Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

NCT05399069 | Unknown Early Phase 1

• 1 other identifier: VGR-R01-001
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

Conditions

Bietti Crystalline Dystrophy

Outcome Measures

Primary Outcomes (5)

• 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.

  Baseline up to Day 365

• 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.

  Baseline up to Day 365

• 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.

  Baseline up to Day 365

• 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.

  Baseline up to Day 365

• 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.

  Baseline up to Day 365

Secondary Outcomes (8)

• Best-Corrected Visual Acuity (BCVA)

  Up to Day 365

• Changes from baseline in Visual Field indexes

  Up to Day 365

• Changes from baseline in Microperimetry indexes

  Up to Day 365

• Changes from baseline in ERG indexes

  Up to Day 365

• Changes from baseline in m-ERG indexes

  Up to Day 365

Study Arms (1)

VGR-R01

EXPERIMENTAL

Subretinal injection of VGR-R01

Drug: VGR-R01

Interventions

VGR-R01 DRUG

Gene Replacement Therapy

VGR-R01

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥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;
• 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. -

You may not qualify if:

• 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;

Sponsors & Collaborators

• Beijing Tongren Hospital: lead

Study Sites (1)

Beijing Tongren Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Officials

• Wenbin Wei

  Vice President of Beijing Tongren Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wenbin Wei

CONTACT

010-58269516; tr_weiwenbin@163.com

Xiuli Zhao

CONTACT

010-58268486-8008; xiulizhao@medmail.com.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 11, 2022
• First Posted: June 1, 2022
• Study Start: September 15, 2022
• Primary Completion: May 1, 2024
• Study Completion: May 1, 2024
• Last Updated: November 9, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: IPD will be shared with other researchers when VGR-R01 is fully approved.
• Access Criteria: IPD will be shared with other researchers when VGR-R01 is fully approved.

Locations

CN (1)