Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-08

Study Completion Date

2028-05-29

Brief Summary

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

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Detailed Description

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Conditions

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Bietti's Crystalline Dystrophy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose level 1

Dose level 1 will be administered

Group Type EXPERIMENTAL

NGGT001

Intervention Type GENETIC

Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration

Dose level 2

Dose level 2 will be administered

Group Type EXPERIMENTAL

NGGT001

Intervention Type GENETIC

Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration

Interventions

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NGGT001

Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

1. 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.
2. 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.
3. 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;
4. The treatment eye has undergone intraocular surgery, such as PDT, vitrectomy, periocular vascular bypass surgery, etc., or requires intraocular surgery during clinical research, such as cataract surgery, retinal laser therapy, etc;
5. Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc;
6. Highly sensitive or allergic to the ingredients in the experimental drug (with a history of allergies to two or more drugs or foods);
7. Abnormal and clinically significant physical examination, vital signs, laboratory tests (such as blood routine, urine routine, blood biochemistry, coagulation function, immunological tests, etc.), or abnormal indicators deemed clinically significant by researchers;
8. There are diseases or medical histories that may affect drug safety or internal processes, especially cardiovascular, liver, kidney, endocrine, digestive, lung, neurological, hematological, tumor, immune or metabolic disorders that researchers consider clinically significant.
9. Participated in clinical trials of other drugs or medical devices within 3 months prior to enrollment;
10. Female patients during pregnancy or lactation;
11. Other researchers believe that it is not suitable to participate in the study.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No