Bietti Crystalline DystrophyPhase 1/2NCT07586306
A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)
A Phase 1/2 study testing VGR-R01 in people with Bietti Crystalline Dystrophy, run by Shanghai Vitalgen BioPharma Co., Ltd. at 2 sites in China.
Trial summary
This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Bietti Crystalline Dystrophy
- What is being tested?
- VGR-R01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesChina
- Study dates
- 1 Jun 2026to 31 Jul 2031
- 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 18 to 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
- Able to provide informed consent and comply with requirements of the study;
- Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.
Who cannot join
- Have insufficient viable retinal photoreceptor cells based on investigator's decision;
- Have current ocular or periocular infections, or endophthalmitis;
- Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
- Have intraocular surgery history except cataract surgery in the study eye;
- Have or potentially require of systemic medications that may cause eye injure;
- Have contraindications for corticosteroids or immunosuppressant;
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- Abnormal coagulation function or other clinically significant abnormal laboratory results;
- Have malignancies or history of malignancies;
- History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
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
Beijing Tongren Hospital
Beijing, Beijing Municipality
Yan Jiang, 086-15802234907, yan.jiang@vitalgen.com
Shanghai General Hospital (Shanghai First People's Hospital)
Shanghai, Shanghai Municipality
Yan Jiang, 086-15802234907, yan.jiang@vitalgen.com
About this study
Official title: A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration
This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.
Read the detailed description
VGR-R01 is a novel Adeno-associated virus (AAV) vector carrying the human Cytochrome P450 Family 4 Subfamily V Member 2 (CYP4V2) coding sequence. This study will evaluate the safety and efficacy of VGR-R01 administered in the contralateral eye (the second treated eye) of subjects enrolled in the VGR-R01-001 and VGR-R01-101 studies, along with assessments of immunogenicity and vector shedding. Additionally, the long-term safety and efficacy of VGR-R01 treatment will be continuously assessed for up to 5 years after the last dose.
- Conditions
- Bietti Crystalline Dystrophy
- Being tested
- VGR-R01
- Keywords
- VGR-R01, CYP4V2, gene therapy
- Medical subject headings
- Bietti Crystalline Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VGR-R01Biological
CYP4v2-coding gene delivered by AAV vector
| Study group | Role | What they receive |
|---|---|---|
| VGR-R01 group | Experimental | Single-dose Subretinal Administration of VGR-R01 Receives: Biological: VGR-R01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events and serious adverse events
Ocular/non-ocular adverse events are collected. The ophthalmic examination will include Best Corrected Visual Acuity (BCVA), Intraocular Pressure (IOP), slit lamp examination, angiography and Optical Coherence Tomography (OCT), etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.
Measured: Up to Year 5
Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings
Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 10 minutes. Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. Ophthalmic Examination will include BCVA, IOP, slit lamp examination, angiography and OCT, etc. Clinical significance of the above signs will be determined at the investigator's discretion.
Measured: Up to Year 5
Secondary outcomes (7)
Change from baseline in BCVA
BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart..
Measured: Year 5
Change from baseline in multi-luminance mobility test (MLMT) score
Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. All light-levels used for testing will be rounded to one of the following specified light levels: 0.1, 1, 4, 10, 50, 125, 250, or 400 lux. The corresponding scores for the above light levels range from 7 to 0, in descending order. -1 point means failing to pass the test at the 400 lux level; the lower the score, the worse the functional vision of the participant.
Measured: Year 5
Change from baseline in optical coherence tomography (OCT)
Change from baseline in central retinal thickness (CRT) as imaged by OCT.
Measured: Year 5
Number of subjects with the presence of immunogenicity
Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product.
Measured: Year 5
Number of subjects with the presence of vector shedding
Assessed as the presence of vector in peripheral blood or collected tear.
Measured: Year 5
Fixation stability
Number of treated eyes with changes from baseline in fixation stability with MP-3 Microperimetry. Fixation stability is rated into three levels: stable fixation, relatively unstable fixation, and unstable fixation, with fixed standard reporting on the device settings.
