UnknownEarly Phase 1

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

China3 participantsStarted 2022-09-15

Plain-language summary

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

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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;

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence of adverse events

Timeframe: Baseline up to Day 365

Incidence of serious adverse events

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Significant Change from Baseline in Vital Signs

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Laboratory Abnormalities

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings

Timeframe: Baseline up to Day 365

Trial details

NCT IDNCT05399069

SponsorBeijing Tongren Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-05

Contact for this trial

Wenbin Wei

010-58269516 tr_weiwenbin@163.com

View on ClinicalTrials.gov More Bietti Crystalline Dystrophy trials

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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;

What they're measuring

Incidence of adverse events

Timeframe: Baseline up to Day 365

Incidence of serious adverse events

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Significant Change from Baseline in Vital Signs

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Laboratory Abnormalities

Timeframe: Baseline up to Day 365

Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings

Timeframe: Baseline up to Day 365