Back to resultsSafety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
Primary Purpose
Bietti Crystalline Dystrophy
Status
Not yet recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
VGR-R01
Sponsored by
About this trial
This is an interventional treatment trial for Bietti Crystalline Dystrophy
Eligibility Criteria
Key Inclusion Criteria: Able to provide informed consent and comply with requirements of the study; ≥18 years and <70 years of age; Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes); BCVA ≤ 60 ETDRS letters in the study eye. Key Exclusion Criteria: Have insufficient viable retinal photoreceptor cells based on investigator's decision; Have current ocular or periocular infections, or endophthalmitis; 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; Have intraocular surgery history except cataract surgery in the study eye; Have or potentially require of systemic medications that may cause eye injure; Have contraindications for corticosteroids or immunosuppressant; Unwilling or unable to have the planned follow-up; Abnormal coagulation function or other clinically significant abnormal laboratory results; Have malignancies or history of malignancies; History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- Shanghai Vitalgen Biopharma Co.,Ltd.
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
VGR-R01
Arm Description
Single-dose Subretinal Administration of VGR-R01
Outcomes
Primary Outcome Measures
Incidence of adverse events
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
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
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
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
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.
Secondary Outcome Measures
Best-Corrected Visual Acuity (BCVA)
BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured.
Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes
The VFI can range from 100% (normal visual field) to 0% (perimetrically blind field).
Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes
Normal values are typically within 0dB and -2dB.
Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes
A typical "normal" dB reading is around 30. The numeric dB graph should be studied next. The dBs tested by the Humphrey analyzer range between 0 and 50 dB (0 is the brightest and 50 is the dimmest).
Changes from baseline in Mobility testing scores
The mobility score range is between -1 (the worst functional vision) and 6 (the best).
Full Information
NCT ID
NCT05694598
First Posted
January 11, 2023
Last Updated
January 20, 2023
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05694598
Brief Title
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
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
March 1, 2023 (Anticipated)
Primary Completion Date
March 1, 2025 (Anticipated)
Study Completion Date
September 1, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bietti Crystalline Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
VGR-R01
Arm Type
Experimental
Arm Description
Single-dose Subretinal Administration of VGR-R01
Intervention Type
Genetic
Intervention Name(s)
VGR-R01
Intervention Description
CYP4v2-coding gene delivered by AAV vector
Primary Outcome Measure Information:
Title
Incidence of adverse events
Description
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.
Time Frame
Baseline up to Week 52
Title
Incidence of serious adverse events
Description
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.
Time Frame
Baseline up to Week 52
Title
Number of Participants with Clinically Significant Change from Baseline in Vital Signs
Description
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.
Time Frame
Baseline up to Week 52
Title
Number of Participants with Clinically Laboratory Abnormalities
Description
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.
Time Frame
Baseline up to Week 52
Title
Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings
Description
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.
Time Frame
Baseline up to Week 52
Secondary Outcome Measure Information:
Title
Best-Corrected Visual Acuity (BCVA)
Description
BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured.
Time Frame
Week 52
Title
Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes
Description
The VFI can range from 100% (normal visual field) to 0% (perimetrically blind field).
Time Frame
Week 52
Title
Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes
Description
Normal values are typically within 0dB and -2dB.
Time Frame
Week 52
Title
Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes
Description
A typical "normal" dB reading is around 30. The numeric dB graph should be studied next. The dBs tested by the Humphrey analyzer range between 0 and 50 dB (0 is the brightest and 50 is the dimmest).
Time Frame
Week 52
Title
Changes from baseline in Mobility testing scores
Description
The mobility score range is between -1 (the worst functional vision) and 6 (the best).
Time Frame
Week 52
Other Pre-specified Outcome Measures:
Title
Patient reported outcome: Changes of NEI-VFQ-25
Description
NEI-VFQ-25 questionnaire measures dimensions of self-reported vision-targeted health status that are most important to persons with eye disease. Total score ranges from 0-100, where a score of 0 represents the worst outcome and 100 represents the best outcome.
Time Frame
Week 52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
69 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Able to provide informed consent and comply with requirements of the study;
≥18 years and <70 years of age;
Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
BCVA ≤ 60 ETDRS letters in the study eye.
Key Exclusion Criteria:
Have insufficient viable retinal photoreceptor cells based on investigator's decision;
Have current ocular or periocular infections, or endophthalmitis;
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;
Have intraocular surgery history except cataract surgery in the study eye;
Have or potentially require of systemic medications that may cause eye injure;
Have contraindications for corticosteroids or immunosuppressant;
Unwilling or unable to have the planned follow-up;
Abnormal coagulation function or other clinically significant abnormal laboratory results;
Have malignancies or history of malignancies;
History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Min Li
Phone
086-18822167237
Email
m.li@vitalgen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Wenbin Wei
Organizational Affiliation
Beijing Tongren Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shanghai Vitalgen Biopharma Co.,Ltd.
City
Shanghai
State/Province
Shanghai
Country
China
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
IPD will be shared with other researchers when VGR-R01 is fully approved.
IPD Sharing Time Frame
IPD will be shared with other researchers when VGR-R01 is fully approved.
IPD Sharing Access Criteria
IPD will be shared with other researchers when VGR-R01 is fully approved.
Learn more about this trial
Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
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