Clinical Trials in China / NCT07586306
Starting soon
Phase 1/2
A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)
NCT07586306 · tracked via the Priya Life Science China tracker
Condition(s) studied
Bietti Crystalline Dystrophy
Investigational drug(s) / intervention(s)
VGR-R01: CYP4v2-coding gene delivered by AAV vector
Study 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.
Eligibility
Key Inclusion Criteria:
1. Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
2. Able to provide informed consent and comply with requirements of the study;
3. Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.
Key Exclusion Criteria:
1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;
2. Have current ocular or periocular infections, or endophthalmitis;
3. 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;
4. Have intraocular surgery history except cataract surgery in the study eye;
5. Have or potentially require of systemic medications that may cause eye injure;
6. Have contraindications for corticosteroids or immunosuppressant;
7. Abnormal coagulation function or other clinically significant abnormal laboratory results;
8. Have malignancies or history of malignancies;
9. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
- Incidence of adverse events and serious adverse events — 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 — 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.
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Beijing Tongren Hospital | Beijing | Beijing Municipality | |
| Shanghai General Hospital (Shanghai First People's Hospital) | Shanghai | Shanghai Municipality |
More Shanghai Vitalgen BioPharma Co., Ltd. trials in China
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency
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Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
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Other trials for the same condition
Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
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Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
Active, not recruiting
Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07586306 on ClinicalTrials.gov ↗ ← All trials in China