Active — Not RecruitingEarly Phase 1

Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

China6 participantsStarted 2023-02-08

Plain-language summary

Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)

Who can participate

Age range

18 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

. Age ≥ 18 years old;
. Gender unlimited.
. Diagnosed as crystalline retinal degeneration (BCD).
. Molecular diagnosis confirms CYP4V2 mutation.
. The best corrected visual acuity is less than 20/60.
. Agree to take contraceptive measures within 1 year from the start of the study until administration.
. Voluntarily sign an informed consent form.

Exclusion criteria

. Insufficient number of photoreceptor cells in the retina, such as retinal thickness less than 100 μ m. Or no atrophy or pigmentation in the posterior pole area\&amp;lt;3- Retinal disc.
. The presence of choroidal neovascularization or other eye lesions caused by BCD, which researchers believe may affect surgical procedures or interfere with the interpretation of clinical endpoints.
. The use of therapeutic drugs within the first 6 months of enrollment may affect experimental observation, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, steroids, 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

BCVA

Timeframe: 1 year

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)

Timeframe: 1 year

Trial details

NCT IDNCT06302608

SponsorXiamen Ophthalmology Center Affiliated to Xiamen University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-05-29

View on ClinicalTrials.gov More Bietti's Crystalline Dystrophy trials