Clinical Trials in China / NCT06699108
Active, not recruiting
Phase 3
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
NCT06699108 · tracked via the Priya Life Science China tracker
Condition(s) studied
Bietti Crystalline DystrophyInherited Retinal Diseases
Investigational drug(s) / intervention(s)
VGR-R01: Subretinal injection of VGR-R01 (0.1 mL)
Study 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.
Eligibility
Key Inclusion Criteria:
1. Able to provide informed consent and comply with requirements of the study;
2. ≥18 years and \<70 years of age;
3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
4. Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study 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. Unwilling or unable to have the planned follow-up;
8. Abnormal coagulation function or other clinically significant abnormal laboratory results;
9. Have malignancies or history of malignancies;
10. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
- Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS — Baseline up to Week 52
Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Shanghai Vitalgen Biopharma Co.,Ltd. | 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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A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)
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Other trials for the same condition
A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)
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Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
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Extension Study of the PUMCH-E101 Clinical Trial:Safety and Efficacy Study of PUMCH-E101 Injection in the Contralateral Eye
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Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
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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 NCT06699108 on ClinicalTrials.gov ↗ ← All trials in China