Measured: Year 5
Light sensitivity
Changes from baseline in light sensitivity with MP-3 Microperimetry. The MP-3 has a stimulus intensity range of 0 to 34 decibels (dB).
Measured: Year 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start1 Jun 2026 (estimated)
- Primary completion31 Jul 2027 (estimated)
- Study completion31 Jul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bietti Crystalline DystrophyStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wenbin Wei, Principal investigator, Beijing Tongren Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07586306
- Sponsor's protocol ID
- VGR-R01-102
- Status last verified
- Apr 2026
- First submitted
- 23 Apr 2026
- Last updated
- 14 May 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 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration
- ClinicalTrials.gov ID
- NCT07586306
- Acronym / sponsor ID
- VGR-R01-102
- Conditions
- Bietti Crystalline Dystrophy
- Keywords
- VGR-R01, CYP4V2, gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Jun 2026 (estimated)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 31 Jul 2031 (estimated)
- First posted
- 14 May 2026 (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 multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.
Detailed description
VGR-R01 is a novel Adeno-associated virus (AAV) vector carrying the human Cytochrome P450 Family 4 Subfamily V Member 2 (CYP4V2) coding sequence. This study will evaluate the safety and efficacy of VGR-R01 administered in the contralateral eye (the second treated eye) of subjects enrolled in the VGR-R01-001 and VGR-R01-101 studies, along with assessments of immunogenicity and vector shedding. Additionally, the long-term safety and efficacy of VGR-R01 treatment will be continuously assessed for up to 5 years after the last dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGR-R01 group | Experimental | Biological: VGR-R01Single-dose Subretinal Administration of VGR-R01 |
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
- Able to provide informed consent and comply with requirements of the study;
- Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.
Key exclusion criteria (9 listed)
- Have insufficient viable retinal photoreceptor cells based on investigator's decision;
- Have current ocular or periocular infections, or endophthalmitis;
- Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
- Have intraocular surgery history except cataract surgery in the study eye;
- Have or potentially require of systemic medications that may cause eye injure;
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 |
|---|---|---|
| VGR-R01CYP4v2-coding gene delivered by AAV vector | Biological | VGR-R01 group |
Primary outcome
Incidence of adverse events and serious adverse events
Time frame: Up to Year 5
Ocular/non-ocular adverse events are collected. The ophthalmic examination will include Best Corrected Visual Acuity (BCVA), Intraocular Pressure (IOP), slit lamp examination, angiography and Optical Coherence Tomography (OCT), etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.
Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings
Time frame: Up to Year 5
Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 10 minutes. Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. Ophthalmic Examination will include BCVA, IOP, slit lamp examination, angiography and OCT, etc. Clinical significance of the above signs will be determined at the investigator's discretion.
7 secondary outcomes
Change from baseline in BCVA
Time frame: Year 5
Change from baseline in multi-luminance mobility test (MLMT) score
Time frame: Year 5
Change from baseline in optical coherence tomography (OCT)
Time frame: Year 5
Number of subjects with the presence of immunogenicity
Time frame: Year 5
Number of subjects with the presence of vector shedding
Time frame: Year 5
Fixation stability
Time frame: Year 5
Light sensitivity
Time frame: Year 5
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 14 May 2026.
- Individual participant dataSharing planned: No
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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.
Wenbin Wei principal investigator
Beijing Tongren Hospital
2 sites 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 VGR-R01 in people with Bietti Crystalline Dystrophy, run by Shanghai Vitalgen BioPharma Co., Ltd. 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 3 inclusion and 9 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: opening soon, as of the sponsor's last update on 14 May 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 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 Yan Jiang (086-15802234907, yan.jiang@vitalgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events and serious adverse events (time frame: Up to Year 5). The study lists 2 primary outcomes in all. 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 14 May 2026.