Retinal Dystrophy Due to Biallelic ABCA4 MutationsPhase 1/2NCT07002398
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
A Phase 1/2 study testing VG801 in people with Retinal Dystrophy Due to Biallelic ABCA4 Mutations and Stargardt Disease 1, run by VeonGen Therapeutics GmbH at 1 site in China.
Trial summary
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
- What is being tested?
- VG801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 23 Dec 2024to Dec 2027
- Phase
- Phase 1/2
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible for study entry, subjects must satisfy all the following criteria:
- Written informed consent.
- Subjects aged ≥ 6 years.
- Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- Poor vision in the study eye.
Who cannot join
- Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- History of intraocular surgery within the previous 6 months.
- Previous participation in a gene therapy trial.
- Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
- Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shanghai General Hospital
Shanghai, Shanghai Municipality, 200080
Shanghai General Hospital, +86-021-36123569
Xiaodong Sun, MD
About this study
Official title: A Single Arm, Ph1/2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
- Being tested
- VG801
- Keywords
- ABCA4 mutation, Gene therapy, Eye disease, Stargardt disease, Retinal disease, Retinal degeneration, VG801
- Medical subject headings
- Stargardt Disease, Eye Diseases, Retinal Diseases, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VG801Drug
Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort
| Study group | Role | What they receive |
|---|---|---|
| VG801 | Experimental | Participants will receive a single dose of subretinal injection of VG801at Day 0. Receives: Drug: VG801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Abnormal examination results will be recorded.
Measured: Baseline to Month 12
Secondary outcomes (5)
Best Corrected Visual Acuity (BCVA)
Abnormal examination results will be recorded.
Measured: Screening to Month 12
Optical coherence tomography (OCT)
Abnormal examination results will be recorded.
Measured: Baseline to Month 12
Fundus autofluorescence
Abnormal examination results will be recorded.
Measured: Baseline to Month 12
Microperimetry
Abnormal examination results will be recorded.
Measured: Baseline to Month 12
Novel Virtual Reality Visual Test (Exploratory)
Measured: Baseline to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2025
- Study start23 Dec 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal Dystrophy Due to Biallelic ABCA4 MutationsStudies of the same condition, open and under way.
- 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- VeonGen Therapeutics GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Xiaodong Sun, MD, Principal investigator, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07002398
- Sponsor's protocol ID
- VG801-2022A
- Status last verified
- Aug 2026
- First submitted
- 23 May 2025
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Ph1/2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)
- ClinicalTrials.gov ID
- NCT07002398
- Acronym / sponsor ID
- VG801-2022A
- Keywords
- ABCA4 mutation, Gene therapy, Eye disease, Stargardt disease, Retinal disease, Retinal degeneration, VG801
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- VeonGen Therapeutics GmbHIndustry (pharma, biotech, devices)
- Study start
- 23 Dec 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 3 Jun 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.
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VG801 | Experimental | Drug: VG801Participants will receive a single dose of subretinal injection of VG801at Day 0. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Written informed consent.
- Subjects aged ≥ 6 years.
- Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- Poor vision in the study eye.
Key exclusion criteria (7 listed)
- Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- History of intraocular surgery within the previous 6 months.
- Previous participation in a gene therapy trial.
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 |
|---|---|---|
| VG801Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort | Drug | VG801 |
Primary outcome
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline to Month 12
Abnormal examination results will be recorded.
5 secondary outcomes
Best Corrected Visual Acuity (BCVA)
Time frame: Screening to Month 12
Optical coherence tomography (OCT)
Time frame: Baseline to Month 12
Fundus autofluorescence
Time frame: Baseline to Month 12
Microperimetry
Time frame: Baseline to Month 12
Novel Virtual Reality Visual Test (Exploratory)
Time frame: Baseline to Month 12
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 2 Sep 2026.
- Individual participant dataSharing planned: Undecided
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.
Xiaodong Sun, MD principal investigator
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VG801 in people with Retinal Dystrophy Due to Biallelic ABCA4 Mutations and Stargardt Disease 1, run by VeonGen Therapeutics GmbH at 1 site 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 6 inclusion and 7 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 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Research Coordinator (+86-021-36123569, shiyilunli@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline to Month 12). 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 2 Sep 2026.