- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05399069
Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
November 8, 2022 updated by: Beijing Tongren Hospital
An Open-Label Safety Study of Single-dose VGR-R01 in Patients With Bietti Crystalline Dystrophy
An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
Study Overview
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 365(±7) days 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.
Study Type
Interventional
Enrollment (Anticipated)
3
Phase
- Early Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Wenbin Wei
- Phone Number: 010-58269516
- Email: tr_weiwenbin@163.com
Study Contact Backup
- Name: Xiuli Zhao
- Phone Number: 010-58268486-8008
- Email: xiulizhao@medmail.com.cn
Study Locations
-
-
Beijing
-
Beijing, Beijing, China, 100730
- Recruiting
- Beijing Tongren Hospital
-
Principal Investigator:
- Xiu-Li Zhao, PhD
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 80 years (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- ≥18 years and <80 years of age;
- Confirmed diagnosis of Bietti Crystalline Dystrophy;
- Molecular diagnosis of CYP4v2 mutations (homozygotes or compound heterozygotes);
- BCVA ≤ 20/200 in the study eye;
- -8 D <diopters< +8 D, 21 mm < axial lengths ≤ 28 mm in the study eye;
- Normal liver function and renal function;
- Agree to use reliable barrier contraception for 1 year after administration of VGR-R01;
- Able to provide informed consent and comply with requirements of the study. -
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;
- Prior medications which may interfere with the interpretation of study endpoints within six months before screening, eg. anti-VEGF drugs;
- Have or potentially require of systemic medications that may cause eye injure;
- Live attenuated vaccines is expected to be required during the study;
- Participation in a clinical study with an investigational drug or medical device within three months before enrollment;
- History of allergy or sensitivity to investigational drug, medications planned for use in the study;
- Use of anticoagulants, or after 10 days cessation of anti-platelet agents the platelet function does not recover;
- Use of any corticosteroids, other immunosuppressive drug(s) or antipsychotic drugs (eg. antidepressant, etc.) within 3 months prior to enrollment;
- Have contraindications for corticosteroids or immunosuppressant;
- Have complicating systemic diseases that would preclude 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);
- Females in lactation period;
- Have a history of alcohol or illicit drug addiction;
- Unable or unwilling to comply with the schedule of visits. -
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: VGR-R01
Subretinal injection of VGR-R01
|
Gene Replacement Therapy
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of adverse events
Time Frame: Baseline up to Day 365
|
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.
|
Baseline up to Day 365
|
Incidence of serious adverse events
Time Frame: Baseline up to Day 365
|
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.
|
Baseline up to Day 365
|
Number of Participants with Clinically Significant Change from Baseline in Vital Signs
Time Frame: Baseline up to Day 365
|
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.
|
Baseline up to Day 365
|
Number of Participants with Clinically Laboratory Abnormalities
Time Frame: Baseline up to Day 365
|
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.
|
Baseline up to Day 365
|
Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings
Time Frame: Baseline up to Day 365
|
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.
|
Baseline up to Day 365
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Best-Corrected Visual Acuity (BCVA)
Time Frame: Up to Day 365
|
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.
Initially, letters are read at a distance of 4 meters from the chart.
If <20 letters are read at 4 meters, testing at 1 meter should be performed.
BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye.
The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity).
An increase in the number of letters read correctly means that vision has improved.
|
Up to Day 365
|
Changes from baseline in Visual Field indexes
Time Frame: Up to Day 365
|
The outcome measeures will be assessed by Humphery perimetry
|
Up to Day 365
|
Changes from baseline in Microperimetry indexes
Time Frame: Up to Day 365
|
The Microperimetry will be applied to assess the retinal sensitivity and the fixation behaviour.
