Clinical Trials in China / NCT05694598
Active, not recruiting
Phase 1
Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
NCT05694598 · tracked via the Priya Life Science China tracker
Condition(s) studied
Bietti Crystalline Dystrophy
Investigational drug(s) / intervention(s)
VGR-R01
VGR-R01: CYP4v2-coding gene delivered by AAV vector
Study summary
A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
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. 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)
- Incidence of adverse events — Baseline up to Week 52
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment. - Incidence of serious adverse events — Baseline up to Week 52
A serious adverse event (SAE) is any untoward medical occurrence at any dose that resulted in death; life threatening; require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect. - Number of Participants with Clinically Significant Change from Baseline in Vital Signs — Baseline up to Week 52
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 5 minutes. Clinical significance of vital signs will be determined at the investigator's discretion. - Number of Participants with Clinically Laboratory Abnormalities — Baseline up to Week 52
Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, 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 Ophthalmic Examination Findings — Baseline up to Week 52
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.
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
Active, not recruiting
A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)
Starting soon
Other trials for the same condition
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 NCT05694598 on ClinicalTrials.gov ↗ ← All trials in China