Clinical trial NCT06699108: Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Bietti Crystalline Dystrophy
Inherited Retinal Diseases
Lead sponsor: Shanghai Vitalgen BioPharma Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-11-21
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

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.

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. 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 outcomes:
measure: Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS
timeFrame: Baseline up to Week 52
Secondary outcomes:
measure: Change from baseline in BCVA
timeFrame: Baseline up to Week 52
measure: Change from baseline in multi-luminance mobility test (MLMT) score of the study eye
timeFrame: Baseline up to Week 52
measure: Changes from baseline in Microperimetry indexes
timeFrame: Baseline up to Week 52
measure: Change from baseline in optical coherence tomography (OCT)
timeFrame: Baseline up to Week 52
measure: Counts, frequencies and percentages of AE, SAE and other safety evaluation
timeFrame: Baseline up to Week 52
measure: Number of subjects with the presence of immunogenicity
timeFrame: Baseline up to Week 52
Other outcomes:
measure: Changes from baseline in visual field index
timeFrame: Baseline up to Week 52
measure: Change from baseline in PRO measures
timeFrame: Baseline up to Week 52
Locations:
city: Shanghai
country: China
facility: Shanghai Vitalgen Biopharma Co.,Ltd.
state: Shanghai
Study officials:
References: