The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of ZVS203e administered via subretinal injection in participants with RP caused by RHO site-specific gene mutation (RHO-RP).
This is a single-arm, open-label, single ascending dose study of ZVS203e in participants with RHO-RP. Up to 9 participants will be enrolled in this study. Safety, efficacy and vector shedding characteristics of ZVS203e are then measured.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
ZVS203e is a rAAV-mediated gene editing drug that silences RHO mutant protein expression by CRISPR/Cas9 editing system.
Peking University Third Hospital
Beijing, Beijing Municipality, China
RECRUITINGIncidence of adverse events (AEs)
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: Baseline up to Week 52
Incidence of serious adverse events (SAEs)
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: Baseline up to Week 52
Mean change from baseline in BCVA after ZVS203e treatment
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart.
Time frame: Baseline up to Week 52
Change from Baseline in visual field
Visual field will be assessed by Humphrey perimetry, changes in VFI, MD, PSD will be analyzed.
Time frame: Baseline up to Week 52
Change from Baseline in contrast sensitivity
Change from baseline in contrast sensitivity will be measured using the CSV-1000E instrument.
Time frame: Baseline up to Week 52
Change from Baseline in multi-luminance mobility test (MLMT)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
MLMT was assessed at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). The score range is between -1 (the worst) and 6 (the best).
Time frame: Baseline up to Week 52
Change from Baseline in retinal thickness
Retinal thickness will be assessed for both eyes using OCT.
Time frame: Baseline up to Week 52
Change from Baseline in fundus autofluorescence (FAF)
FAF is a noninvasive test to explore the health and metabolic status of retinal pigment epithelial cell/photoreceptor complex.
Time frame: Baseline up to Week 52
Change from Baseline in color vision
Subjects' color vision was classified and graded by Farnsworth Munsell 100 hue.
Time frame: Baseline up to Week 52
Change from Baseline in mfERG
The measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV).
Time frame: Baseline up to Week 52
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: Baseline up to Week 52