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The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NGGT001 | Experimental | Single Arm: This study is a single-arm design in which all participants receive the NGGT001 gene therapy administered via subretinal injection. Participants are divided into three dose-escalation groups to evaluate safety and efficacy. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NGGT001 | Biological | Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events (AEs) from baseline to 52 weeks. | To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD. | 52 weeks |
| Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52. | To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12, 26 and 52. | Week 12, Week 26 and Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52. | Retinal sensitivity will be assessed using the MP-3 Type Microperimeter. This will be measured in dB (decibels), and the retinal sensitivity analysis will be conducted based on these readings. | Week 12, Week 26 and Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of Static Visual Fields (SVF) changes compared to baseline at Week 12, 26 and 52. | Static Visual Fields (SVF): Changes in visual field sensitivity will be evaluated at Weeks 12, 26 and 52, compared to baseline. The measurements will be expressed in light sensitivity units (dB). | Week 12, Week 26 and Week 52 |
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University) | Chongqing | Chongqing Municipality | 400000 | China |
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| ID | Term |
|---|---|
| C535440 | Bietti Crystalline Dystrophy |
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| Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52. |
Changes in contrast sensitivity (CS) will be evaluated, and the measurements will be analyzed in decibels (dB). |
| Week 12, Week 26 and Week 52 |
| Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52. | Retinal thickness will be measured using Optical Coherence Tomography (OCT), and the outcome will be expressed in micrometers (µm). | Week 12, Week 26 and Week 52 |
| Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52. | Multi-Luminance Mobility Test (MLMT) will assess subjects' daily life quality across different lighting conditions. The results will be categorized by the different light intensity levels, and the aggregate scores will be used to assess mobility performance | Week 12, Week 26 and Week 52 |
| Assessment of Fundus Autofluorescence (FAF) retinal structural changes compared to baseline at Week 12, 26 and 52. |
Retinal structural changes will be assessed at Week 12 and 26, compared to baseline. Fundus Autofluorescence (FAF) imaging will be used to evaluate the intensity of autofluorescence, and changes will be assessed using a dB threshold to measure variations over time in comparison to baseline. |
| Week 12, Week 26 and Week 52 |
| Assessment of Quality of Life Questionnaire score changes compared to baseline at Week 12, 26 and 52. | Quality of Life Questionnaire score will be measured at Week 12, 26 and 52, compared to baseline. | Week 12, Week 26 and Week 52 |
| Assessment of MNRead Test changes compared to baseline at Week 12, 26 and 52. | MNRead Test results will be evaluated at Week 12, 26 and 52, compared to baseline, assessing vision function. | Week 12, Week 26 and Week 52 |
| Xiamen Eye Center of Xiamen University | Xiamen | Fujian | 361000 | China |