug treatment that may affect the observation of the trial within the three months prior to screening;
- The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy;
- 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;
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZVS106eAlso: rAAV8ZVS106e is A colorless, clear and transparent liquid, containing two active ingredients, ZVS106E-A and ZVS106E-B. The viral vectors used for both active ingredients are rAAV8 | Drug | Single arm |
Primary outcome
Evaluate the safety and tolerability of subretinal injection of ZVS106e solution
Time frame: 52 weeks post-treatment
Types, severity, and incidence of adverse events (AE) and serious adverse events (SAE) in the eyes and throughout the body within 52 weeks post-treatment, including dose-limiting toxicities (DLT) during the dose escalation phase.
4 secondary outcomes
Change from baseline in best-corrected visual acuity (BCVA)
Time frame: 52 weeks post-treatment
Change from Baseline in Visual function metrics
Time frame: 52 weeks post-treatment
Change from Baseline in OCT
Time frame: 52 weeks post-treatment
Change from Baseline in Fundus autofluorescence (FAF)
Time frame: 52 weeks post-treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2026.
- Individual participant dataSharing planned: No
Related trials for Stargardt
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A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
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Active, not recruitingPhase 1/2
Oral Metformin for Treatment of ABCA4 Retinopathy
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A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
qingjiong zhang, M.D study director
PI
lin Lv, M.D principal investigator
PI
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing ZVS106e in people with Stargardt, run by Zhongshan Ophthalmic Center, Sun Yat-sen University at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact dayou Ding (010-82266699, dingdayou@chinagene.cc), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety and tolerability of subretinal injection of ZVS106e solution (time frame: 52 weeks post-treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2026.