The purpose of this study was to evaluate the safety and efficacy of ZVS101e administered by subretinal injection in subjects with Bietti's crystalline dystrophy (BCD) and to select the optimal effective dose.
This is a single-arm, open-label, and multi-center study of ZVS101e in patients with BCD. Up to 24 subjects are expected to be enrolled. Each participant will receive ZVS101e by subretinal injection in one eye on a single occasion. Safety, efficacy and vector shedding characteristics of ZVS101e are then measured over 180 days. Participants will subsequently enter a long-term follow-up study over a 4.5-year period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
ZVS101e contains recombinant adeno-associated virus serotype 8 (rAAV8) vectors which carry human wild type CYP4V2 gene.
Peking University Third Hospital
Beijing, Beijing Municipality, China
West China Hospital, Sichuan University
Chengdu, Sichuan, China
Tianjin Medical University Eye Hospital
Tianjin, Tianjin Municipality, China
Incidence of ocular and systemic adverse events (AEs) after ZVS101e treatment
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.
Time frame: Up to day 180
Incidence of ocular and systemic serious adverse events (SAEs) after ZVS101e treatment
A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.
Time frame: Up to day 180
Mean change from baseline in BCVA (LogMAR)
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart.
Time frame: Up to day 180
Change from Baseline in visual field
Visual field will be assessed by Humphrey perimetry, changes in retinal light sensitivity will be analyzed.
Time frame: Up to day 180
Change from Baseline in contrast sensitivity
Change from baseline in contrast sensitivity at all spatial frequency will be measured using the CSV-1000E instrument.
Time frame: Up to day 180
Change from Baseline in microperimetry
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Microperimetry will be measured using MP-3,changes in retinal sensitivity (dB) will be analyzed.
Time frame: Up to day 180
Change from Baseline in mfERG
The measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV). Response Amplitude Density will be analyzed.
Time frame: Up to day 180
Change from Baseline in retinal thickness
Retinal thickness will be assessed for both eyes using OCT.
Time frame: Up to day 180
Change from Baseline in NEI VFQ-25 total score
National eye institute 25-item visual function questionnaire (NEI VFQ-25) consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively.
Time frame: Up to day 180
Change from Baseline in color vision
Subjects' color vision was classified and graded by having them identify the pictures within Color Vision Examination Plates.
Time frame: Up to day 180