Clinical trial NCT05694598: Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
Official title: A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Bietti Crystalline Dystrophy
Lead sponsor: Shanghai Vitalgen BioPharma Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2023-01-23
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

Detailed description: VGR-R01 is a novel AAV vector carrying the human CYP4V2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo at least 52 weeks of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five years.

Eligibility criteria: 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 outcomes:
measure: Incidence of adverse events
timeFrame: Baseline up to Week 52
measure: Incidence of serious adverse events
timeFrame: Baseline up to Week 52
measure: Number of Participants with Clinically Significant Change from Baseline in Vital Signs
timeFrame: Baseline up to Week 52
measure: Number of Participants with Clinically Laboratory Abnormalities
timeFrame: Baseline up to Week 52
measure: Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings
timeFrame: Baseline up to Week 52
Secondary outcomes:
measure: Best-Corrected Visual Acuity (BCVA)
timeFrame: Week 52
measure: Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes
timeFrame: Week 52
measure: Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes
timeFrame: Week 52
measure: Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes
timeFrame: Week 52
measure: Changes from baseline in Mobility testing scores
timeFrame: Week 52
Other outcomes:
measure: Patient reported outcome: Changes of NEI-VFQ-25
timeFrame: Week 52
Locations:
city: Shanghai
country: China
facility: Shanghai Vitalgen Biopharma Co.,Ltd.
state: Shanghai
Study officials:
affiliation: Beijing Tongren Hospital
name: Wenbin Wei
role: PRINCIPAL_INVESTIGATOR
References: