Bietti Crystalline DystrophyPhase 3NCT06699108
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
A Phase 3 study testing VGR-R01 in people with Bietti Crystalline Dystrophy and Inherited Retinal Diseases, run by Shanghai Vitalgen BioPharma Co., Ltd. at 1 site in China.
Trial summary
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Bietti Crystalline Dystrophy, Inherited Retinal Diseases
- What is being tested?
- VGR-R01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteChina
- Study dates
- 13 Dec 2024to 30 Jun 2027
- Phase
- Phase 3
- 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
- Able to provide informed consent and comply with requirements of the study;
- ≥18 years and <70 years of age;
- Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
- Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study 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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- Unwilling or unable to have the planned follow-up;
- 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
1 site in 1 country.
China1 site
Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai, Shanghai Municipality
About this study
Official title: A Multi-Center, Randomized, Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of VGR-R01 in Subjects With Bietti Crystalline Dystrophy
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy.
This is a multicenter, randomized controlled study which will enroll 45 subjects.
Read the detailed description
VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to assess the efficacy of VGR-R01 in subjects with Bietti crystalline dystrophy (BCD) based on the change from baseline in best corrected visual acuity (BCVA) of the study eyes.
30 subjects will be enrolled in the intervention group and will receive monocular administration of VGR-R01, and 15 subjects in the control group will not receive any intervention. After completion of the trial, the untreated eyes will receive administration of VGR-R01 in subsequent clinical trial.
- Being tested
- VGR-R01
- 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-R01Drug
Subretinal injection of VGR-R01 (0.1 mL)
| Study group | Role | What they receive |
|---|---|---|
| interventional group | Experimental | Not described Receives: Drug: VGR-R01 |
| Control group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS
Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls
Measured: Baseline up to Week 52
Secondary outcomes (6)
Change from baseline in BCVA
BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. Testing Protocol will be followed (confidential). Change in the BCVA assessment from baseline in treated eyes of intervention group will be compared to controls.
Measured: Baseline up to Week 52
Change from baseline in multi-luminance mobility test (MLMT) score of the study eye
Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. The mobility testing will follow a standardized administration and data acquisition protocol and may only be administered by site staff certified in the methodology.
Measured: Baseline up to Week 52
Changes from baseline in Microperimetry indexes
Number of subjects with change in fixation stability or light sensitivity in treated eyes of intervention group will be compared to controls
Measured: Baseline up to Week 52
Change from baseline in optical coherence tomography (OCT)
Change from baseline in central retinal thickness (CRT) as imaged by OCT
Measured: Baseline up to Week 52
Counts, frequencies and percentages of AE, SAE and other safety evaluation
Ocular/non-ocular adverse events are collected. The ophthalmic examination will include BCVA, IOP, slitlamp examination, angiography and SD-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: Baseline up to Week 52
Number of subjects with the presence of immunogenicity
Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product
Measured: Baseline up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2024
- Study start13 Dec 2024
- Primary completion1 Dec 2026 (estimated)
- Study completion30 Jun 2027 (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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06699108
- Sponsor's protocol ID
- VGR-R01-301
- Status last verified
- Nov 2025
- First submitted
- 19 Nov 2024
- Last updated
- 24 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared with other researchers when VGR-R01 is fully approved
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Randomized, Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of VGR-R01 in Subjects With Bietti Crystalline Dystrophy
- ClinicalTrials.gov ID
- NCT06699108
- Acronym / sponsor ID
- VGR-R01-301
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Dec 2024 (actual)
- Primary completion
- 1 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 21 Nov 2024 (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 phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy.
This is a multicenter, randomized controlled study which will enroll 45 subjects.
Detailed description
VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to assess the efficacy of VGR-R01 in subjects with Bietti crystalline dystrophy (BCD) based on the change from baseline in best corrected visual acuity (BCVA) of the study eyes.
30 subjects will be enrolled in the intervention group and will receive monocular administration of VGR-R01, and 15 subjects in the control group will not receive any intervention. After completion of the trial, the untreated eyes will receive administration of VGR-R01 in subsequent clinical trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| interventional group | Experimental | Drug: VGR-R01 |
| Control group | No intervention | – |
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Able to provide informed consent and comply with requirements of the study;
- ≥18 years and <70 years of age;
- Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
- Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study eye;
Key exclusion criteria (10 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-R01Subretinal injection of VGR-R01 (0.1 mL) | Drug | interventional group |
Primary outcome
Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS
Time frame: Baseline up to Week 52
Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls
6 secondary outcomes
Change from baseline in BCVA
Time frame: Baseline up to Week 52
Change from baseline in multi-luminance mobility test (MLMT) score of the study eye
Time frame: Baseline up to Week 52
Changes from baseline in Microperimetry indexes
Time frame: Baseline up to Week 52
Change from baseline in optical coherence tomography (OCT)
Time frame: Baseline up to Week 52
Counts, frequencies and percentages of AE, SAE and other safety evaluation
Time frame: Baseline up to Week 52
Number of subjects with the presence of immunogenicity
Time frame: Baseline up to Week 52
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 24 Dec 2025.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when VGR-R01 is fully approved
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing VGR-R01 in people with Bietti Crystalline Dystrophy and Inherited Retinal Diseases, run by Shanghai Vitalgen BioPharma Co., Ltd. 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 4 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 24 Dec 2025. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS (time frame: Baseline up to Week 52). 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 24 Dec 2025.