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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose level 1 | Experimental | Dose level 1 will be administered |
|
| Dose level 2 | Experimental | Dose level 2 will be administered |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NGGT001 | Genetic | Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration |
|
| Measure | Description | Time Frame |
|---|---|---|
| BCVA | ETDRS visual acuity | 1 year |
| Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) | Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) | 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Li | Xiamen Ophthalmology Center Affiliated to Xiamen University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Xiuju Chen | Xiamen | Fujian | 361000 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39786763 | Derived | Chen X, Liu X, Cui S, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2025 Feb 1;143(2):126-133. doi: 10.1001/jamaophthalmol.2024.5619. |
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| ID | Term |
|---|---|
| C535440 | Bietti Crystalline Dystrophy |
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