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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration.
Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)
Chongqing, Chongqing Municipality, China
Xiamen Eye Center of Xiamen University
Xiamen, Fujian, China
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.
Time frame: 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.
Time frame: Week 12, Week 26 and Week 52
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.
Time frame: Week 12, Week 26 and Week 52
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).
Time frame: 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).
Time frame: 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
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Time frame: Week 12, Week 26 and Week 52