|
Up to Day 365
|
Changes from baseline in ERG indexes
Time Frame: Up to Day 365
|
The measeurement will be performed based on the standards of International Society for Clinical Electrophysiology of Vision (ISCEV)
|
Up to Day 365
|
Changes from baseline in m-ERG indexes
Time Frame: Up to Day 365
|
The measeurement will be performed based on the standards of International Society for Clinical Electrophysiology of Vision (ISCEV)
|
Up to Day 365
|
Change from Baseline in NEI Visual Function Questionnaire (NEI-VFQ-25)
Time Frame: Up to Day 365
|
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.
|
Up to Day 365
|
Changes from Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT)
Time Frame: Up to Day 365
|
Up to Day 365
|
|
Changes from Baseline in Optical Coherence Tomography Angiography(OCT-A)
Time Frame: Up to Day 365
|
Up to Day 365
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Wenbin Wei, Vice President of Beijing Tongren Hospital
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
September 15, 2022
Primary Completion (ANTICIPATED)
May 1, 2024
Study Completion (ANTICIPATED)
May 1, 2024
Study Registration Dates
First Submitted
May 11, 2022
First Submitted That Met QC Criteria
May 26, 2022
First Posted (ACTUAL)
June 1, 2022
Study Record Updates
Last Update Posted (ACTUAL)
November 9, 2022
Last Update Submitted That Met QC Criteria
November 8, 2022
Last Verified
April 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VGR-R01-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD 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.
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Bietti Crystalline Dystrophy
-
Peking University Third HospitalRecruitingBietti's Crystalline DystrophyChina
-
Shanghai Vitalgen BioPharma Co., Ltd.Not yet recruitingBietti Crystalline DystrophyChina
-
Xiamen Ophthalmology Center Affiliated to Xiamen...Active, not recruitingBietti's Crystalline DystrophyChina
-
Chigenovo Co., LtdRecruitingBietti's Crystalline DystrophyChina
-
Beijing Tongren HospitalRecruitingBietti's Crystalline DystrophyChina
-
Yune ZhaoCompletedCrystalline Cataract | Lens; AnomalyChina
-
Avidity Biosciences, Inc.RecruitingPhase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)Muscular Dystrophies | Muscular Dystrophy, Facioscapulohumeral | FSHD | Facio-Scapulo-Humeral Dystrophy | FMD | Facioscapulohumeral Muscular Dystrophy 1 | FSHD2 | FSHD1 | FMD2 | Fascioscapulohumeral Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy Type 1 | Fascioscapulohumeral Muscular Dystrophy... and other conditionsUnited States, United Kingdom, Canada
-
Manhattan Eye, Ear & Throat HospitalNorthwell HealthWithdrawnPattern Dystrophy of MaculaUnited States
-
Massachusetts General HospitalUniversity of Pittsburgh; Boston Children's Hospital; Brigham and Women's Hospital and other collaboratorsRecruitingDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Myotonic Dystrophy | Facioscapulohumeral Muscular DystrophyUnited States
-
Wyeth is now a wholly owned subsidiary of PfizerCompletedBecker Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy | Limb-Girdle Muscular DystrophyUnited States
Clinical Trials on VGR-R01
-
Shanghai Vitalgen BioPharma Co., Ltd.Not yet recruitingBietti Crystalline DystrophyChina
-
Sir Run Run Shaw HospitalHangzhou Neoantigen Therapeutics Co., Ltd.RecruitingDigestive System NeoplasmsChina
-
Sir Run Run Shaw HospitalHangzhou Neoantigen Therapeutics Co., Ltd.Recruiting
-
Stanford UniversityPliant Therapeutics, Inc.RecruitingIdiopathic Pulmonary Fibrosis | Primary Sclerosing Cholangitis | Covid19 PneumoniaUnited States
-
Sir Run Run Shaw HospitalHangzhou Neoantigen Therapeutics Co., Ltd.RecruitingDigestive System NeoplasmsChina
-
Stanford UniversityNational Cancer Institute (NCI)CompletedPancreatic Carcinoma | Healthy SubjectUnited States